Scholar Rock Submits Biologics License Application (BLA) to the U.S. FDA for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA)
Scholar Rock (SRRK) has submitted a Biologics License Application (BLA) to the FDA for apitegromab, a muscle-targeted therapy for Spinal Muscular Atrophy (SMA) patients. The submission is based on the successful Phase 3 SAPPHIRE trial, which demonstrated a statistically significant 1.8-point improvement in motor function compared to placebo at week 52, measured by the Hammersmith Functional Motor Scale-Expanded.
The company has received Fast Track, Orphan Drug, and Rare Pediatric Disease Designations from the FDA, and plans to submit a Marketing Authorisation Application to the European Medicines Agency in Q1 2025. Scholar Rock has requested Priority Review, which could reduce FDA review time to six months. Additionally, the company plans to initiate the Phase 2 OPAL trial in mid-2025 to study apitegromab in SMA patients under two years of age.
Scholar Rock (SRRK) ha presentato una Domanda di Licenza Biologici (BLA) alla FDA per apitegromab, una terapia mirata ai muscoli per i pazienti affetti da Atrofia Muscolare Spinale (SMA). La presentazione si basa sul successo del trial di Fase 3 SAPPHIRE, che ha dimostrato un miglioramento statisticamente significativo di 1,8 punti nella funzione motorie rispetto al placebo alla settimana 52, misurato con la Hammersmith Functional Motor Scale-Expanded.
L’azienda ha ricevuto le designazioni di Fast Track, Orphan Drug e Rare Pediatric Disease dalla FDA, e prevede di presentare una Domanda di Autorizzazione al Marketing all’Agenzia Europea dei Farmaci nel primo trimestre del 2025. Scholar Rock ha richiesto una Revisione Prioritaria, che potrebbe ridurre il tempo di revisione della FDA a sei mesi. Inoltre, l'azienda intende avviare il trial di Fase 2 OPAL a metà del 2025 per studiare apitegromab nei pazienti SMA di età inferiore ai due anni.
Scholar Rock (SRRK) ha presentado una Solicitud de Licencia Biológica (BLA) a la FDA para apitegromab, una terapia dirigida a los músculos para pacientes con Atrofia Muscular Espinal (SMA). La presentación se basa en el exitoso ensayo de Fase 3 SAPPHIRE, que demostró una mejora estadísticamente significativa de 1.8 puntos en la función motora en comparación con el placebo a la semana 52, medida por la Hammersmith Functional Motor Scale-Expanded.
La compañía ha recibido las designaciones de Fast Track, Orphan Drug y Rare Pediatric Disease de la FDA, y planea presentar una Solicitud de Autorización de Comercialización a la Agencia Europea de Medicamentos en el primer trimestre de 2025. Scholar Rock ha solicitado una Revisión Prioritaria, lo que podría reducir el tiempo de revisión de la FDA a seis meses. Además, la compañía planea iniciar el ensayo de Fase 2 OPAL a mediados de 2025 para estudiar apitegromab en pacientes de SMA menores de dos años.
스콜라 록(SRRK)는 FDA에 아피테그로맙에 대한 생물학적 제제 허가 신청(BLA)을 제출했습니다. 이는 척수성 근육 위축(SMA) 환자를 위한 근육 표적 치료제입니다. 제출은 3상 SAPPHIRE 시험의 성공을 기반으로 하며, 52주차에 Hammersmith Functional Motor Scale-Expanded를 통해 분석한 결과, 위약 대비 운동 기능이 통계적으로 유의미하게 1.8점 향상된 것으로 나타났습니다.
회사는 FDA로부터 패스트 트랙, 고아 의약품 및 희귀 소아질환 지정 승인을 받았으며, 2025년 1분기 내에 유럽 의약청에 마케팅 승인 신청서를 제출할 계획입니다. 스콜라 록은 우선 심사를 요청했으며, 이를 통해 FDA 심사 시간이 6개월로 단축될 수 있습니다. 또한 회사는 2025년 중반에 2상 OPAL 시험을 시작하여 2세 미만의 SMA 환자에서 아피테그로맙을 연구할 계획입니다.
Scholar Rock (SRRK) a soumis une Demande de Licence Biologique (BLA) à la FDA pour apitegromab, une thérapie ciblée sur les muscles pour les patients atteints d'Atrophie Musculaire Spinale (SMA). La soumission est basée sur le succès du essai de Phase 3 SAPPHIRE, qui a démontré une amélioration statistiquement significative de 1,8 point de la fonction motrice par rapport au placebo à la semaine 52, mesurée par l'échelle de Hammersmith Functional Motor Scale-Expanded.
L'entreprise a reçu les désignations Fast Track, Orphan Drug et Rare Pediatric Disease de la FDA, et prévoit de soumettre une Demande d'Autorisation de Mise sur le Marché à l'Agence Européenne des Médicaments au premier trimestre 2025. Scholar Rock a demandé une Révision Prioritaire, ce qui pourrait réduire le temps de révision de la FDA à six mois. De plus, l'entreprise prévoit d'initier l'essai de Phase 2 OPAL au milieu de 2025 pour étudier apitegromab chez les patients atteints de SMA âgés de moins de deux ans.
Scholar Rock (SRRK) hat einen Antrag auf Zulassung für Biologika (BLA) bei der FDA für apitegromab eingereicht, eine muskelgerichtete Therapie für Patienten mit spinaler Muskelatrophie (SMA). Die Einreichung basiert auf der erfolgreichen Phase-3-Studie SAPPHIRE, die eine statistisch signifikante Verbesserung der motorischen Funktion um 1,8 Punkte gegenüber dem Placebo in Woche 52 zeigte, gemessen mit der Hammersmith Functional Motor Scale-Expanded.
Das Unternehmen erhielt von der FDA die Auszeichnungen Fast Track, Orphan Drug und Rare Pediatric Disease und plant, im ersten Quartal 2025 einen Antrag auf Marktzulassung bei der Europäischen Arzneimittel-Agentur einzureichen. Scholar Rock hat eine Prioritätsprüfung beantragt, die die Überprüfungszeit der FDA auf sechs Monate verkürzen könnte. Zudem plant das Unternehmen, Mitte 2025 die Phase-2-Studie OPAL zu initiieren, um apitegromab bei SMA-Patienten unter zwei Jahren zu untersuchen.
- Phase 3 SAPPHIRE trial met primary endpoint with statistically significant improvement
- Multiple FDA designations received (Fast Track, Orphan Drug, Rare Pediatric Disease)
- Potential for expedited 6-month FDA review if Priority Review is granted
- Planned expansion into younger patient population through Phase 2 OPAL trial
- European market entry delayed until at least 2025
- Additional clinical trials required for under-two age group
Insights
The BLA submission for apitegromab marks a pivotal moment in SMA treatment development, particularly significant as it represents a first-in-class muscle-directed therapy. The 1.8-point improvement in HFMSE scores, while appearing modest, is clinically meaningful in SMA patients where maintaining or slightly improving motor function can significantly impact quality of life.
The drug's positioning as a complementary therapy to existing SMN-targeted treatments (nusinersen, risdiplam and onasemnogene abeparvovec) is strategically sound, potentially creating a multi-billion dollar market opportunity. Current SMA treatments focus on SMN protein production, while apitegromab's unique mechanism targeting muscle growth offers a complementary approach that could become standard combination therapy.
The regulatory momentum is substantial, with Fast Track, Orphan Drug and Rare Pediatric Disease designations in the US, plus PRIME and Orphan designations in Europe. These designations not only expedite the review process but also provide market exclusivity and potential priority review voucher benefits. The planned OPAL trial in patients under two years old is particularly strategic, as early intervention in SMA is important for optimal outcomes.
The parallel pursuit of US and European approvals, with the MAA submission planned for Q1 2025, demonstrates strong execution of the global commercialization strategy. If approved, apitegromab could become the first muscle-directed therapy for SMA, potentially transforming the treatment paradigm from single to combination therapy approaches.
- Scholar Rock’s BLA submission is based on the Phase 3 SAPPHIRE trial that demonstrated a statistically significant improvement in motor function for patients receiving apitegromab compared to placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at week 52
- The FDA has granted apitegromab Fast Track, Orphan Drug and Rare Pediatric Disease Designations in SMA
- Scholar Rock remains on track to submit the apitegromab Marketing Authorisation Application (MAA) to the European Medicines Agency in 1Q 2025, with PRIME and Orphan Medicinal Product Designations
“We are gratified that in patients already on a SMN-targeted treatment, the SAPPHIRE trial met its primary endpoint for the main efficacy population showing a statistically significant 1.8-point improvement for patients receiving apitegromab compared to placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at week 52,” said Jing Marantz, M.D., Ph.D., Chief Medical Officer of Scholar Rock. “With the strength of our Phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the FDA through the review of our BLA on behalf of patients and families living with SMA.”
The review of the apitegromab BLA submission will be conducted by the Division of Neurology Products in FDA’s Center for Drug Evaluation and Research. The submission is supported by data from the Phase 3 SAPPHIRE trial and the Phase 2 TOPAZ trial. The Company shared positive topline data from the SAPPHIRE trial in 2024, and additional data will be presented at the 2025 MDA Clinical & Scientific Conference being held March 16-19 in
Scholar Rock has requested Priority Review which, if granted, would shorten the FDA’s review time to six months from the date of filing acceptance. The FDA has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations to apitegromab for the treatment of SMA.
Scholar Rock plans to also study apitegromab in SMA patients under two years of age in the Phase 2 OPAL trial, with a planned initiation in mid-2025. The trial will evaluate apitegromab in patients who have been or are continuing to be treated with any currently approved SMN therapy (nusinersen, risdiplam and onasemnogene abeparvovec).
About Apitegromab
Apitegromab is an investigational fully human monoclonal antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle. It is the first muscle-targeted treatment candidate to demonstrate clinically meaningful and statistically significant motor function improvement in a pivotal Phase 3 trial in spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. The
About the Phase 3 SAPPHIRE Trial
SAPPHIRE was a randomized, double-blind, placebo-controlled Phase 3 clinical trial that evaluated the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 SMA who were receiving current standard of care (either nusinersen or risdiplam). SAPPHIRE enrolled 156 patients aged 2-12 years old in the main efficacy population. These patients were randomized 1:1:1 to receive either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks for 12 months. An exploratory population including 32 patients aged 13-21 years old was also evaluated. These patients were randomized 2:1 to receive either apitegromab 20 mg/kg or placebo every 4 weeks for 12 months.
The SAPPHIRE trial met its primary endpoint for the main efficacy population with a statistically significant 1.8-point improvement for all patients receiving apitegromab 10 mg/kg and 20 mg/kg (with an SMN-targeted treatment) compared to placebo (SMN-targeted treatments), as measured by the Hammersmith Functional Motor Scale-Expanded at week 52.
About Scholar Rock
Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need. As a global leader in the biology of the transforming growth factor beta (TGFβ) superfamily and named for the visual resemblance of a scholar rock to protein structures, the clinical-stage company is focused on advancing innovative treatments where protein growth factors are fundamental. Over the past decade, Scholar Rock has created a pipeline with the potential to advance the standard of care for neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted drugs can play a transformational role.
This commitment to unlocking fundamentally different therapeutic approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. By harnessing cutting-edge science in disease spaces that are historically under-addressed through traditional therapies, Scholar Rock works every day to create new possibilities for patients. Learn more about our approach at ScholarRock.com and follow @ScholarRock and on LinkedIn.
Scholar Rock® is a registered 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 www.scholarrock.com, 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 Twitter and LinkedIn. The information that we post on our website or on 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, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its progress and plans for apitegromab. The use of words such as “may,” “could,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, that preclinical and clinical data, including the results from the Phase 3 trial of apitegromab, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidate; Scholar Rock’s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; the success of Scholar Rock’s current and potential future collaborations; Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials; Scholar Rock’s ability to manage expenses and to obtain additional funding when needed to support its business activities; its ability to establish or maintain strategic business alliances; its ability to receive priority or expedited regulatory review or to obtain regulatory approval of apitegromab; its ability to expand globally and the anticipated commercial launch in
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