Remibrutinib

Novartis has pegged multibillion-dollar peak sales for remibrutinib based on its potential to expand into multiple immunology indications. Novartis has scored the FDA’s approval for a second BTK inhibitor that’s for a non-cancer indication, teeing off a potential competition with Sanofi down the line.The FDA has blessed Novartis’ remibrutinib, to be marketed under the brand name Rhapsido, as an oral drug for adult patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite H1 antihistamine treatment.CSU, also known as chronic hives, is a familiar area for Novartis, which has been selling the injectable Xolair together with Roche for the immune disorder in the U.S. since 2014. In April, the FDA also cleared Sanofi and Regeneron’s blockbuster antibody Dupixent to treat CSU. Rhapsido comes with a novel mechanism of action. As BTK plays an important role in mast cell degranulation and hence the release of histamine that causes hives, as well as in B cell-mediated autoantibody production, inhibiting BTK signaling could tackle both the allergic and autoimmune types of CSU, Victor Bulto, Novartis’ U.S. president, explained in a recent interview with Fierce Pharma.Despite its availability for over a decade, adoption of Xolair in CSU has been limited mostly to patients who have exhausted all other options, Bulto observed. That’s partly because dermatologists are generally not used to administering injectables, and Xolair carries a black box warning. For Rhapsido, Novartis is angling the oral med right after antihistamines have stopped working, Bulto said. And the trigger for the search for another treatment option, according to Bulto, is typically when patients can’t sleep right.By that standard, Novartis estimates the target CSU population in the U.S. is about 400,000, out of about 1.7 million people who live with the disease in the country, Bulto said. Of those target patients, only about 20% are being treated with either Xolair or Dupixent today, leaving a big space for Rhapsido.Rhapsido proved its worth in CSU through the phase 3 Remix-1 and Remix-2 trials. Among patients with symptomatic CSU despite second-generation antihistamines, Rhapsido showed superiority over placebo in change from baseline in itch, hives and weekly urticaria activity at Week 12. Longer-term data presented last year showed that almost half of patients were completely free of itch and hives at the one-year point. Compared with placebo, significantly more patients treated with Rhapsido achieved well-controlled disease as early as Week 2 and at Week 12.For a serious disease that can cause debilitating symptoms and unpredictable flares, Rhapsido represents “an exciting new option that has the potential to help a broad range of patients get fast relief,” Mark Lebwohl, M.D., from the Icahn School of Medicine at Mount Sinai and a member of the steering committee for the Remix clinical trial program, said in a Sept. 30 release.Besides its efficacy in controlling hives and itching that plague CSU patients, Rhapsido “gives patients an oral option that can easily be incorporated into their daily lives,” Giselle Mosnaim, M.D., from the University of Chicago and an investigator of the Remix program, said in a statement.Rhapsido has demonstrated a safety profile that does not require any lab monitoring, according to Novartis. This feature will give dermatologists confidence to treat resistant patients with the drug rather than referring them to allergists, Bulto noted.Rhapsido’s approval follows an FDA go-ahead a month ago for Sanofi’s BTK inhibitor Wayrilz in immune thrombocytopenia (ITP). The French pharma has already moved the drug into a phase 3 study in chronic hives following a positive midstage trial readout.One notable difference between the two BTK inhibitors is that Wayrilz’s binding to BTK is reversible with both noncovalent and covalent binding regions, whereas Rhapsido works by forming irreversible covalent bond with BTK. In cancer treatment, reversible noncovalent BTK inhibitors are generally considered safer than their covalent counterparts.Novartis has not seen any alarming safety signal with Rhapsido across trials in multiple indications, Bulto said. Both companies are eager to expand their options into other immunology indications, which underpin Novartis’ multibillion-dollar peak sales estimate for Rhapsido.The Novartis drug recently showed promise in food allergy in a phase 2 trial, as the Swiss pharma spotlights its potential to become the first oral allergen-agnostic treatment. If successful, food allergy would be a huge opportunity with a patient population of 20 million in the U.S., according to Bulto.Besides CSU, Novartis expects pivotal readouts for Rhapsido in chronic inducible urticaria and multiple sclerosis in 2026. The drug is also being pushed into phase 3 studies for hidradenitis suppurativa and generalized myasthenia gravis.For Sanofi, the French drugmaker is also testing Wayrilz in asthma, focal segmental glomerulosclerosis, warm autoimmune hemolytic anemia and IgG4-related disease.“We want to go to areas where we have the confidence that our scientists, our drug developers, our commercial teams know well,” and Novartis can use that know-how to understand whether its compound can win in the target areas, Bulto said.Novartis is beefing up its immunology portfolio with the likes of Rhapsido as its Cosentyx, which generated nearly $3.2 billion sales in the first half of 2025, is slated to lose patent protection toward the end of the decade.

Rhapsido helps to inhibit release of histamine and proinflammatory mediators by targeting BTK, offering unique approach to CSU treatment 1 Well-controlled disease observed as fast as two weeks, with demonstrated safety profile that requires no lab monitoring 1 1.7 million people in US live with CSU; more than half remain symptomatic despite increasing doses of antihistamines 2,3 Remibrutinib also in clinical development for chronic inducible urticaria, food allergy, and hidradenitis suppurativa, expanding Novartis Immunology portfolio EAST HANOVER, N.J., Sept. 30, 2025 /PRNewswire/ -- Novartis announced today that Rhapsido® (remibrutinib) received US Food and Drug Administration (FDA) approval as an oral treatment for adult patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite H1 antihistamine treatment. Rhapsido is a pill taken twice daily and does not require injections or lab monitoring. It is the first FDA-approved Bruton's tyrosine kinase inhibitor (BTKi) for CSU. Rhapsido helps to inhibit the release of histamine and other proinflammatory mediators by targeting BTK, offering a unique approach to CSU treatment.1 "CSU is a serious disease that can cause debilitating symptoms and unpredictable flares. It's difficult to diagnose and manage," said Mark Lebwohl, MD, Dean for Clinical Therapeutics at the Icahn School of Medicine at Mount Sinai and member of the steering committee for the remibrutinib REMIX Phase III clinical trial program. "Remibrutinib represents a new way of treating CSU. By blocking the activity of BTK, remibrutinib stops a key pathway of the immune response in CSU. This is an exciting new option that has the potential to help a broad range of patients get fast relief." CSU is a mast cell-driven condition thought to be caused by immune dysregulation. In people with CSU, the immune system can become activated through allergic (IgE) or autoimmune (IgG) pathways.4 This causes certain immune cells—mast cells and basophils—to activate the BTK protein. While not fully understood, it is believed that once activated, BTK leads to the release of histamine and other proinflammatory mediators that may cause the red, swollen, and itchy hives commonly seen in CSU.5,6 CSU symptoms are unpredictable, recurring for six weeks or more without an identified cause.7 Diagnosis can take up to 24 months.8 Many CSU patients say their symptoms negatively impact their sleep, work, and mental health.9,10,11 Antihistamines are the first-line treatment, but over half of patients still have symptoms, even at higher doses.2 Injectable treatments exist for those who don't respond to antihistamines, yet fewer than 20% of eligible patients receive them.12 "The approval of remibrutinib is an important development in CSU care. It quickly reduces symptoms, offering patients control of the hives and itching that they experience on a daily basis," said Giselle Mosnaim, MD, MS, an Allergist and Immunologist from Endeavor Health, Clinical Associate Professor at the University of Chicago Pritzker School of Medicine and REMIX trial investigator. "This is significant because it expands beyond existing injectable treatments and gives patients an oral option that can easily be incorporated into their daily lives." "Many CSU patients feel misunderstood and settle for treatments that don't fully meet their needs," said Lynda Mitchell, CEO of Allergy & Asthma Network. "We support new treatment options that empower patients to choose what works best for them. This convenient new oral therapy offers a promising new way to manage CSU and potentially improve daily life for those living with this challenging condition." Clinical data supporting approval The FDA approval of Rhapsido in CSU is based on results from the Phase III REMIX-1 (NCT05030311) and REMIX-2 (NCT05032157) clinical trials in patients who remained symptomatic on second-generation H1 antihistamines. Rhapsido demonstrated superiority in change from baseline versus placebo in itch (ISS7), hives (HSS7), and weekly urticaria activity (UAS7) at Week 12.13 Significantly more patients treated with Rhapsido versus placebo achieved well-controlled disease (UAS7≤6) as early as Week 2 and at Week 12, and about one-third of patients achieved complete absence of itch and hives at Week 12.13 Rhapsido has a demonstrated safety profile that requires no lab monitoring.13 The most common adverse events (incidence ≥3%) were nasal congestion, sore throat, and runny nose (nasopharyngitis), bleeding, headache, nausea, and abdominal pain.13 Novartis has completed regulatory submissions for Rhapsido for the treatment of CSU across many countries, including in the European Union, Japan, and China, with priority review granted in China. Transforming care in Immunology "This approval of Rhapsido as the first and only BTK inhibitor in CSU is an important milestone in our journey to reshape care for overlooked immune-related conditions and offer more patients the potential to find fast relief," said Victor Bultó, President, US, Novartis. "Building on our legacy in advancing the treatment of allergic, dermatologic, and rheumatologic conditions, we are deeply committed to further investing in innovative, patient-focused therapies across immunology." Discovered and developed by Novartis to target the BTK pathway as a driver of inflammation, remibrutinib is being investigated in ongoing clinical trials across a variety of immune-related conditions, including chronic inducible urticaria (CIndU), hidradenitis suppurativa (HS), and food allergy. RHAPSIDO® (remibrutinib) Indication RHAPSIDO is a prescription medicine used to treat adults with chronic spontaneous urticaria (CSU) who continue to have symptoms that are not controlled with antihistamine treatment. RHAPSIDO should not be used to treat any other forms of hives (urticaria). It is not known if RHAPSIDO is safe and effective in children. RHAPSIDO Important Safety Information Before taking RHAPSIDO, patients should tell their health care provider about all of their medical conditions, including if they: Have had a recent surgery or plan to have surgery. Health care providers may tell patients to stop taking RHAPSIDO for at least 3 to 7 days before and after any planned medical or surgical procedures Have bleeding problems or are taking a blood thinner medicine Have liver problems Are pregnant or plan to become pregnant. It is not known if RHAPSIDO will harm an unborn baby. If patients become pregnant during treatment with RHAPSIDO, they should talk to their health care provider about registering in the pregnancy exposure registry for RHAPSIDO Are breastfeeding or plan to breastfeed. It is not known if RHAPSIDO passes into breast milk Have recently received or are scheduled to receive an immunization (vaccine). Patients should talk to their health care provider about receiving any immunizations before taking RHAPSIDO. They should not receive live or live-attenuated vaccines during treatment with RHAPSIDO Patients should tell their health care provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking RHAPSIDO with certain other medications may affect how RHAPSIDO or other medications work, and can cause side effects. RHAPSIDO may cause serious side effects, including the risk of bleeding. Bleeding may happen while being treated with RHAPSIDO. Patients' health care providers will monitor them for signs and symptoms of bleeding and may stop their treatment if bleeding happens. Patients' risk of bleeding may increase if they are also taking a blood thinner medicine. Patients should tell their health care provider right away if they get any signs or symptoms of bleeding, such as: bruising or red or purple skin marks; pink or brown urine; headache, dizziness, confusion, or feeling weak; red or black stools that look like tar. The most common side effects with RHAPSIDO may include nasal congestion, sore throat, and runny nose (nasopharyngitis), bleeding, headache, nausea, and abdominal pain. Please see full Prescribing Information, including Patient Information. Disclaimer This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "may," "could," "would," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise. About Novartis Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide. Reimagine medicine with us: Visit us at and connect with us on LinkedIn US, X/Twitter US and Instagram. References Rhapsido® (remibrutinib) Prescribing Information. Novartis Pharmaceuticals Corp. Maurer M, Weller K, Bindslev-Jensen C, et al. Unmet clinical needs in chronic spontaneous urticaria. A GA²LEN task force report. Allergy. 2011;66:317-330. doi:10.1111/j.1398-9995.2010.02496.x The World Bank. Population, total. Accessed June 2025. Kolkhir P, Muñoz M, Asero R, et al. Autoimmune chronic spontaneous urticaria. J Allergy Clin Immunol. 2022;149(6):1819-1831. doi:10.1016/j.jaci.2022.04.010 Kolkhir, P., Giménez-Arnau, A.M., Kulthanan, K. et al. Urticaria. Nat Rev Dis Primers. 8, 61 (2022). Mendes-Bastos P, Brasileiro A, Kolkhir P, et al. Bruton's tyrosine kinase inhibition-An emerging therapeutic strategy in immune-mediated dermatological conditions. Allergy. 2022;77(8):2355-2366. Maurer M, Costa C, Gimenez Arnau A, et al. Antihistamine-resistant chronic spontaneous urticaria remains undertreated: 2-year data from the AWARE study. Clin Exp Allergy. 2020;50:1166-1175. doi: 10.1111/cea.13716 Friedman A, Kwatra SG, Yosipovitch G. A Practical Approach to Diagnosing and Managing Chronic Spontaneous Urticaria. Dermatol Ther (Heidelb). 2024;14(6):1371-1387. doi: 10.1007/s13555-024-01173-5 Mendelson M, Bernstein J, Gabriel S, et al. Patient-reported impact of chronic urticaria compared with psoriasis in the United States. J Dermatolog Treat. 2017; 28(3):229-236. doi:10.1080/09546634.2016.1227421 Maurer M, Abuzakouk M, Bérard F, et al. The burden of chronic spontaneous urticaria is substantial: Real-world evidence from ASSURE-CSU. Allergy. 2017;72(12):2005-2016. doi:10.1111/all.13209 Balp M-M, Krupsky K, Balkaran BL, et al. Oral presentation at: European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2023; June 9–11, 2023; Hamburg, Germany. Maurer M, Raap U, Staubach P, et al. Antihistamine-resistant chronic spontaneous urticaria: 1-year data from the AWARE study. Clin Exp Allergy.2019; 49: 655–662. Metz M, Giménez-Arnau A, Hide M, et al. Long-term efficacy and safety of remibrutinib in patients with chronic spontaneous urticaria in the Phase 3 REMIX-1 and REMIX-2 studies. Presented as a late oral abstract session on clinical trials at EAACI 2024; May 31-June 3, 2024; Valencia, Spain. # # # Novartis Media Relations E-mail: [email protected] Novartis Investor Relations Central investor relations line: +41 61 324 7944 E-mail: [email protected] SOURCE Novartis Pharmaceuticals Corporation WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED