RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Priority Review (China), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (Australia), Accelerated assessment (European Union), Special Review Project (China)

Structure/Sequence

Molecular FormulaC22H23N7O

InChIKeyASKZRYGFUPSJPN-UHFFFAOYSA-N

CAS Registry1825352-65-5

Clinical Trials associated with Risdiplam

NL-OMON61218

/ SuspendedPhase 4IIT

Open label, phase 4, single center, non-interventional, prospective study evaluating the clinical effectiveness of risdiplam in Dutch patients with genetically confirmed 5q SMA > 25 years of age at the start of treatment. - Effectiveness of risdiplam in SMA patients > 25

Start Date03 Jun 2026

Sponsor / Collaborator-

NCT07047144

/ RecruitingPhase 2

A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)

This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score <55.

Start Date15 Sep 2025

Sponsor / Collaborator

Scholar Rock, Inc.

JPRN-jRCTs031240475

/ RecruitingNot Applicable

A specific clinical study on the efficacy and safety of combined therapy with Risdiplam treatment and HAL-based robotic rehabilitation in patients with spinal muscular atrophy (HARMONY-SMA)

Start Date08 Nov 2024

Sponsor / Collaborator-

100 Clinical Results associated with Risdiplam

100 Translational Medicine associated with Risdiplam

100 Patents (Medical) associated with Risdiplam

301

Literatures (Medical) associated with Risdiplam

01 Mar 2027·Value in Health Regional Issues

Cost-Effectiveness of Universal Newborn Screening and Disease-Modifying Therapies for Spinal Muscular Atrophy in Taiwan

Article

Author: Hsieh, Hui-Min ; Chen, Huey-Fen ; Prosser, Lisa A ; Jong, Yuh-Jyh ; Chiang, Wan-Ling ; Chen, Chung-Yu

OBJECTIVES:

Spinal muscular atrophy (SMA) is a rare neuromuscular disorder and an important genetic cause of infant mortality. Universal newborn screening (NBS) enables presymptomatic disease-modifying therapy (DMT) but increases upfront costs. This study evaluated the cost-effectiveness of universal SMA NBS in Taiwan.

METHODS:

This study built a state-transition simulation model for a hypothetical SMA birth cohort using local epidemiology and costs and published clinical inputs. Strategies were as follows: (1) NBS with presymptomatic DMTs and (2) clinical identification (CI) with symptomatic DMTs. DMTs included nusinersen, onasemnogene abeparvovec, and risdiplam (reimbursed by Taiwan's National Health Insurance). Incremental costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) were estimated over 10-year and lifetime (90-year) horizons from Taiwan's payer and societal perspectives. The willingness-to-pay (WTP) threshold was 3 times Taiwan's 2022 GDP per capita (New Taiwan Dollar (NTD) 2 917 650/QALY). One-way, probabilistic sensitivity, and scenario analyses assessed uncertainty.

RESULTS:

All ICERs were below WTP threshold. From the payer perspective, ICERs for NBS with presymptomatic DMT versus CI with symptomatic DMT were NTD 1 157 970 (USD 38 599) /QALY (10 years) and NTD 2 108 499 (USD 70 283)/QALY (lifetime). From the societal perspective, ICERs were NTD 261 789 (USD 8726)/QALY (10 years) and NTD 1 494 002 (USD 49 800)/QALY (lifetime). Results were most sensitive to DMT costs (particularly nusinersen and risdiplam) and health-state utility values.

CONCLUSIONS:

Universal SMA NBS with presymptomatic DMT is likely cost-effective in Taiwan from both payer and societal perspectives under the WTP threshold, supporting earlier treatment to slow progression and improve quality of life.

01 Dec 2026·SLEEP MEDICINE

Respiration during sleep in children with spinal muscular atrophy type 2 and 3, treated with nusinersen or risdiplam

Article

Author: Hansen, Christine Rønne ; Dereksson, Kristján ; Eklund, Erik A ; Kötz, Karsten ; Lund, Sigrún Helga ; Markström, Agneta ; Lindqvist, Johan Fredén ; Alberg, Lars

Spinal muscular atrophy (SMA) is associated with sleep-disordered breathing (SDB) and sleep-hypoventilation in children. Weakened intercostal muscles and preserved diaphragm strength cause thoraco-abdominal asynchrony (TAA) during sleep in this group. Our aim was to study if disease-modifying treatment (DMT) could stabilise or improve sleep-related respiration in children with SMA type 2 or 3 over a four-year period. We followed 34 children and adolescents with SMA type 2 or 3 who initiated either of the DMTs nusinersen or risdiplam between 2017 and 2023. Respiratory polygraphy was performed at treatment start and repeatedly for 4 years. Respiratory outcomes were measured, including TAA prevalence, expressed as the percentage of total sleep time (TST) with a phase angle >40° between thorax and abdomen. Median follow-up was 3.6 years. Use of non-invasive ventilation was unchanged throughout the study. In SMA type 2, AHI decreased by 5.1 [95%CI -7.6 to -2.6] and the obstructive-AHI and oxygen desaturation index showed a similar decrease, while minimal-SpO₂ increased by 4.2% [0.2 to 8.2]. In SMA type 3 no change was seen in the above factors, but TAA prevalence decreased by 14.1% [-25.3 to -2.9] from the baseline value of 34.5% of TST. The proportion of subjects with abnormal TAA, defined as >10% TST, fell from 86.7% to 40.0%. Over the study period, DMTs were associated with stabilization or improvement of sleep-related respiration in both SMA type 2 and 3. This included reduced SDB in type 2 and improved TAA in type 3, with no evidence of respiratory decline.

01 Nov 2026·SLEEP MEDICINE

Sleep disordered breathing and autonomic outcomes in adult patients with spinal muscular atrophy (SMA) receiving Risdiplam

Article

Author: Alonge, Paolo ; Tomasello, Vito ; Marrone, Oreste ; Di Stefano, Vincenzo ; Crescimanno, Grazia ; Brighina, Filippo ; Lupica, Antonino

OBJECTIVES:

Investigations on effects of risdiplam therapy in adult patients with Spinal Muscular Atrophy (SMA) have been limited to diurnal respiratory and motor function. We explored whether risdiplam can influence nocturnal respiratory events and autonomic function.

METHODS:

Sixteen consecutive subjects ≥16 years old with SMA type 2 or 3 were studied before and 12 months after risdiplam therapy. They underwent nocturnal cardiorespiratory polygraphy for analysis of respiratory function and heart rate variability (HRV). In addition, their motor function was evaluated using the Revised Upper Limb Module (RULM) and the Hammersmith Functional Motor Scale Expanded (HFMSE) scales.

RESULTS:

After therapy, apnoea/hypopnoea Index (AHI) and oxygen desaturation index decreased (p = 0.02), while time with oxygen saturation <90% and lowest oxygen saturation did not change. HRV analysis showed a decrease in the SD2/SD1 ratio (p = 0.03), suggesting a decrease in sympathovagal balance. RULM and HFMSE scores showed a borderline improvement. The SD2/SD1 change was correlated with the change in AHI but not with the change in respiratory function or motor scale scores.

CONCLUSIONS:

In adult patients with SMA, risdiplam therapy may be associated with improvements in nocturnal respiratory function and autonomic activity. The reduction in respiratory events during sleep may contribute to improved autonomic balance.

276

News (Medical) associated with Risdiplam

14 Sep 2026

·allsci.com

US FDA approves Scholar Rock’s Isembyld as first muscle-targeted SMA therapy

The US FDA has approved Isembyld (apitegromab-mstn) for spinal muscular atrophy (SMA) in adults and children aged two years and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. The approval, granted to Cambridge, Massachusetts-based Scholar Rock (Nasdaq: SRRK), introduces a muscle-directed mechanism to a treatment landscape that has until now focused on motor neuron preservation. Isembyld is administered as an intravenous infusion at 10 mg/kg once every four weeks. It is a fully human monoclonal IgG4 antibody that binds to promyostatin and latent myostatin, blocking myostatin activation and thereby inhibiting the signaling pathway that suppresses muscle growth. Unlike SMN2-targeted therapies such as nusinersen (Spinraza) and risdiplam (Evrysdi), Isembyld acts directly on muscle and is intended to be used alongside existing disease-modifying treatment. The approval was based on the Phase III SAPPHIRE trial (NCT05156320), which enrolled 188 patients aged 2–21 years with 5q SMA receiving background SMN2-targeted therapy. In the primary efficacy population of patients aged 2–12 years (n=103), Isembyld produced a 2.2-point treatment difference versus placebo on the Hammersmith Functional Motor Scale-Expanded at one year (nominal p=0.0121). The US label is broader than the pivotal efficacy population, covering adults and children aged two years and older who are receiving an SMN2-targeted therapy. Scholar Rock was also awarded a Rare Pediatric Disease Priority Review Voucher with the approval. The SMA treatment landscape has expanded considerably since the first SMN-targeted approvals, with Biogen's high-dose Spinraza regimen receiving FDA clearance and Novartis's gene therapy Itvisma (onasemnogene abeparvovec) now approved in older patients in Europe. Roche discontinued its myostatin inhibitor emugrobart in SMA after lackluster findings, leaving Scholar Rock as the only company to reach approval with a muscle-targeted agent in this setting. Scholar Rock said it has previously navigated manufacturing complications during the regulatory process, which involved removing the Catalent Indiana fill-finish site from the US BLA under FDA guidance and proceeding with an alternate facility. In Europe, Scholar Rock withdrew the MAA and plans to resubmit with the alternate facility. Spot something wrong? Report an issue with this article

Phase 3Drug ApprovalClinical ResultPriority ReviewGene Therapy

13 Sep 2026

·www.biospace.com

Scholar Rock Announces FDA Approval of ISEMBYLD™ (apitegromab-mstn), the First and Only Muscle-Targeted Treatment for Children and Adults with Spinal Muscular Atrophy (SMA)

ISEMBYLD is approved for use in all adults and children ≥2 years of age with SMA who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment ISEMBYLD recommended dose of 10 mg/kg showed a robust, clinically meaningful 2.2-point improvement in motor function as measured by the gold-standard Hammersmith Functional Motor Scale-Expanded (HFMSE) compared to placebo, with all patients receiving SMN2-targeted background therapy (p = 0.0121*) ISEMBYLD showed a ≥ 3-point increase in HFMSE in 34.2% of patients compared to 13.5% of patients on placebo (odds ratio 3.8; p = 0.0125*) ISEMBYLD U.S. commercial launch underway with product available to ship in the coming days; Scholar Rock Supports™ dedicated support team now available to assist patients and caregivers Management to host investor call Monday, September 14, 2026, at 8:00 a.m. ET * nominal p-value CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of patients with rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology, today announced that the FDA has approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in adults and children two years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. SMA is a rare, severe neuromuscular disease that results in irreversible loss of motor neurons and progressive muscle wasting, causing continuous motor function decline throughout life and diminishing the independence of both adults and children. ISEMBYLD is the first and only muscle-targeted treatment to demonstrate motor function improvement in individuals with SMA currently receiving an SMN2-targeted treatment. In the Phase 3 randomized, placebo-controlled SAPPHIRE study, individuals receiving ISEMBYLD demonstrated a robust, clinically meaningful improvement in motor function after one year of treatment while those on an SMN2-targeted treatment alone experienced a loss of motor function. “Today’s FDA approval of ISEMBYLD marks a defining moment for the SMA community as we now launch the world’s first-ever muscle targeted treatment for children and adults living with SMA in the U.S.,” said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. “After decades of failed industry-wide efforts to unlock the potential of myostatin inhibition, Scholar Rock has delivered a therapeutic breakthrough with ISEMBYLD. Our U.S. commercial team is now engaging physicians, SMA care teams, and payers on behalf of the SMA community and our Scholar Rock Supports™ team stands ready to provide dedicated, comprehensive assistance to patients and caregivers. I would like to extend my deepest thanks to our clinical study investigators, Cure SMA, and other patient advocacy groups for their dedication and support on this journey. Above all, I want to express my heartfelt gratitude to the patients and families affected by SMA who participated in our clinical trials for their trust in Scholar Rock and unwavering resilience every step of the way.” “The approval of ISEMBYLD as the first-ever treatment to directly target the muscular component of SMA is a significant turning point for adults and children who have been waiting for innovative therapeutic options to improve motor function,” said Kenneth Hobby, President of Cure SMA. “We appreciate the FDA’s recognition, as reflected in this approval that supports access for a broad population within the SMA community, that improving motor function is a significant unmet need that must be addressed with urgency. Such improvements are fundamental to maintaining independence and to enabling participation in important activities of daily living from self-care to work and social interactions.” With the approval of ISEMBYLD, Scholar Rock was awarded a Rare Pediatric Disease Priority Review Voucher, which may be used to obtain priority review for a future marketing application. ISEMBYLD Robust Clinical Efficacy and Safety Pro Phase 3 SAPPHIRE Study The approval of ISEMBYLD was based on positive results from the Phase 3 pivotal, randomized, double-blind, placebo-controlled SAPPHIRE study. The SAPPHIRE study met its primary endpoint, and demonstrated a robust, clinically meaningful 2.2-point improvement in the gold-standard Hammersmith Functional Motor Scale-Expanded (HFMSE) in patients receiving ISEMBYLD 10 mg/kg and an SMN2-targeted treatment compared to patients receiving an SMN2-targeted treatment alone at one year (nominal p = 0.0121; main efficacy population 2 - 12 years of age, n = 103). Additionally, 34.2% of ISEMBYLD-treated patients showed a ≥ 3-point increase in HFMSE compared to 13.5% of placebo-treated patients (odds ratio of 3.8; nominal p = 0.0125). ISEMBYLD has a well-characterized safety profile. The safety database includes more than 500 individuals across all apitegromab clinical studies globally, some of whom have been on treatment for more than 7 years. Ninety-eight percent of participants treated in SAPPHIRE elected to continue in the ONYX long-term extension study. In the SAPPHIRE study, the most common adverse reactions were upper respiratory tract infections, vomiting, cough, other viral infections, headache, gastroenteritis, pharyngitis, and hypersensitivity. Fractures occurred in 9% of patients treated with ISEMBYLD 10 mg/kg vs. 2% in placebo. For further information, see Important Safety Information below. “Today’s approval of ISEMBYLD marks a new era for the treatment of SMA,” said Dr. Basil Darras, M.D., Associate Neurologist-in-Chief, Director of the Neuromuscular Center and Spinal Muscular Atrophy Program at Boston Children’s Hospital, and a principal investigator in the SAPPHIRE study. “As neurologists, families consistently tell us that their top priority is gaining motor function, and we are now able to directly target the muscle, not just the motor neuron, for people living with SMA.” ISEMBYLD Commercial Availability and Patient Access Scholar Rock Supports is now available to assist patients who have been prescribed ISEMBYLD. This program is designed to provide personalized support for patients and families, and includes help to understand insurance coverage, financial assistance programs for eligible patients, and disease and treatment education. Scholar Rock Supports will also help patients and families navigate their site of care options, as well as ongoing infusion logistics. Based on eligibility, infusions can be given at convenient locations including hospital, home, or infusion center. ISEMBYLD will be available to ship in the coming days. Scholar Rock is working closely with top commercial and government payers to establish reliable, broad access to ISEMBYLD for appropriate patients. Healthcare providers and patients can learn more about Scholar Rock’s patient support services by visiting or calling 833-777-5444 (833-SRRK-444). For more information about ISEMBYLD and U.S. Prescribing Information, visit . Conference Call Information Scholar Rock will host a conference call and webcast on Monday, September 14, 2026, at 8:00 a.m. ET. To access the live audio webcast, please go to “Events and Presentations” in the Investors section of the Scholar Rock website at . To participate via telephone, please register in advance here . Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call. A replay of the webcast will be available on the Company’s website for approximately 90 days. About ISEMBYLD ISEMBYLD is a fully human monoclonal IgG4 antibody that binds to promyostatin and latent myostatin and inhibits the activation of myostatin, blocking myostatin signaling. ISEMBYLD is approved in the United States for the treatment of spinal muscular atrophy (SMA) in adults and pediatric patients 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. For more information, visit . Indication and Important Safety Information What is ISEMBYLD (apitegromab-mstn)? ISEMBYLD is a prescription medicine used to treat spinal muscular atrophy (SMA) in adults and children 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. IMPORTANT SAFETY INFORMATION Before taking ISEMBYLD, tell your healthcare provider about all of your medical conditions, including if you: have a history of low bone density or bone fractures are pregnant or plan to become pregnant. If you are pregnant or are planning to become pregnant, ask your healthcare provider for advice before taking this medicine. It is not known if ISEMBYLD will harm your unborn baby. Tell your healthcare provider right away if you become pregnant during treatment with ISEMBYLD are breastfeeding or plan to breastfeed. It is not known if ISEMBYLD passes into breast milk. Talk to your healthcare provider about the best way to feed your baby while on treatment with ISEMBYLD Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Keep a list of them to show your healthcare provider, including your pharmacist, when you get a new medicine. What are the possible side effects of ISEMBYLD? Fractures. Treatment with ISEMBYLD may increase the risk of bone fractures, including serious fractures. Bone fractures may happen with or without a fall or other injury. Your healthcare provider may consider stopping treatment with ISEMBYLD if you experience a bone fracture during treatment. The most common side effects of ISEMBYLD include: upper respiratory tract infections vomiting cough viral infections headache stomach flu (gastroenteritis) sore throat (pharyngitis) hypersensitivity These are not all of the possible side effects of ISEMBYLD. Call your healthcare provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. Please see full Prescribing Information and Patient Information . About the SAPPHIRE Clinical Trial SAPPHIRE ( NCT05156320 ) was a global, multi-national, randomized, double-blind, placebo-controlled Phase 3 clinical trial that evaluated the safety and efficacy of ISEMBYLD (apitegromab-mstn) in a total of 188 patients in 9 countries with a diagnosis of 5q SMA who were 2 to 21 years of age. Patients were randomized in a 1:1:1 ratio to receive ISEMBYLD 20 mg/kg (2 times the recommended dosage), ISEMBYLD 10 mg/kg (the recommended dosage), or placebo, respectively, via intravenous infusion once every 4 weeks for approximately 1 year. All patients enrolled in this trial were receiving an approved SMN2-targeted treatment (either nusinersen or risdiplam). About SMA Spinal muscular atrophy (SMA) is a rare, severe, genetic neuromuscular disease. The disease is characterized by the irreversible loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk, and progressive muscle wasting that causes continuous motor function decline throughout life and can diminish the independence of both children and adults. Motor function decline in SMA patients is affected by motor neuron health and muscle responsiveness. SMN-targeted treatments are designed to prevent motor neuron loss but do not directly address muscle. It is estimated that approximately 35,000 SMA patients globally have been treated with an SMN-targeted treatment. About Scholar Rock Scholar Rock is delivering muscle-targeted breakthroughs to transform the treatment of spinal muscular atrophy (SMA) and other rare neuromuscular diseases where muscle atrophy remains a critical unmet need. Scholar Rock intends to commercialize ISEMBYLD (apitegromab-mstn) globally, beginning in the U.S. for individuals living with SMA who are 2 years of age and older and currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. As a global leader in myostatin biology, a field focused on proteins that regulate muscle mass, the biopharmaceutical company is named for the visual resemblance of a scholar rock to protein structures. Our commitment to unlock fundamentally different treatment approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. Scholar Rock works every day to create new possibilities for patients through its highly innovative anti-myostatin program, including opportunities in additional rare neuromuscular diseases. Learn more about our neuromuscular franchise at ScholarRock.com and follow @ScholarRock on X and on LinkedIn. Scholar Rock ® is a registered trademark and ISEMBYLD ™ is a trademark of Scholar Rock, Inc. Availability of Other Information About Scholar Rock Investors and others should note that we communicate with our investors and the public using our company website , including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly known as Twitter) and LinkedIn. The information that we post on our website or on X (formerly known as Twitter) or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to: Scholar Rock’s expectations regarding the U.S. commercial launch of ISEMBYLD, ISEMBYLD’s ability to support a broad patient population, the potential approval and launch of apitegromab in other geographies, ISEMBYLD’s ability to change patient lives, the anticipated benefits of ISEMBYLD for patients with SMA, and the Company’s business strategy, plans and prospects. The words “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “anticipate,” “plan,” “believe,” “intend,” “estimate,” “potential,” “continue,” “target,” “goal,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on the Company’s current expectations, intentions, and beliefs regarding future events, and are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation: the Company’s ability to successfully commercialize ISEMBYLD in the U.S.; the Company’s ability to obtain and maintain regulatory approval of apitegromab in other jurisdictions; risks related to market acceptance, competition, pricing, reimbursement and access; manufacturing and supply chain risks; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; and Scholar Rock’s dependence on third parties for development and manufacture of ISEMBYLD, as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law. Multimedia Assets: Contacts Scholar Rock: Investors Laura Ekas, Ph.D. ir@scholarrock.com 917-439-0374 Media Jeff Smith 682-401-8428 Molly MacLeod, Ph.D. 802-579-5995 media@scholarrock.com

Clinical ResultPhase 3Drug ApprovalPriority Review

03 Aug 2026

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2026 Financial Results

– Robust Q2 performance with total revenue of $361 million, including $239 million of product revenue – – Full-year 2026 expected total revenue increased to $1.18 to $1.28 billion, with full-year product revenue guidance raised to $850 to $950 million – – Sephience™ (sepiapterin) Q2 2026 revenue of $151 million, with continued broad uptake and geographical expansion – – Strong cash position of $2.2 billion as of June 30, 2026 – WARREN, N.J., July 30, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ended June 30, 2026. "Our teams delivered another strong quarter enabling us to again raise 2026 product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "We also made a number of advances in our R&D pipeline, including identification of our next small molecule splicing development candidate, PTC303, targeting somatic expansion disorders including Huntington's disease and myotonic dystrophy. In addition, we initiated the Phase 1 study of PTC612, our differentiated NLRP3 small molecule." Key Corporate Updates Q2 2026 total revenue of $361 million, including $239 million of product revenue Sephience global launch continues with strong momentum Q2 2026 revenue of $151 million, with the majority from the US and increasing contributions internationally 1,647 patients on commercial therapy worldwide as of June 30, 2026 Positive results from votoplam PIVOT-HD long-term extension study reported and enrollment in global Phase 3 INVEST-HD study ongoing PIVOT-HD 24-month results demonstrated dose-dependent disease slowing on cUHDRS scale in Stage 2 participants, reaching 52% at 10 mg dose level Plan to engage with FDA in 2H 2026 to discuss PIVOT-HD 24-month results being finalized INVEST-HD study being conducted and funded by partner Novartis with target enrollment of approximately 770 individuals with early symptomatic disease INVEST-HD initiation triggered a $50 million milestone payment from Novartis to PTC in Q2 2026 Vatiquinone PROVE-FA study to support NDA resubmission for treatment of Friedreich's ataxia expected to initiate in Q3 2026 Global study will enroll approximately 120 individuals with Friedreich's ataxia age 7 to 21 Study includes open-label treatment arm with matched natural history control group PTC pipeline continues to advance PTC303 named as MSH3 splicing program development candidate targeting Huntington's disease and myotonic dystrophy with plan to enter clinic in 2027 Phase 1 study of PTC612, an oral NLRP3 inhibitor, initiated in Q2 2026 Phase 2a study of PTC844, a next-generation DHODH inhibitor, expected to initiate in Q3 2026 Hege Sollie-Zetlmayer has been appointed to PTC's Board of Directors. Ms. Sollie-Zetlmayer has over 30 years of experience in the biopharmaceutical and medical device industry as a global business operations and human resources leader. She most recently served as PTC's Chief Human Resources Officer prior to her retirement in June 2026. Second Quarter 2026 Financial Highlights Total net product revenue and royalty revenue was $309.9 million for the second quarter of 2026, compared to $175.9 million for the second quarter of 2025. Total net product revenue across the commercial portfolio was $238.8 million for the second quarter of 2026, compared to $118.3 million for the second quarter of 2025, representing over 100% increase. Sephience net product revenues were $151.3 million for the second quarter of 2026, representing 21% growth compared to the first quarter of 2026. Translarna™ (ataluren) net product revenues were $42.2 million for the second quarter of 2026, compared to $59.5 million for the second quarter of 2025. Emflaza® (deflazacort) net product revenues were $24.6 million for the second quarter of 2026, compared to $36.4 million for the second quarter of 2025, due to continued generic erosion. Roche reported Evrysdi® (risdiplam) year-to-date sales of approximately 968 CHF million, resulting in royalty revenue of $71.1 million to PTC for the second quarter of 2026, compared to $57.6 million to PTC for the second quarter of 2025. In the second quarter of 2026, PTC recorded a development milestone of $50.0 million from Novartis for the first patient dosed in the ongoing INVEST-HD Phase 3 study. This sales milestone was recorded as collaboration revenue. Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $99.2 million for the second quarter of 2026, compared to $113.0 million for the second quarter of 2025. Non-GAAP R&D expenses were $88.6 million for the second quarter of 2026, excluding $10.5 million in non-cash, stock-based compensation expense, compared to $104.0 million for the second quarter of 2025, excluding $9.0 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $80.6 million for the second quarter of 2026, compared to $85.3 million for the second quarter of 2025. Non-GAAP SG&A expenses were $67.9 million for the second quarter of 2026, excluding $12.7 million in non-cash, stock-based compensation expense, compared to $75.7 million for the second quarter of 2025, excluding $9.5 million in non-cash, stock-based compensation expense. Net income was $83.5 million for the second quarter of 2026, compared to net loss of $64.8 million for the second quarter of 2025. In June 2026, PTC issued $550.0 million of senior convertible notes due in 2031 at 0% coupon and a conversion price representing a 40% premium over the stock's closing price at the time of the deal. Concurrent with the transaction, PTC repurchased the majority of its 1.5% senior convertible notes due in September 2026. Cash, cash equivalents, and marketable securities were $2,229.1 million on June 30, 2026, compared to $1,945.4 million on December 31, 2025. Shares issued and outstanding as of June 30, 2026, were 83,327,286. PTC Updates Full-Year 2026 Financial Guidance Expected total revenue increased to $1.18 to $1.28 billion, with total product revenue guidance raised to $850 to $950 million from $750 to $850 million GAAP R&D and SG&A expense guidance remains $775 to $815 million Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million Non-GAAP Financial Measures In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in accordance with GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms CHF: Confoederatio Helvetica Francs (Swiss francs) cUHDRS: Composite Unified Huntington's Disease Rating Scale DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: US Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease NDA: New Drug Application nmDMD: Nonsense mutation Duchenne muscular dystrophy R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder To access the live webcast, please visit the "Events & Presentations" page within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance here to receive dial-in details. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X, Facebook and Instagram. For more information please contact: Investors: Ellen Cavaleri +1 (615) 618-8228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "PTC Updates Full-Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and total revenue guidance and (ii) 2026 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, meetings with regulatory agencies, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," "aim," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including commercialization and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the US on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; expectations regarding PTC's product candidates, including the timing of clinical trials and studies; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 21% more press release views with Request a Demo

Phase 1Financial StatementExecutive ChangePhase 2Phase 3

100 Deals associated with Risdiplam

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KEGG	Wiki	ATC	Drug Bank
D11406	Risdiplam	M09A	DB15305

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Spinal Muscular Atrophy	United States	Genentech, Inc.	07 Aug 2020

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	United States	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	United States	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Japan	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Japan	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Belgium	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Belgium	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Brazil	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Brazil	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Croatia	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Croatia	F. Hoffmann-La Roche Ltd.	19 Dec 2016

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02913482 (FIREFISH, Pubmed \| Lancet Child Adolesc Health)	Phase 2	HMN (Hereditary Motor Neuropathy) Proximal Type I	58	Risdiplam	xzpmjpxrsg(ieprzrkhas) = Most adverse events were mild or moderate. The most frequently reported adverse events were pyrexia (65% [n=40]), upper respiratory tract infection (63% [n=39]), and pneumonia (50% [n=31]). lxrerqylwn (oyvsgtrjoe ) View more	Positive	01 Sep 2026
NCT02908685 (SUNFISH, AAN2026)	Phase 2	SMN protein	344	Risdiplam	gblvilsvxf(sfmphyzptj) = opmlkvgmxm phjseiafia (wunfyfpbol ) View more	Positive	25 Apr 2026
AAN 12026 \| AAN 22026 \| AAN 32026	Not Applicable	Spinal Muscular Atrophy	268	Risdiplam	nhntgioiih(fhfvnlbvqi) = qfnxmdeslx mhpzdvjmld (pnnjbtedqo ) View more	Positive	25 Apr 2026
NCT03779334 (RAINBOWFISH, MDA2026)	Phase 2	Spinal Muscular Atrophy	23	Risdiplam	vnzatyzzav(pvslnysshb) = mnqirlwcen ofeexsotxr (xacxiudwov ) View more	Positive	08 Mar 2026
MDA2026	Not Applicable	Spinal Muscular Atrophy	950	Risdiplam	zazpmpiuut(phzctompir) = encbofdwnl msbkycearq (pwhowhcfrj ) View more	Negative	08 Mar 2026
MDA2026	Not Applicable	Spinal Muscular Atrophy	950	Nusinersen	zazpmpiuut(phzctompir) = imdfuxooka msbkycearq (pwhowhcfrj ) View more	Negative	08 Mar 2026
NCT03032172 (Jewelfish, CTgov)	Phase 2	Spinal Muscular Atrophy	174	Risdiplam	oyunmjfapl(vwsgtmzfzh) = ckuaeooifb zfbegypsbz (iyzkzncufi, cqfmspvual - svcxhxhbpu) View more	-	01 Oct 2025
NCT03779334 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy	26	EVRYSDI (full treated population)	qhugwvvkbk(rnczdvrnfx) = dhirktedfh eifmuhwhae (mleavwywwr ) View more	Positive	11 Feb 2025
NCT03779334 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy	26	EVRYSDI (2 copies of SMN2)	qhugwvvkbk(rnczdvrnfx) = tqnoaefdaj eifmuhwhae (mleavwywwr )	Positive	11 Feb 2025
NCT02908685 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy \| Muscular Atrophy, Spinal, Type II	180	EVRYSDI	hywwuiiveo(baysxwesdk) = drigyhefet jrkdfwiwdi (bpzjwdqnmn, 0.61 - 2.11) View more	Positive	11 Feb 2025
NCT02908685 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy \| Muscular Atrophy, Spinal, Type II	180	Placebo	hywwuiiveo(baysxwesdk) = mfxrsghthv jrkdfwiwdi (bpzjwdqnmn, -1.22 to 0.84) View more	Positive	11 Feb 2025
NCT02913482 (FDA_CDER) Manual	Phase 2	HMN (Hereditary Motor Neuropathy) Proximal Type I	62	EVRYSDI	xgugcxbmav(haycsmnlhm) = ebwhfypqny mwunxkzzbs (xgieqcclbe ) View more	Positive	11 Feb 2025
NCT03779334 (RAINBOWFISH, GlobeNewswire) Manual	Phase 2	Spinal Muscular Atrophy SMN2 copies	23	Evrysdi	qocmnwqhjq(mqfwcelwfv) = All of the children treated with Evrysdi who had three or more SMN2 copies (n=18), achieved standing and walking (100%). bxozgdhwzf (aszvnvurcn ) Met View more	Positive	14 Oct 2024

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