HIF-2α

April 24, 2025 6:30 am ET Total Worldwide Sales Were $15.5 Billion, a Decrease of 2% From First Quarter 2024; Excluding the Impact of Foreign Exchange, Sales Grew 1% KEYTRUDA Sales Grew 4% to $7.2 Billion; Excluding the Impact of Foreign Exchange, Sales Grew 6% WINREVAIR Sales Were $280 Million Animal Health Sales Grew 5% to $1.6 Billion; Excluding the Impact of Foreign Exchange, Sales Grew 10% GARDASIL/GARDASIL 9 Sales Declined 41% to $1.3 Billion; Excluding the Impact of Foreign Exchange, Sales Declined 40% GAAP EPS Was $2.01; Non-GAAP EPS Was $2.22 Presented Compelling Data From a Diverse Range of Programs, Including: Phase 3 Trial of Subcutaneous Pembrolizumab With Berahyaluronidase Alfa Phase 3 ZENITH Trial of WINREVAIR for the Treatment of Adults With PAH (WHO Group 1) Functional Class III or IV at High Risk of Mortality Phase 3 Trials Evaluating Investigational, Once-Daily, Oral Two-Drug Regimen of Doravirine/Islatravir for the Treatment of Adults With Virologically Suppressed HIV-1 Infection Expanded Pipeline Through Exclusive License Agreement With Hengrui Pharma for an Investigational Oral Small Molecule Lp(a) Inhibitor; Transaction Expected to Close in Second Quarter 2025 Full-Year 2025 Financial Outlook Continues To Expect Worldwide Sales To Be Between $64.1 Billion and $65.6 Billion Now Expects Non-GAAP EPS To Be Between $8.82 and $8.97; Outlook Revised to Reflect Negative Impact From Anticipated One-Time Charge of Approximately $0.06 per Share Related to License Agreement With Hengrui Pharma Outlook Absorbs an Estimated $200 Million of Additional Costs for Tariffs Implemented to Date KEYTRUDA Sales Grew 4% to $7.2 Billion; Excluding the Impact of Foreign Exchange, Sales Grew 6% WINREVAIR Sales Were $280 Million Animal Health Sales Grew 5% to $1.6 Billion; Excluding the Impact of Foreign Exchange, Sales Grew 10% GARDASIL/GARDASIL 9 Sales Declined 41% to $1.3 Billion; Excluding the Impact of Foreign Exchange, Sales Declined 40% Phase 3 Trial of Subcutaneous Pembrolizumab With Berahyaluronidase Alfa Phase 3 ZENITH Trial of WINREVAIR for the Treatment of Adults With PAH (WHO Group 1) Functional Class III or IV at High Risk of Mortality Phase 3 Trials Evaluating Investigational, Once-Daily, Oral Two-Drug Regimen of Doravirine/Islatravir for the Treatment of Adults With Virologically Suppressed HIV-1 Infection Continues To Expect Worldwide Sales To Be Between $64.1 Billion and $65.6 Billion Now Expects Non-GAAP EPS To Be Between $8.82 and $8.97; Outlook Revised to Reflect Negative Impact From Anticipated One-Time Charge of Approximately $0.06 per Share Related to License Agreement With Hengrui Pharma Outlook Absorbs an Estimated $200 Million of Additional Costs for Tariffs Implemented to Date RAHWAY, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced financial results for the first quarter of 2025. “Our company made strong progress to start the year, with increasing contributions from our newer commercialized medicines and vaccines and continued advancement of our pipeline,” said Robert M. Davis, chairman and chief executive officer, Merck. “We are working with focus and urgency to both realize the full potential of our near-term opportunities and to rapidly progress the next wave of innovation that will positively impact the lives of patients and drive future value creation for all of our stakeholders.” Financial Summary $ in millions, except EPS amounts First Quarter 2025 2024 Change Change Ex- Exchange Sales $15,529 $15,775 -2% 1% GAAP net income1 5,079 4,762 7% 12% Non-GAAP net income that excludes certain items1,2* 5,611 5,279 6% 11% GAAP EPS 2.01 1.87 7% 13% Non-GAAP EPS that excludes certain items2* 2.22 2.07 7% 12% *Refer to table on page 7. For the first quarter of 2025, Generally Accepted Accounting Principles (GAAP) earnings per share (EPS) assuming dilution was $2.01 and non-GAAP EPS was $2.22. GAAP and non-GAAP EPS in the first quarter of 2024 include a charge of $0.26 per share for the acquisition of Harpoon Therapeutics, Inc. (Harpoon). Non-GAAP EPS excludes acquisition- and divestiture-related costs, costs related to restructuring programs, and income and losses from investments in equity securities. First-Quarter Sales Performance The following table reflects sales of the company’s top products and significant performance drivers. First Quarter $ in millions 2025 2024 Change Change Ex-Exchange Commentary Total Sales $15,529 $15,775 -2% 1% Pharmaceutical 13,638 14,006 -3% -1% Decline driven by vaccines, virology and immunology, partially offset by growth in oncology, cardiology and diabetes. KEYTRUDA 7,205 6,947 4% 6% Growth driven by increased global uptake in earlier-stage indications, including triple-negative breast cancer, renal cell carcinoma and non-small cell lung cancer, as well as continued strong global demand from metastatic indications, including increased uptake in bladder, endometrial and microsatellite instability-high (MSI-H) cancers, partially offset by timing of wholesaler purchases in the U.S. GARDASIL/GARDASIL 9 1,327 2,249 -41% -40% Decline primarily due to lower demand in China, partially offset by higher demand in most international regions, particularly in Japan, as well as higher pricing and demand in the U.S. Excluding China, sales grew 14%, or 16% excluding impact of foreign exchange. JANUVIA/JANUMET 796 670 19% 21% Increase primarily due to higher net pricing in the U.S., partially offset by lower demand in most international markets due to ongoing generic competition, and in the U.S. due to competitive pressure. PROQUAD, M-M-R II and VARIVAX 539 570 -5% -5% Decrease primarily reflects lower U.S. sales of PROQUAD that resulted from borrowing of doses from the U.S. Centers for Disease Control and Prevention Pediatric Vaccine Stockpile, partially offset by higher U.S. sales of M-M-R II largely attributable to private-sector buy-in due to measles outbreaks and higher pricing. BRIDION 441 440 - 1% Relatively flat compared with prior year, as higher demand and pricing in the U.S. were offset by lower demand in several international markets due to ongoing generic competition. Lynparza* 312 292 7% 8% Increase primarily due to higher demand in the U.S. and certain international markets. WINREVAIR 280 - - - Represents continued uptake since second-quarter 2024 launch in the U.S. Lenvima* 258 255 1% 2% Increase primarily due to higher demand in the U.S. VAXNEUVANCE 230 219 5% 7% Growth largely driven by higher demand in Europe and the Asia Pacific region, partially offset by lower demand in the U.S. due to competitive pressure. PREVYMIS 208 174 19% 22% Growth primarily due to higher demand in the U.S. WELIREG 137 85 62% 63% Growth primarily driven by higher demand in the U.S. CAPVAXIVE 107 - - - Represents continued uptake since third-quarter 2024 launch in the U.S. LAGEVRIO 102 350 -71% -69% Decline largely driven by lower demand in the Asia Pacific region, particularly in Japan. SIMPONI - 184 -100% -100% Marketing rights in former Merck territories reverted to Johnson & Johnson on Oct. 1, 2024. Animal Health 1,588 1,511 5% 10% Growth primarily due to higher demand for Livestock products, as well as inclusion of sales from Elanco aqua business that was acquired in July 2024. Livestock 924 850 9% 16% Growth primarily driven by higher demand across all species, a benefit from timing of ruminant product sales, as well as inclusion of sales from Elanco aqua business that was acquired in July 2024. Companion Animal 664 661 - 3% Sales consistent with prior year. Sales of BRAVECTO were $327 million and $332 million in current and prior year quarters, respectively, which represents decline of 1%, or growth of 2% excluding impact of foreign exchange. Other Revenues** 303 258 17% 16% Increase primarily due to higher payments received for out-licensing arrangements and higher royalties, partially offset by lower revenue from third-party manufacturing arrangements. *Alliance revenue for this product represents Merck’s share of profits, which are product sales net of cost of sales and commercialization costs. **Other revenues are comprised primarily of revenues from third-party manufacturing arrangements and miscellaneous corporate revenues, including revenue-hedging activities. First-Quarter Expense, EPS and Related Information The table below presents selected expense information. $ in millions GAAP Acquisition- and Divestiture- Related Costs3 Restructuring Costs (Income) Loss From Investments in Equity Securities Non- GAAP2 First Quarter 2025 Cost of sales $3,419 $620 $36 $ - $2,763 Selling, general and administrative 2,552 23 - - 2,529 Research and development 3,621 7 - - 3,614 Restructuring costs 69 - 69 - - Other (income) expense, net (35) (3) - (107) 75 First Quarter 2024 Cost of sales $3,540 $463 $116 $- $2,961 Selling, general and administrative 2,483 21 5 - 2,457 Research and development 3,992 16 2 - 3,974 Restructuring costs 123 - 123 - - Other (income) expense, net (33) (4) - (116) 87 GAAP Expense, EPS and Related Information Gross margin was 78.0% for the first quarter of 2025 compared with 77.6% for the first quarter of 2024. The increase was primarily due to the favorable impacts of product mix and lower restructuring costs, partially offset by higher amortization of intangible assets and the unfavorable impact of foreign exchange. Selling, general and administrative (SG&A) expenses were $2.6 billion in the first quarter of 2025, an increase of 3% compared with the first quarter of 2024. The increase was primarily due to higher administrative and promotional costs, partially offset by the favorable impact of foreign exchange. Research and development (R&D) expenses were $3.6 billion in the first quarter of 2025, a decrease of 9% compared with the first quarter of 2024. The decrease was primarily due to a $656 million charge for the acquisition of Harpoon in the first quarter of 2024 and the favorable impact of foreign exchange. The decrease was partially offset by a $100 million charge in the first quarter of 2025 associated with the achievement of a developmental milestone related to the 2024 acquisition of Eyebiotech Limited (EyeBio), increased compensation and benefit costs, higher clinical development costs, and increased discovery research and early drug development costs. Other (income) expense, net, was $35 million of income in the first quarter of 2025 compared with $33 million of income in the first quarter of 2024. The effective tax rate was 13.9% for the first quarter of 2025. GAAP EPS was $2.01 for the first quarter of 2025 compared with $1.87 for the first quarter of 2024. The increase was primarily driven by a $0.26 per share charge included in the first quarter of 2024 for the acquisition of Harpoon, partially offset by the unfavorable impact of foreign exchange. Non-GAAP Expense, EPS and Related Information Non-GAAP gross margin was 82.2% for the first quarter of 2025 compared with 81.2% for the first quarter of 2024. The increase was primarily due to the favorable impact of product mix, partially offset by the unfavorable impact of foreign exchange. Non-GAAP SG&A expenses were $2.5 billion in the first quarter of 2025, an increase of 3% compared with the first quarter of 2024. The increase was primarily due to higher administrative and promotional costs, partially offset by the favorable impact of foreign exchange. Non-GAAP R&D expenses were $3.6 billion in the first quarter of 2025, a decrease of 9% compared with the first quarter of 2024. The decrease was primarily due to a $656 million charge for the acquisition of Harpoon in the first quarter of 2024 and the favorable impact of foreign exchange. The decrease was partially offset by a $100 million charge in the first quarter of 2025 associated with the achievement of a developmental milestone related to the 2024 acquisition of EyeBio, increased compensation and benefit costs, higher clinical development costs, and increased discovery research and early drug development costs. Non-GAAP other (income) expense, net, was $75 million of expense in the first quarter of 2025 compared with $87 million of expense in the first quarter of 2024. The non-GAAP effective tax rate was 14.2% for the first quarter of 2025. Non-GAAP EPS was $2.22 for the first quarter of 2025 compared with $2.07 for the first quarter of 2024. The increase was primarily driven by a $0.26 per share charge included in the first quarter of 2024 for the acquisition of Harpoon, partially offset by the unfavorable impact of foreign exchange. A reconciliation of GAAP to non-GAAP net income and EPS is provided in the table that follows. First Quarter $ in millions, except EPS amounts 2025 2024 EPS GAAP EPS $2.01 $1.87 Difference 0.21 0.20 Non-GAAP EPS that excludes items listed below2 $2.22 $2.07 Net Income GAAP net income1 $5,079 $4,762 Difference 532 517 Non-GAAP net income that excludes items listed below1,2 $5,611 $5,279 Excluded Items: Acquisition- and divestiture-related costs3 $647 $496 Restructuring costs 105 246 Income from investments in equity securities (107) (116) Decrease to net income before taxes 645 626 Estimated income tax (benefit) expense4 (113) (109) Decrease to net income $532 $517 Pipeline and Portfolio Highlights In the first quarter, Merck continued to advance its broad and diverse pipeline, achieving key regulatory and clinical milestones across a range of therapeutic areas. In oncology, at the European Lung Cancer Congress 2025, Merck presented pivotal data from the 3475A-D77 Phase 3 trial evaluating the subcutaneous administration of pembrolizumab with berahyaluronidase alfa (subcutaneous pembrolizumab). Based on these data, applications for subcutaneous pembrolizumab are under review in the U.S. and Europe; in the U.S., the Prescription Drug User Fee Act (PDUFA) date is Sept. 23, 2025. If approved, subcutaneous pembrolizumab has the potential to become a new, meaningful treatment option that may increase access and save time needed for administration compared to intravenous (IV) KEYTRUDA. Merck also announced the initiation of waveLINE-010, a Phase 3 trial evaluating a combination regimen that incorporates zilovertamab vedotin, an investigational antibody-drug conjugate (ADC) targeting receptor tyrosine kinase-like orphan receptor 1, for the treatment of patients with previously untreated diffuse large B-cell lymphoma (DLBCL). Additional regulatory milestones include the U.S. Food and Drug Administration (FDA) granting Priority Review to Merck’s application for KEYTRUDA plus standard of care as perioperative treatment for resectable locally advanced head and neck squamous cell carcinoma (LA-HNSCC), following compelling results from the KEYNOTE-689 trial. The FDA has set a PDUFA date of June 23, 2025. In addition, Merck received approval from the European Commission (EC) for KEYTRUDA plus chemotherapy as first-line treatment for adult patients with unresectable non-epithelioid metastatic malignant pleural mesothelioma, based on results from the Phase 2/3 IND.227/KEYNOTE-483 trial. The company also received conditional EC approval for two indications for WELIREG, making it the first and only oral hypoxia-inducible factor-2 alpha inhibitor approved in the European Union, based on results from the Phase 2 LITESPARK-004 and Phase 3 LITESPARK-005 trials. In vaccines and infectious diseases, Merck received EC approval for CAPVAXIVE for the prevention of invasive pneumococcal disease and pneumococcal pneumonia in adults, marking the fourth approval for CAPVAXIVE following the U.S., Canada and Australia. Merck also received approval for GARDASIL 9 for males in China, making it the first 9-valent HPV vaccine approved for the prevention of certain HPV-related cancers and diseases in Chinese males 16-26 years of age. At the 32nd Conference on Retroviruses and Opportunistic Infections, Merck presented positive results from two pivotal Phase 3 trials of the investigational, once-daily, oral two-drug regimen of doravirine/islatravir (DOR/ISL) in adults with virologically suppressed HIV-1 infection. Merck plans to begin submitting applications for marketing authorization of DOR/ISL by mid-2025. In cardiovascular disease, results were presented from the Phase 3 ZENITH trial evaluating WINREVAIR when added to background therapy in adults with pulmonary arterial hypertension (PAH, Group 1 PH) WHO functional class (FC) III or IV at high risk of mortality. The results, presented at the American College of Cardiology’s 74th Annual Scientific Session and Expo, demonstrated that WINREVAIR reduced the risk of a composite of all-cause death, lung transplantation and hospitalization for PAH by 76% compared to placebo. The trial was stopped early due to overwhelming efficacy at the interim analysis. Merck continued executing on its business development strategy to expand and diversify its pipeline. The company entered into an exclusive license agreement with Jiangsu Hengrui Pharmaceuticals Co., Ltd. (Hengrui Pharma) for HRS-5346, an investigational oral small molecule Lipoprotein(a) [Lp(a)] inhibitor currently being evaluated in a Phase 2 clinical trial in China. The transaction is expected to close in the second quarter of 2025. Notable recent news releases on Merck’s pipeline and portfolio are provided in the table that follows. Oncology FDA Granted Priority Review for Merck’s Application for KEYTRUDA Plus Standard of Care as Perioperative Treatment for Resectable LA-HNSCC; Based on Results From Phase 3 KEYNOTE-689 Trial; FDA Set PDUFA Date of June 23, 2025 (Read Announcement) WELIREG Received First Conditional EC Approval for Two Indications; Based on Results From Phase 2 LITESPARK-004 and Phase 3 LITESPARK-005 Trials (Read Announcement) Merck’s Investigational Subcutaneous Pembrolizumab With Berahyaluronidase Alfa Demonstrated Noninferior Pharmacokinetics Compared to IV KEYTRUDA in Pivotal 3475A-D77 Trial; FDA Set PDUFA Date of Sept. 23, 2025 (Read Announcement) Merck Announced Phase 3 waveLINE-010 Trial Initiation Evaluating Zilovertamab Vedotin, an Investigational ADC, for the Treatment of Patients With Previously Untreated DLBCL (Read Announcement) Vaccines EC Approved CAPVAXIVE for Prevention of Invasive Pneumococcal Disease and Pneumococcal Pneumonia in Adults (Read Announcement) Cardiovascular WINREVAIR Reduced Risk of a Composite of All-Cause Death, Lung Transplantation and Hospitalization for PAH by 76% Compared to Placebo in Phase 3 ZENITH Trial (Read Announcement) Infectious Diseases Merck Announced Positive Data From Phase 3 Trials That Show the Investigational, Once-Daily, Oral Two-Drug Regimen of Doravirine/Islatravir (DOR/ISL) Maintained HIV-1 Viral Suppression at Week 48 (Read Announcement) Manufacturing and R&D Investment Merck is making long-term investments in its U.S. manufacturing and R&D capabilities. Merck has allocated more than $12 billion toward U.S. capital investment since 2018 and expects to invest over $9 billion more by the end of 2028. This includes the recently announced opening of a new, $1 billion, 225,000-square-foot facility dedicated to vaccine manufacturing at Merck’s Durham, North Carolina, site. Upcoming Investor Event Merck will host an Oncology Investor Event to coincide with the American Society for Clinical Oncology Annual Meeting on Monday, June 2, 2025, 6 p.m. CT, at which senior management will provide an update on the company’s oncology strategy and program. The event will take place in Chicago and will be accessible via live audio webcast at this weblink. Full-Year 2025 Financial Outlook The following table summarizes the company’s full-year financial outlook. Full Year 2025 Updated Prior Sales* $64.1 billion to $65.6 billion $64.1 billion to $65.6 billion Non-GAAP Gross margin2 Approximately 82% Approximately 82.5% Non-GAAPOperating expenses2** $25.6 billion to $26.6 billion $25.4 billion to $26.4 billion Non-GAAPOther (income) expense, net2 $300 million to $400 million expense $300 million to $400 million expense Non-GAAPEffective tax rate2 15.5% to 16.5% 16.0% to 17.0% Non-GAAPEPS2*** $8.82 to $8.97 $8.88 to $9.03 Share count (assuming dilution) Approximately 2.51 billion Approximately 2.53 billion *The company does not have any non-GAAP adjustments to sales. **Includes $300 million for an anticipated milestone payment to LaNova Medicines Ltd. (LaNova) associated with the technology transfer for MK-2010 and an anticipated $200 million upfront payment to Hengrui Pharma upon closing of the license agreement. Outlook does not assume any additional significant potential business development transactions. ***Includes expected one-time charges of approximately $0.15 per share in the aggregate related to the $300 million payment to LaNova upon completion of the technology transfer for MK-2010 and the $200 million upfront payment to Hengrui Pharma upon closing of the license agreement. Merck has not provided a reconciliation of forward-looking non-GAAP gross margin, non-GAAP operating expenses, non-GAAP other (income) expense, net, non-GAAP effective tax rate and non-GAAP EPS to the most directly comparable GAAP measures, given it cannot predict with reasonable certainty the amounts necessary for such a reconciliation, including intangible asset impairment charges, legal settlements, and income and losses from investments in equity securities either owned directly or through ownership interests in investment funds, without unreasonable effort. These items are inherently difficult to forecast and could have a significant impact on the company’s future GAAP results. Merck continues to expect full-year 2025 sales to be between $64.1 billion and $65.6 billion, including a revised negative impact of foreign exchange of approximately 1% at mid-April 2025 exchange rates. Merck’s outlook includes the impact of tariffs implemented to date by the U.S. government on imports from other countries, plus the tariffs imposed by foreign governments on the U.S., the most significant of which relate to China. Merck estimates the impact of these tariffs will lead to incremental costs of approximately $200 million, which will primarily be recorded in Cost of Sales, negatively impacting gross margin. Merck now expects its full-year non-GAAP effective income tax rate to be between 15.5% and 16.5%. Merck now expects its full-year non-GAAP EPS to be between $8.82 and $8.97, including a negative impact of foreign exchange of more than $0.20 per share. This revised non-GAAP EPS range now reflects an anticipated one-time charge of $200 million, or approximately $0.06 per share, for an upfront payment to be made upon closing of the license agreement with Hengrui Pharma, which is expected in the second quarter of 2025. If not for this newly anticipated charge, the updated non-GAAP EPS outlook would have been unchanged from the prior outlook. The guidance range continues to reflect an anticipated one-time charge of $300 million, or approximately $0.09 per share, related to the payment to LaNova that will be recognized upon completion of the technology transfer for MK-2010. In 2024, non-GAAP EPS of $7.65 was negatively impacted by a net charge of $1.28 per share related to certain asset acquisitions, licensing agreements and collaborations. Consistent with past practice, the financial outlook does not assume additional significant potential business development transactions. Earnings Conference Call Investors, journalists and the general public may access a live audio webcast of the call on Thursday, April 24, at 9 a.m. ET via this weblink. A replay of the webcast, along with the sales and earnings news release, supplemental financial disclosures and slides highlighting the results, will be available at www.merck.com. All participants may join the call by dialing (800) 369-3351 (U.S. and Canada Toll-Free) or (517) 308-9448 and using the access code 9818590. About Merck At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn. Forward-Looking Statement of Merck & Co., Inc., Rahway, N.J., USA This news release of Merck & Co., Inc., Rahway, N.J., USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions. The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s Annual Report on Form 10-K for the year ended December 31, 2024 and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site (www.sec.gov). Appendix Generic product names are provided below. Pharmaceutical BRIDION (sugammadex) CAPVAXIVE (Pneumococcal 21-valent Conjugate Vaccine) GARDASIL (Human Papillomavirus Quadrivalent [Types 6, 11, 16 and 18] Vaccine, Recombinant) GARDASIL 9 (Human Papillomavirus 9-valent Vaccine, Recombinant) JANUMET (sitagliptin and metformin HCl) JANUVIA (sitagliptin) KEYTRUDA (pembrolizumab) LAGEVRIO (molnupiravir) Lenvima (lenvatinib) Lynparza (olaparib) M-M-R II (Measles, Mumps and Rubella Virus Vaccine Live) PREVYMIS (letermovir) PROQUAD (Measles, Mumps, Rubella and Varicella Virus Vaccine Live) SIMPONI (golimumab) VARIVAX (Varicella Virus Vaccine Live) VAXNEUVANCE (Pneumococcal 15-valent Conjugate Vaccine) WELIREG (belzutifan) WINREVAIR (sotatercept-csrk) Animal Health BRAVECTO (fluralaner) ____________________ 1 Net income attributable to Merck & Co., Inc. 2 Merck is providing certain 2025 and 2024 non-GAAP information that excludes certain items because of the nature of these items and the impact they have on the analysis of underlying business performance and trends. Management believes that providing this information enhances investors’ understanding of the company’s results because management uses non-GAAP results to assess performance. Management uses non-GAAP measures internally for planning and forecasting purposes and to measure the performance of the company along with other metrics. In addition, annual employee compensation, including senior management’s compensation, is derived in part using a non-GAAP pretax income metric. This information should be considered in addition to, but not as a substitute for or superior to, information prepared in accordance with GAAP. For a description of the non-GAAP adjustments, see Table 2a attached to this release. 3 Reflects expenses related to business combinations, including the amortization of intangible assets, intangible asset impairment charges, and expense or income related to changes in the estimated fair value measurement of liabilities for contingent consideration. Also includes integration, transaction and certain other costs associated with acquisitions and divestitures, as well as amortization of intangible assets related to collaborations and licensing arrangements. 4 Includes the estimated tax impacts on the reconciling items based on applying the statutory rate of the originating territory of the non-GAAP adjustments. 1Q25 1Q24 $ 15,529 $ 15,775 -2% 3,419 3,540 -3% 2,552 2,483 3% 3,621 3,992 -9% 69 123 -44% (35 ) (33 ) 6% 5,903 5,670 4% 818 903 5,085 4,767 7% 6 5 $ 5,079 $ 4,762 7% $ 2.01 $ 1.87 7% 2,531 2,544 13.9 % 15.9 % $ 3,419 620 36 656 $ 2,763 2,552 23 23 2,529 3,621 7 7 3,614 69 69 69 – (35 ) (3 ) (107 ) (110 ) 75 5,903 (647 ) (105 ) 107 (645 ) 6,548 818 (117 ) (3 ) (18 ) (3 ) 22 (3 ) (113 ) 931 5,085 (530 ) (87 ) 85 (532 ) 5,617 5,079 (530 ) (87 ) 85 (532 ) 5,611 $ 2.01 (0.21 ) (0.03 ) 0.03 (0.21 ) $ 2.22 13.9 % 14.2 % 2025 2024 $15,529 $15,775 $16,112 $16,657 $15,624 $64,168 -2 1 13,638 14,006 14,408 14,943 14,042 57,400 -3 -1 7,205 6,947 7,270 7,429 7,836 29,482 4 6 312 292 317 337 365 1,311 7 8 258 255 249 251 255 1,010 1 2 137 85 126 139 160 509 62 63 119 71 90 100 110 371 68 68 1,327 2,249 2,478 2,306 1,550 8,583 -41 -40 539 570 617 703 594 2,485 -5 -5 230 219 189 239 161 808 5 7 228 216 163 193 139 711 5 7 107 47 50 97 - - 41 61 59 68 74 263 -33 -30 441 440 455 420 449 1,764 - 1 208 174 188 208 215 785 19 22 83 73 92 96 79 340 13 13 70 56 62 64 70 252 24 27 280 70 149 200 419 - - 106 98 106 102 109 415 8 8 68 70 72 72 73 287 -3 1 102 350 110 383 121 964 -71 -69 90 111 89 102 92 394 -19 -17 67 56 60 65 69 249 19 24 45 42 39 42 40 163 7 7 50 46 53 78 45 222 8 13 184 172 189 543 -100 -100 39 35 41 114 -100 -100 549 419 405 278 232 1,334 31 33 247 251 224 204 255 935 -2 2 729 632 618 638 699 2,590 16 18 1,588 1,511 1,482 1,487 1,397 5,877 5 10 924 850 837 886 889 3,462 9 16 664 661 645 601 508 2,415 - 3 303 258 222 227 185 891 17 16 Media Contacts: Robert Josephson robert.josephson@merck.com Michael Levey michael.levey@merck.com Investor Contacts: Peter Dannenbaum (732) 594-1579 Steven Graziano (732) 594-1583

Check back to this tracker for regulatory updates on marketed drugs, including expansions into new geographies and indications. Welcome to Fierce Pharma's regulatory tracker for the first half of 2025. On this page, we're recording the regulatory progress of in-market products, including expansions into key geographies and new indications. Be sure to come back regularly for the latest updates.UPDATED: Friday, March 14 at 9:25 a.m. ETChina's National Medical Products Administration (NMPA) has approved Innovent's Sycume as a treatment for thyroid eye disease (TED).The drug, also known as teprotumumab N01, becomes the first anti-insulin-like growth factor 1 receptor (IGF-1R) antibody to score approval in the country.In a press release, Innovent said the drug's approval "has ended a 70-year drought of no new treatment option for TED in China."In the U.S., teprotumumab is marketed by Amgen as Tepezza. In Europe, officials have expanded the label for Bristol Myers Squibb's cell therapy Breyanzi.Specifically, the European Commission approved the drug for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.The approval is based on results from the global phase 2 study called TRANSCEND FL, which enrolled patients with relapsed or refractory indolent non-Hodgkin lymphoma (NHL), including FL. In the third-line-plus treatment setting, 97.1% of patients responded to the drug, and 94.2% of patients achieved a complete response.Before this approval, Breyanzi had scored EU nods to treat certain patients with diffuse large B-cell lymphoma, high grade B-cell lymphoma and primary mediastinal large B-cell lymphoma.UPDATED: Thursday, March 13 at 8:30 a.m. ETKelun-Biotech's TROP 2 ADC sacituzumab tirumotecan (sac-TMT) has scored its second approval in China.The country's National Medical Products Administration (NMPA) approved the drug to treat certain adults with EGFR-positive locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC). The nod covers the drug's use in patients who have progressed after treatment with EGFR-tyrosine kinase inhibitor (TKI) therapy and platinum-based chemotherapy, Kelun said in a press release.With the approval, sac-TMT becomes the first TROP2 ADC approved to treat lung cancer anywhere in the world, according to the company.In a pivotal study, the drug "demonstrated a statistically significant and clinically meaningful improvement" in objective response rate, progression-free survival and overall survival compared with docetaxel, Kelun said. The study tested the drugs in patients with locally advanced or metastatic EGFR-mutant NSCLC who failed after prior treatment with an EGFR-TKI and platinum-based chemo.The approval is sac-TMT's second in the country after the NMPA approved it to treat certain patients with  triple negative breast cancer last year.In 2022, Merck & Co. licensed ex-China rights to the drug.UPDATED: Friday, March 7 at 9:35 a.m. ETThe European Commission (EC) has signed off on Bristol Myers Squibb’s immunotherapy combination of Opdivo and Yervoy to treat first-line patients with advanced or unresectable hepatocellular carcinoma (HCC).The nod was based on Opdivo and Yervoy showing an overall survival edge in the CheckMate-9DW trial over an investigator's choice of oral treatments.The Opdivo-Yervoy combo also faces an FDA decision date of April 21 to treat unresectable HCC. In 2020, the FDA granted accelerated approval to Opdivo-Yervoy to treat second-line advanced HCC.Opdivo and Yervoy were originally approved to treat melanoma, in 2014 and 2011 respectively. In combination, they have been blessed by the FDA in four other cancer types: renal cell carcinoma (2018), colorectal cancer (2018), mesothelioma (2020) and lung cancer (2020).Approximately 90% of liver cancer diagnoses are of the HCC type, BMS said. Liver cancer is the world’s third leading cause of cancer deaths.The FDA has expanded its approval for ALK’s immunotherapy Odactra for house dust mite (HDM) allergies to children ages 5 through 11. The under-the-tongue tablet was originally approved for adults in 2017 and then for adolescents ages 12 to 17 in 2023.UPDATED: Thursday, March 6 at 9:45 a.m. ETARS Pharmaceuticals needle-free epinephrine option, neffy nasal spray, can now be used to treat type 1 allergic reactions including anaphylaxis in children.To win the pediatric nod from the FDA, ARS ran studies proving that pharmacokinetic and pharmacodynamic responses from neffy in both children and adults were consistent with that of standard epinephrine injection products. The label expansion comes after the drug’s August approval as the first major innovation in epinephrine delivery in more than 35 years. Needle-free delivery is especially significant in the pediatric population as many children and caregivers fear needle-based auto-injectors, standing to delay lifesaving treatment.Human factor studies also show that children as young as 10 can use the nasal spray effectively and that untrained individuals, such as babysitters or teachers, can also correctly administer the drug. In the U.S., nearly 4 out of every 10 epinephrine prescriptions are for children under the age of 18 and some one in 13 children have severe food allergies, with more than 40% having experienced severe reactions, according to ARS. UPDATED: Wednesday, March 5 at 10:10 a.m. ETRoche's supplemental biologics application for Gazyva to treat lupus nephritis has been accepted for review by the FDA, the company said in a Wednesday press release.The filing leverages results from the phase 3 REGENCY study, which showed that 46.4% of patients with lupus nephritis who received Gazyva plus standard care achieved a complete renal response after 76 weeks. For patients who received standard care alone, the number was 33.1%, according to study results published last month.First approved in 2013, Gazyva is already cleared to treat chronic lymphocytic leukemia and follicular lymphoma in certain situations.The FDA is expected to make a decision on the lupus nephritis filing by October, according to Roche.UPDATED: Monday, March 3 at 4:30 p.m. ETDespite a reconsideration after a prior rejection, Australian drug regulators ultimately passed on Eisai's BioArtic-partnered Alzheimer’s disease treatment Leqembi. The Therapeutic Goods Administration (TGA) of Australia first decided against approval for patients with early Alzheimer’s disease in October. Eisai then requested reconsideration of the decision in December, proposing the same narrow indication given to the drug by the Medicines and Healthcare products Regulatory Agency (MHRA) and European Medicines Agency (EMA). Those approvals stipulate that Leqembi can only be used in patients with one or no copies of the ApoE4 gene due to safety concerns linking two copies of the gene to increased risks of brain swelling and bleeding, a side effect known as Amyloid Related Imaging Abnormalities (ARIAs).However, the TGA did not agree with the safety profile of Leqembi in ApoE4 heterozygotes, or those with one copy of the ApoE4 gene. Instead, the regulator offered an alternative narrow indication covering only ApoE4 noncarriers. Eisai floated other indications instead, including one that would keep the ApoE4 noncarrier and heterozygote patient populations but require heterozygotes to receive treatment in specialist centers with expert physician supervision. Still, the TGA rejected those options.BioArtic, Eisai’s long-time research partner that originally developed the antibody that became Leqembi, said it’s “very disappointed” by TGA’s decision, CEO Gunilla Osswald said in a statement. Eisai said it remains committed to ensuring eligible Australians can access the treatment and is “exploring options” to achieve this including a potential review by Australia’s Administrative Review Tribunal. UPDATED: Friday, Feb. 28 at 10:30 a.m. ETThe CDC said it's investigating five hospitalizations related to heart and nervous system events following vaccination with Valneva's Ixchiq among people 65 years and older. Ixchiq became the first FDA-approved chikungunya vaccine in November 2023. The CDC currently recommends the live-attenuated shot to be used for adults traveling to a country or territory where there is a chikungunya outbreak. Based on information from the CDC, at least four of the five individuals had also received other vaccines, and most of them had significant underlying medical conditions, a Valneva spokesperson said in a statement to Fierce Pharma."Valneva has not identified any safety signal concerns through post-marketing safety monitoring, including periodic safety reports and routine signal detection activities, and Ixchiq’s safety profile remains consistent with clinical trial data, unchanged and positive," the spokesperson said.The CDC said findings from its investigation will be discussed with experts at an Advisory Committee on Immunization Practices (ACIP) meeting. This year's first ACIP meeting, which was supposed to take place this week, was postponed with no new time provided by the CDC.The U.K. has approved Moderna's respiratory syncytial virus (RSV) vaccine mRESVIA to protect patients ages 60 and over against lower respiratory tract disease caused by the virus.In a study conducted in about 37,000 older adults, people who received mRESVIA had a 79% reduction in their risk of getting lower respiratory tract disease caused by RSV, compared with those who received placebo, around four months after vaccination.The shot will be manufactured at the Moderna Innovation and Technology Centre in Oxfordshire, which will be fully operational later this year, CEO Stéphane Bancel said in a statement.As is the case in the U.S., Moderna's mRNA shot will compete with GSK's Arexvy and Pfizer's Abrysvo in the U.K. as well.UPDATED: Thursday, Feb. 27 at 8:32 a.m. ETAfter being turned away by the FDA nearly one year ago, Regeneron this week said that the U.S. agency has accepted for review its biologics license application for bispecific antibody odronextamab.The FDA is expected to act on the application by July 30, Regeneron said in a Wednesday press release.In March of 2024, the agency rejected an accelerated approval filing for the drug based on the timeline of Regeneron's ongoing confirmatory trials. That decision spurred some confusion at Regeneron—and beyond—about a new requirement from the agency that confirmatory trials be "well underway" by the time it awards accelerated approvals.Early this year, the FDA sought to clear up the issue with new guidance on its expectations around accelerated approvals and confirmatory trial timelines.Nevertheless, Regeneron's review for the drug to treat patients with relapsed/refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy now appears back on track. In its latest press release, Regeneron said the resubmission "follows the achievement of an FDA-mandated enrollment target for the Phase 3 confirmatory trial." As the company notes, the enrollment target was the "sole approvability issue identified by the FDA" in its last submission.In Europe, regulators last year approved the drug for patients with R/R FL and R/R diffuse large B-cell lymphoma after two or more lines of systemic therapy. The medicine carries the brand name Ordspono in the region.In a phase 2 study, 80% of patients with FL who were treated with odronextamab responded to the therapy, with 74% achieving a complete response.UPDATED: Tuesday, Feb. 25 at 9:30 a.m. ETMerck's Keytruda, already boasting some 40 FDA approvals, has picked up a priority review in a proposed new use.The company's filing for the drug as part of a perioperative regimen for patients with resectable locally advanced head and neck squamous cell carcinoma has been accepted for a speedy review, Merck said on Tuesday.The filing seeks approval for Keytruda as a neoadjuvant treatment prior to surgery, followed by its administration as an adjuvant treatment in combination with standard of care radiotherapy—with or without cisplatin—after surgery. Afterward, the company's filing proposes Keytruda being used as a single agent.Merck's application leverages results from the KEYNOTE-689 trial, which showed that the perioperative regimen significantly reduced the risk of disease recurrence, worsening or death in certain patients with resected HNSCC compared with traditional postsurgical radiotherapy alone.The FDA is expected to act on the application by June 23.In Japan, Sanofi's Sarclisa has picked up an approval to treat newly diagnosed patients with multiple myeloma.Japan's Ministry of Health, Labour and Welfare (MHLW) approved the drug as part of a combination with bortezomib, lenalidomide, and dexamethasone (VRd) in the patient group based on results from the phase 3 IMROZ trial. The study showed that Sarclisa's addition to VRd significantly improved progression-free survival compared with VRd alone in patients with newly diagnosed, transplant-ineligible multiple myeloma.Sarclisa launched in 2020 in Japan and is approved as a part of four different regimens in the country, Sanofi noted in a press release.UPDATED: Monday, Feb. 24 at 10:52 a.m. ETGilead's application for twice-yearly lenacapavir has been accepted by the European Medicines Agency for accelerated review as an HIV pre-exposure prophylaxis (PrEP) option. Simultaneously, the drug will be assessed under EU-Medicines for all, which can be used to facilitate expedited review processes in countries outside the EU, including in low- and lower-middle-income countries. The FDA has put lenacapavir's PrEP application under priority review last week with a target decision date set for June 19, 2025.Several companies have launched their biosimilar products referencing Johnson & Johnson's star inflammatory disease drug Stelara in the U.S., following Amgen's January launch of Wezlana as the first biosimilar referencing the star IL-12/23 inhibitor. Teva and Alvotech just rolled out Selarsdi covering all indications and presentations of the original Stelara. Teva can start to claim interchangeability after an exclusivity period for Wezlana ends on April 30. Sandoz announced the launch of its Samsung Bioepis-partnered Pyzchiva, which the Swiss company views as a key driver in its global growth strategy. Sandoz touts Pyzchiva's extended stability, including the ability to be re-refrigerate, as an advantage over the original Stelara.In addition, Biocon Biologics has also entered the U.S. Stelara biosimilar market with Yesintek. It marks the Biocon subsidiary's "first product launch in the United States since becoming a fully integrated global biosimilars organization," CEO Shreehas Tambe said in a release. The FDA has granted priority review to Bristol Myers Squibb's application for Opdivo and Yervoy in first-line unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (mCRC). The goal date is June 23, 2025.The filing was based on data from the phase 3 CheckMate-8HW trial. The combo lowered the risk of disease progression or death by 38% versus Opdivo alone in MSI-H/dMMR mCRC, according to an update of the study presented in January.The FDA has updated the label of Indivior's Sublocade for severe opioid use disorder. The new label allows healthcare providers to start treatment with Sublocade after a single dose of transmucosal buprenorphine and a one-hour observation period. This rapid initiation "may lessen some of the practical obstacles to treatment induction," according to Indivior.In addition, the FDA has approved additional injection sites for Sublocade, an extended-release formulation of buprenorphine. The drug can now be administered in the abdomen, thigh, buttock or back of the upper arm.UPDATED: Friday, Feb. 21 at 9:30 a.m. ETGilead's seladelpar has secured a conditional marketing nod in Europe to treat adults with primary biliary cholangitis (PBC).The approval covers seladelpar's use in combination with ursodeoxycholic acid (UDCA) for those who have an inadequate response to UDCA alone, or as a monotherapy for people who can't tolerate UDCA.In the U.S., seladelpar picked up FDA approval last year and is branded as Livdelzi.A chronic autoimmune disease, PCB affects roughly 22 out of 100,000 people in Europe. The disease is more common in women and causes liver damage that can progress to liver failure if untreated. Symptoms include chronic itch and fatigue, and there is no cure for the disease.The approval is largely based on results from the phase 3 Response study, which showed that roughly three times as many patients on seladelpar achieved a composite biochemical response than those on placebo. The conditional approval will be contingent on positive results from future confirmatory studies.In Japan, authorities have approved CSL's subcutaneous Andembry for the prevention of acute attacks of hereditary angioedema (HAE).The drug offers patients the first pre-filled pen presentation for monthly subcutaneous administration for long-term prophylaxis of HAE, CSL said in a press release.The Japanese nod adds to the drug's other approvals in Europe, Australia and the U.K.UPDATED: Thursday, Feb. 20 at 10:00 a.m. ETBristol Myers Squibb's Breyanzi has won reimbursement backing from England's National Institute for Health and Care Excellence (NICE) in second-line large B-cell lymphoma, becoming the first CAR-T therapy to be recommended by the agency for this cancer type. The endorsement comes after NICE had originally rejected Breyanzi in October 2024 during its draft assessment. The drug cost watchdog changed its mind after BMS "offered an improved commercial arrangement" to the NHS based on its 297,000 pound sterling (about $375,000) list price per person, according to a press release.About 600 people in England could benefit from the therapy per year, according to NICE, which called the one-time CAR-T treatment "a significant advance in lymphoma care." In another reversal, NICE has also changed a previous rejection and signed off on UCB's Fintepla as an add-on treatment for seizures associated with Lennox-Gastaut syndrome (LGS) in people ages 2 years and above. The drug already has coverage under NHS for treating seizures from Dravet syndrome.LGS is a rare but severely debilitating form of epilepsy that begins in early childhood. By NICE's estimate, about 1,400 patients live with the disease in its jurisdiction.NICE changed its mind after UCB offered "an improved discount" to the drug's list price, the agency noted. Using a cost comparison approach, UCB convinced NICE's drug reviewers that Fintepla provides similar or greater health benefits compared to cannabidiol plus clobazam—which is already covered for LGS in the NHS—at a similar or lower cost.  The drug is covered only if the frequency of drop seizures is checked every six months, at which point the frequency is reduced by at least 30% compared with before starting treatment. UPDATED: Tuesday, Feb. 18 at 11:04 a.m. ETMerck’s Welireg snagged its first two approvals in Europe, with the nods covering certain patients with von Hippel-Lidau (VHL) disease and advanced clear renal cell carcinoma (RCC).The European Commission specifically approved the medicine as a treatment for those with VHL disease who have associated, localized RCC, central nervous system hemangioblastomas or pancreatic neuroendocrine tumors. The drug is approved in cases when localized procedures are unsuitable, making Welireg the first available treatment for VHL-associated tumors in Europe. The med also won a nod for adult patients with advanced clear cell RCC that has progressed following two or more lines of therapy, including a PD-1/L1 and at least two VEGF-targeted therapies.The European nods, which are conditional authorizations, follow similar approvals in the U.S. Merck will make the drug commercially available individual European countries after the complication of national reimbursement procedures, among other launch timing factors, the company said.Gilead’s twice-yearly pre-exposure prophylaxis (PrEP) candidate lenacapavir is on the FDA’s review table with a decision date of June 19.The drug, which is currently approved as Sunlenca for multi-drug resistant HIV, would be the first twice-yearly PrEP option on the market. The company’s FDA bid is the first step of its detailed global launch and access strategy.Gilead’s lenacapivir application is backed by two phase 3 trials, one of which showed an impressive 100% risk reduction. The company describes the med as a “foundation” for potential future HIV therapies, it said in a release.Sanofi and Regeneron’s immunology powerhouse Dupixent is aiming to secure a new use in chronic skin disease bullous pemphigoid (BP) with an FDA decision expected by June 20.If approved, the med would be the only targeted BP treatment available in the U.S. The disease is hallmarked by intense itch and blisters and impacts a population of around 27,000 adults, typically elderly adults, whose condition is not controlled by systemic corticosteroids.  The companies’ FDA bid has been granted priority review and is based on a study of 106 adults with moderate-to-severe BP. In the trial, five times more patients who received Dupixent achieved sustained disease remission compared to those on placebo, according to Sanofi. UPDATED: Friday, Feb. 14 at 9:20 a.m. ETCSL's hereditary angioedema (HAE) therapy Andembry has secured approval from the European Commission.The drug is the first once-monthly treatment targeting factor XIIa to prevent HAE attacks in patients 12 and older. In a phase 3 trial, the drug significantly reduced investigator-confirmed HAE attacks per month versus placebo, according to results published in The Lancet in April 2023.Last month, regulators in Australia and the U.K. signed off on the medicine."Andembry, CSL's first approved recombinant monoclonal antibody discovered and developed entirely by CSL, underscores our more than 40-year legacy in HAE research and treatment optimization and our decades-long journey to bring this innovation to patients," Bill Mezzanotte, M.D., CSL's head of R&D, said in a press release.Now, CSL plans to engage in access and reimbursement negotiations with national authorities across Europe as the company progresses with the rollout.Also in Europe, officials signed off on CSL and Arcturus Therapeutics' self-amplifying mRNA COVID-19 vaccine, Kostaive, for use in individuals ages 18 and older.The approval is based on results from an integrated phase 1/2/3 study and late-stage booster trials, the partners said in a press release. Late last year, the European Medicines Agency recommended the vaccine for approval.Besides Europe, the vaccine is approved in Japan.Galderma's efforts to expand the market for Nemluvio are bearing more fruit.In Europe, officials approved the subcutaneous drug for the treatment of moderate-to-severe atopic dermatitis in patients ages 12 and older. In addition, the drug scored an endorsement to treat prurigo nodularis in adults.In the late-stage Arcadia and Olympia clinical trial programs, the drug "significantly improved itch, skin lesions and sleep disturbance, in patients with moderate-to-severe atopic dermatitis and adults with prurigo nodularis, respectively," Galderma said in a Friday press release.The drug is also approved in the U.S. and is under review in Canada, Brazil, South Korea and other countries.China has approved its 22nd PD-1/L1 product. The approval went to SinoCellTech's finotonlimab for its use alongside chemo in first-line head and neck cancer. In a China phase 3 trial, the combo reduced the risk of death by 27% versus chemo alone, extending the median overall survival by 3.6 months to 14.1 months, according to results published in Nature Medicine.The go-ahead marks the first for a PD-1/L1 inhibitor that covers all head and neck cancer patients in China. In the country, Merck & Co.'s Keytruda is allowed as a monotherapy only in PD-L1-positive disease, SinoCellTech noted in a securities filing (Chinese). UPDATED: Wednesday, Feb. 12 at 10:20 a.m. ETAcoramidis, the transthyretin amyloid cardiomyopathy (ATTR-CM) drug developed by BridgeBio and licensed by Bayer in Europe, has been approved by the European Commission. Bayer plans to make the transthyretin stabilizer available in Europe under the brand name Beyonttra in the first half of this year.The approval is based on results from the phase 3 ATTRibute-CM study trial showing that Beyonttra significantly reduced the composite endpoint of all-cause mortality and cardiovascular-related hospitalizations compared with placebo. During the open-label extension phase of the study, Beyonttra demonstrated a relative risk reduction in deaths of 34% after 42 months.Beyonttra's launch is one of Bayer's “must-win battles” as the German company navigates the loss of exclusivity of its top-selling drug, blood thinner Xarelto, Stefan Oelrich, head of Bayer’s pharma division, said at the J.P. Morgan Healthcare Conference in January.Travere Therapeutics said it has aligned with the FDA on its plan to submit Filspari for traditional approval in focal segmental glomerulosclerosis (FSGS), a rare kidney disease. The move comes after a Filspari phase 3 trial in that indication failed on its primary endpoint of eGFR slope, a measurement of kidney disease progression. It also comes as findings from a collaborative global project called ARASOL linked a decrease in proteinuria over 24 months to a reduction in the risk of kidney failure in FSGS. Filspari has shown a benefit in lowering proteinuria. Travere plans to file this application around the end of March using existing data from the phase 3 DUPLEX and phase 2 DUET studies. The company will request a priority review, which Leerink Partners analysts believe is likely to be granted as there are no approved therapies for FSGS. Given that the understanding around proteinuria as a potential full approval endpoint is new, Leerink analysts said it would not be surprising if the FDA wanted to discuss the results during an advisory committee meeting.An approval in FSGS would be "transformational" for Filspari and Travere, Leerink said in a Tuesday note, because it could give the drug $1.1 billion in peak sales across the U.S. and EU based on a "relatively conservative peak penetration" of less than 20%. For Filspari sales, the FSGS indication could eventually surpass IgA nephropathy, for which Filspari is currently approved and is starting to face more competition, according to Leerink Partners.UPDATED: Monday, Feb. 10 at 9:45 a.m. ETBristol Myers Squibb’s cancer combo Opdivo plus Yervoy has scored an approval recommendation in the E.U., thanks to an endorsement from the EMA’s Committee for Medicinal Products for Human Use (CHMP) as a first-line treatment in adults with unresectable or advanced hepatocellular carcinoma.With the thumbs up from CHMP, the Opvido-Yervoy combo now moves forward to the European Commission (EC) for a final approval decision.The CHMP based its green light on results from the combo’s phase 3 CheckMate -9DW trial, in which patients on Opdivo and Yervoy charted a median overall survival of 23.7 months, compared to 20.6 months in patients who received an alternative regimen of Lenvima (lenvatinib) or sorafenib.BMS’ cancer combo is up for a similar approval in the U.S., with the FDA scheduled to make its decision on a potential first-line unresectable HCC nod by April 21.The drugmaker also won a CHMP endorsement for its CAR-T therapy Breyanzi to treat adults with relapsed or refractory follicular lymphoma (FL) who’ve previously tried two or more systemic therapies.BMS used data from its phase 2 TRANSCEND FL study to support the recommendation. In the trial, Breyanzi charted an overall response rate of 97.1% and a complete response rate of 94.2%, which was sufficient to meet the study’s primary and secondary endpoints, BMS said in a release.Breyanzi already carries multiple oncology approvals in Europe, including in relapsed or refractory diffuse large B-cell lymphoma and high grade B-cell lymphoma.Late last month, the FDA gave a thumbs up to German drugmaker medac’s alkylating agent Grafapex, also known as treosulfan, alongside the antimetabolite medicine fludarabine, as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adults and kids ages one and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).Alkylating agents cause breakage of DNA strand to help thwart the ability of cancer cells to multiply.Medac’s approval was backed up by a study looking primarily at overall survival in 570 adults ages 18 to 70 years old with AML or MDS who were randomized to receive either Grafapex or busulfan with fludarabine as a preparative regimen for allogeneic transplant. UPDATED: Wednesday, Feb. 5 at 9:05 a.m. ETNovartis' Kisqali has secured a label expansion from the U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA).The new approval covers Kisqali's use in combination with an aromatase inhibitor (AI) in patients with HR+/HER2- early breast cancer at high risk of recurrence. Previously, the drug was approved by the MHRA to treat advanced breast cancer, according to a Feb. 4 press release from Novartis.The latest approval is based on results from the phase 3 NATALEE trial, which showed that the addition of Kisqali to treatment with an AI cut the risk of invasive disease, recurrence, or death compared to AI treatment alone in patients with HR+/HER2- early breast cancer.Last fall, the drug picked up an FDA nod in patients with HR+/HER2- early breast cancer based on the NATALEE study results.Vaccine specialist Valneva has picked up an approval from the MHRA, as well.The agency approved the company's chikungunya vaccine, Ixchiq, for use in people 18 years of age and older. Late-stage trial data show the vaccine "induces a rapid and strong immune response," according to a press release from the company.Valneva manufactures the shot at its facility in Livingston, Scotland.Previously, the vaccine secured approvals in the United States, Canada and Europe. UPDATED: Friday, Jan. 31 at 9:35 a.m. ETVertex's groundbreaking launch of Casgevy keeps racking up more milestones around the globe.Friday, the company said it has reached a reimbursement agreement with NHS England that'll allow eligible patients with sickle cell disease access to the CRISPR/Cas9 gene-edited therapy. Along with the NHS England agreement, the National Institute for Health and Care Excellence (NICE) has signed off on the drug's use, Vertex said in a press release.The reimbursement deal follows a similar agreement, unveiled last summer, for the drug in patients with transfusion-dependent beta thalassemia.“We are pleased to have reached this new agreement that ensures both eligible SCD and TDT patients can now be treated with Casgevy, recognizing the value a one-time treatment can provide to patients, their families and the healthcare system," Vertex International senior vice president Ludovic Fenaux said in a statement.In its approval bid for Fabhalta in C3 glomerulopathy, Novartis will no longer face an FDA advisory committee meeting, CEO Vas Narasimhan said on Friday.The advisory committee meeting had been scheduled for next month but will no longer take place.Speaking with members of the media on Friday, Narasimhan said the "FDA no longer deemed it necessary to hold an advisory committee meeting.""We think it's based on, you know, the overall data package we've been able to generate, and the additional follow on data that we've provided the agency," the CEO continued. So we look forward to now bringing forward that medicine as a third indication approval for Fabhalta."Amid Europe's ongoing regulatory review of Eisai and Biogen's Alzheimer's disease medicine Leqembi, the European Commission (EC) has flagged new safety information for review.In November, Europe's Committee for Medicinal Products for Human Use (CHMP) recommended market approval of the drug. Since then, EC has asked the committee to "consider information on the safety of lecanemab that became available after the adoption of the CHMP opinion," the partners said in a Friday press release. The companies didn't reveal the nature of the new safety information flagged by the EC. CHMP will dig into the new info during a meeting next month, according to the release."We believe that the EC’s requests can be addressed with existing information and will be evaluated by the CHMP because of the clear and sufficient information available," the partners said in the release. "We will continue to work closely with the authorities toward approval in the EU."UPDATED: Monday, Jan. 27 at 10:43 a.m. ETMerck’s Welireg is up for a potential FDA label expansion by May 26. The agency granted a priority review to the company's bid to expand the drug's label into advanced, unresectable, or metastatic pheochromocytoma and paraganglioma (PPGL), which would add to currently approved indications in advanced renal cell carcinoma and von Hippel-Lindau disease.Welireg generated $139 million in sales during 2024’s third quarter and is the only approved HIF-2α inhibitor in the U.S.Merck’s application in PPGL is based on objective response rate and duration of response rate data from the phase 2 LITESPARK-015 trial, which enrolled 322 patients and will be presented at an upcoming medical meeting, according to the company. Up to 2,000 new cases of the tumors are diagnosed yearly in the U.S.Merck is evaluating Welireg across other rare oncologic diseases both on its own and in combination with other therapies. The FDA is evaluating Alvotech and Teva’s proposed biosimilar to Johnson & Johnson’s Simponi and Simponi Aria.Alvotech, which is responsible for development and manufacturing under its longstanding collaboration with Teva, announced positive top line results from its confirmatory trial of the Simponi copycat back in April. The partners expect the FDA to complete its review process in the fourth quarter of 2025. Simponi was first approved in 2009 and treats a variety of inflammatory conditions including rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis, among others. Simponi is an injected drug, while Simponi Aria is the infused version. The franchise picked up $2.19 billion for J&J in 2023 sales.Alvotech and Teva's product is the first biosimilar to the blockbuster arthritis med to be accepted for review.UPDATED: Thursday, Jan. 23 at 10:00 a.m. ETBiogen said its applications for a high-dose version of the spinal muscular atrophy med Spinraza have been accepted for review by the FDA and the European Medicines Agency. The new regimen comprises a more rapid loading regimen of two 50 mg doses given two weeks apart and a maintenance regimen given at 28 mg every four months. Currently, the drug's FDA-approved loading regimen features three doses given 14 days apart, plus a fourth dose 30 days after the third. The drug is currently approved in 12 mg doses.The high-dose applications are based on results from the phase 2/3 Devote trial, which favored the high-dose version versus an external sham control and the currently available dose on the measure of patients' reduction in plasma neurofilament light chain (NfL), a marker for neurodegeneration.The higher dose regimen also reduced the risk of death or permanent ventilation by 68% relative to sham and 30% compared with the 12 mg regimen.The high-dose Spinraza is Biogen's answer to competitors such as Novartis' gene therapy Zolgensma.Amneal has won FDA approvals for generics referencing AbbVie's Alzheimer's combo drug Namzaric and Novartis' cancer therapy Afinitor Disperz. Amneal has launched the Namzaric generic with 180-day market exclusivity.Separately, Amneal said it has won tentative approval from the FDA for a generic to Bausch Health's star irritable bowel syndrome med Xifaxan. Bausch Health has previously reached settlement agreements with Teva, Sun Pharma and Sandoz, pushing their generic entry dates into 2028 or upon an earlier launch of a rival generic product. Xifaxan is one of 15 drugs up for the second cycle of Medicare price negotiations under the Inflation Reduction Act. UPDATED: Tuesday, Jan. 21 at 8:57 a.m. ETThe European Commission has approved Johnson & Johnson's combination of Lazcluze and Rybrevant for the first-line treatment of certain adults with advanced non-small cell lung cancer (NSCLC), the company said in a press release.Specifically, the combo is approved in Europe for patients with epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or exon 21 L858R (L858R) substitution mutations.In approving the use, EU regulators reviewed results from the phase 3 MARIPOSA study. The trial showed that the combination reduced the risk of disease progression or death by 30% compared with AstraZeneca's Tagrisso in patients with newly diagnosed, EGFR-mutant non-small cell lung cancer. The results yielded a U.S. approval for the J&J combination last summer. Earlier this month, J&J touted an overall survival win from the study. At the time, a company executive heralded the regimen as "a new standard of care for patients with EGFR non-small cell lung cancer."Also in Europe, officials have endorsed GSK's Jemperli to treat all adult patients with primary advanced or recurrent endometrial cancer, including those with mismatch repair proficient (MMRp)/microsatellite stable (MSS) tumors. MMRp and MSS cases represent approximately 75% of patients diagnosed with endometrial cancer, GSK said in a press release.The approval covers Jemperli's use in combination with chemotherapy.Late last year, Europe's Committee for Medicinal Products for Human Use (CHMP) recommended the label expansion.In England, the National Institute for Health and Care Excellence (NICE) recommended AstraZeneca's Tagrisso for treating people with EGFR mutation-positive non-small-cell lung cancer (NSCLC) after surgery.The recommendation means that hundreds of patients will benefit from routine access to the drug, which is taken daily alongside chemotherapy, according to a notice from the Roy Castle Lung Cancer Foundation.Previously, EGFR mutation-positive NSCLC patients relied on chemotherapy to prevent cancer recurrence after surgery, according to the foundation.“I am pleased we have been able to recommend that this targeted treatment for a specific gene mutation of lung cancer will be routinely available on the NHS," NICE's director of medicines evaluation, Helen Knight, said in a statement.UPDATED: Thursday, Jan. 16 at 10:25 a.m. ETEli Lilly's Omvoh has picked up its second major FDA nod. After the IL-23 inhibitor's approval in 2023 to treat ulcerative colitis, the agency has now signed off on Omvoh to treat Crohn's disease. Specifically, the FDA approved Omvoh to treat moderately to severely active Crohn's disease in adults.In a phase 3 study, 53% of patients treated with Omvoh achieved clinical remission after a year of treatment. That number compared with 36% for those on placebo.In addition, 46% of patients treated with Omvoh had visible healing of the intestinal lining after a year. In the placebo group, the number was 23%.The trial enrolled patients with moderately to severely active Crohn's disease who had experienced an inadequate response, loss of response or who could not tolerate corticosteroids, immunomodulators and/or biologics.With the FDA nod, Lilly's drug will go up against some heavy hitters in the Crohn's space, including Johnson & Johnson's Stelara and AbbVie's duo of Skyrizi and Rinvoq.UPDATED: Wednesday, Jan. 15 at 8:12 a.m. ETSanofi's Sarclisa has gained approval from China's National Medical Products Administration (NMPA) to treat adults with multiple myeloma who've received at least one prior line of therapy, including lenalidomide and a proteasome inhibitor.The approval covers Sarclisa's use in combination with pomalidomide and dexamethasone (Pd).Regulators in China endorsed the drug based on results from the phase 3 ICARIA-MM study, as well as bridging data from a China-based study, Sanofi said in a press release. In ICARIA-MM, the addition of Sarclisa to Pd resulted in a 40% reduction in patients' risk of disease progression or death. "We look forward to continuing to build strong partnerships with the medical community, local companies, and authorities in China as we work to bring more innovative treatments to patients," Sanofi's head of oncology, Olivier Nataf, said in a statement.As Biogen and Eisai work to grow Leqembi in Alzheimer's disease, the FDA has accepted a biologics license application for the drug's subcutaneous maintenance dosing option. With a potential approval, Leqembi would become the first anti-amyloid therapy to offer at-home maintenance dosing.The FDA is set to make a decision on the filing by August 31, according to a Biogen press release.UPDATED: Thursday, Jan. 9 at 11:15 a.m. ETEngland’s National Institute for Health and Care Excellence (NICE) recommended AstraZeneca’s Lynparza for use in the region’s National Health Service, clearing the way for around 1,200 eligible patients to reap the benefits of the treatment.The recommendation covers Lynparza’s use in patients with HER2-negative, locally advanced or metastatic breast cancer who have BRCA1 or BRCA2 mutations and have had chemotherapy. NICE based its decision in part on results from the company’s OlympiAD phase 3 study, which proved that the drug could cut the risk of disease worsening or death by 42%.NICE had originally begun its assessment of the treatment in that indication in 2018, but ended it citing a lack of evidence submitted by the company. This time around, NICE assessed the med using a faster cost comparison process that compared Lynparza to Pfizer’s Talzenna in benefits and cost. Stallergenes Greer’s peanut allergy treatment Palforzia scored a label expansion in Europe, extending the drug’s use to toddlers aged 1 through 3 years old.Palforzia is now cleared for use in those aged 1 to 17 years old and is the only approved oral immunotherapy in the U.S. and Europe for toddlers with a confirmed peanut allergy diagnosis. The drug is meant to gradually increase the body’s ability to tolerate small amounts of peanuts through carefully controlled initial dose escalation and up-dosing.The European Commission based its nod on the company’s POSEIDON phase 3 study, which was a double-blind, placebo-controlled food challenge after six months of treatment. Agios Pharmaceuticals’ Pyrukynd is up for an FDA decision in non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia on Sept. 7.The company filed its bid for approval last month after finding success in two phase 3 trials assessing the drug in the non-transfusion-dependent and transfusion-dependent populations. Thalassemia is a rare, inherited blood disorder with limited treatment options. The disease is hallmarked by the abnormal production of hemoglobin, which causes chronic anemia and can lead to other complications.Pyruknd won its first approval in 2022 for pyruvate kinase (PK) deficiency, marking the first drug approved to treat the disease. Merck’s Gardasil HPV vaccine won approval from the National Medical Products Administration of China for males aged 9 to 26 years old, making the first option of its kind available for the country's male population.The vaccine was previously approved for use in females who are 9 to 45 years of age and has helped protect over 50 million women in China from certain HPV-related cancers and diseases since its first approval, Merck’s president of Human Health International, Joseph Romanelli, said in a release.Gardasil has recently seen slumping sales in China, where it's being marketed by Merck’s regional commercial partner Zhifei Biological Products. Rob David previously called the expansion to the male population a “significant opportunity.” UPDATED: Tuesday, Jan. 7 at 3:57 p.m. ETThe FDA tacked on additional warning information to the prescribing information for Agios’ pyruvate kinase (PK) deficiency drug Pyrukynd, flagging instances of liver deficiency in certain situations.The label update follows liver injury observations in patients who received Pyrukynd at a dose higher than is recommended for PK deficiency, according to an SEC filing from the company.The drug was approved by the FDA in 2022 as the first medicine to treat hemolytic anemia in adults with PK deficiency. Besides this use, the company is exploring expansions into beta thalassemia and sickle cell disease.The updated "Warnings and Precautions" section urges prescribers to run liver tests on patients before initiating treatment and monthly for the first six months of treatment. Patients should stop treatment if hepatic injury is suspected.Bayer is vying for a third indication for its Nubeqa (darolutamide) in China, this time for its use in combination with androgen deprivation therapy (ADT) in patients with metastatic hormone-sensitive prostate cancer.The company filed an application for the label expansion with China’s National Medical Products Administration, leveraging data from its phase 3 ARANOTE trial. In the study, Nubeqa and ADT significantly reduced the risk of radiological progression or death by 46% compared with placebo plus ADT.The nod would add to the drug’s two existing approvals in China, which cover other prostate cancer patient populations. It is estimated that the prevalence of prostate cancer in China will exceed 161,000 cases annually by 2026, according to Bayer. Dizal’s non-small cell lung cancer (NSCLC) candidate sunvozertinib will get a speedy FDA review after the agency granted priority review to the company’s new drug application.The company is specifically going after an approval to treat locally advanced or metastatic non-small cell lung cancer (NSCLC) in patients with epidermal growth factor receptor (EGFR) exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy. Sunvozertinib won its world-first approval in China in 2023. UPDATED: Monday, Jan. 6 at 10:25 a.m. ETAmgen's DLL3-targeted bispecific antibody Imdylltra, known as Imdelltra in the U.S., has won a conditional marketing authorization from the U.K.'s Medicines and Healthcare products Regulatory Agency as a third-line therapy for small cell lung cancer.The conditional nod is based on results from the phase 2 DeLLphi-301 trial, which showed that the T-cell engager triggered a tumor response rate of 41%, with the median response lasting 9.7 months. It follows an FDA approval in May 2024.China has approved Astellas' first-in-class Claudin 18.2 drug Vyloy, following similar moves by drug regulators in Japan and the U.S.The drug's Chinese indication is in first-line, CLDN18.2-positive, HER2-negative advanced gastric or gastroesophageal junction adenocarcinoma. The CLDN18.2 biomarker is expressed by about 35% of Chinese stomach cancer patients, according to Astellas.The approval was backed by two global phase 3 trials, dubbed Glow and Spotlight, which evaluated Vyloy in combination with the chemotherapy regimens CAPOX and mFOLFOX6, respectively. Both trials linked the drug's chemo combo to longer life expectancy and progression-free survival time compared with chemo alone.UPDATED: Friday, Jan. 3 at 2:45 p.m. ETGSK’s IL-5 inhibitor Nucala scored a nod in China to treat people with inadequately controlled chronic rhinosinusitis with nasal polyps (CRSwNP), a condition that impacts nearly 30 million people in China.The drug was approved by China’s National Medical Products Administration for use as an add-on therapy along with intranasal corticosteroids for patients with CRSwNP who don’t achieve adequate disease control with systemic corticosteroids or surgery. The indication is Nucala’s third in China and comes after prior approvals in eosinophilic asthma and eosinophilic granulomatosis with polyangiitis.An FDA advisory committee panel is scheduled to discuss the merits of Novartis’ oral factor B inhibitor Fabhalta in the rare kidney disease C3 glomerulopathy (C3G).The Cardiovascular and Renal Drugs Advisory Committee will meet on February 24, according to an FDA document (PDF). The drug was approved by the agency in 2023 as a treatment for paroxysmal nocturnal hemoglobinuria, winning the title of the first oral monotherapy approved for the rare blood disorder. Fabhalta received a second nod for immunoglobulin A nephropathy (IgAN) in August.In studies, Fabhalta proved that it could reduce proteinuria for C3G patients, a marker for delaying progression to kidney failure. So far, there are no approved treatments for the ultra-rare disease, which causes about 50% of patients to progress to kidney failure within ten years of diagnosis. UPDATED: Thursday, Jan. 2 at 1:45 p.m. ETShortly before the new year, Merck & Co. scored an approval for its potential blockbuster Winrevair in the U.K. The pulmonary arterial hypertension (PAH) drug, which also goes by the name sotatercept, was originally picked up via Merck’s $11.5 billion acquisition of Acceleron back in 2021.The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) approved Winrevair in combination with other PAH therapies to improve exercise capacity in adult patients who live with moderate or marked limitations of physical activity.Patients with PAH suffer from high blood pressure in the blood vessels that supply the lungs, which can ultimately damage the right side of the heart. Winrevair, for its part, targets the causes of PAH responsible for the narrowing of the arteries in the lungs, in turn making it easier for the heart to pump blood to the pair of organs, according to the U.K. government’s release.The MHRA based its approval on a study of 323 PAH patients that saw Winrevair best placebo at improving exercise ability. The main measure of effectiveness in the trial was the difference in distance patients could walk in six minutes before and after treatment.At the trial’s 24-week treatment mark, patients on Winrevair plus other PAH medicines improved their walking distance by around 34 meters, versus one meter in patients who received placebo.Merck’s U.K. nod for Winrevair follows green lights from the U.S. FDA last March and the European Commission in August.At the time of the drug’s U.S. approval last year, J.P. Morgan’s Chris Schott estimated Winrevair could eventually reel in peak sales between $3 billion and $4 billion. 

MSD has invested $12bn in US capital since 2018. Credit: Katherine Welles/Shutterstock. MSD has opened a $1bn facility in Durham, in the US state of North Carolina, to increase vaccine manufacturing capacity. The development is part of a broader strategy, with MSD having invested $12bn in US capital since 2018, aimed at widening domestic manufacturing and research and development offerings to generate new employment opportunities in the country. An additional investment of $8bn is anticipated by 2028. The 225,000ft² facility is set to incorporate best practices and insights from across the company’s manufacturing division network. MSD manufacturing division president and executive vice-president Sanat Chattopadhyay stated: “Expanding our state-of-the-art manufacturing facility in Durham marks a significant milestone in our efforts to strengthen our production and manufacturing capabilities in the US. “The cutting-edge technologies employed here empower our workforce and underscore our leadership in innovation to support patients everywhere.” The facility also incorporates technical and digital capabilities, which include generative AI, data analytics and three-dimensional (3D) printing, alongside a training centre featuring a digital twin. The digital twin is a virtual model of the shop floor manufacturing process system to expedite training for new employees and to test process changes before implementation. MSD Durham manufacturing division site in Durham plant manager and vice-president Amanda Taylor stated: “This level of investment and commitment speaks so powerfully to the work we do here in Durham. To see the pride and the energy of the people who work here and are helping drive this evolution in our capabilities is just phenomenal.” In February 2025, the company received conditional approval from the European Commission for the oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, Welireg, for adults with von Hippel-Lindau disease who have advanced clear cell renal cell carcinoma.