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CAMBRIDGE, Mass. - Scholar Rock (SRRK), a biopharmaceutical company valued at $3.2 billion, announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, a potential treatment for spinal muscular atrophy (SMA). The FDA’s priority review means a decision is expected by September 22, 2025, signaling the treatment’s promise for significant improvement over existing therapies for the serious condition. The company’s stock has shown remarkable momentum, with InvestingPro data showing a 293% surge over the past six months.
The European Medicines Agency (EMA) is also reviewing the company’s Marketing Authorization Application for apitegromab, indicating concurrent regulatory processes in the U.S and Europe.
Apitegromab, a muscle-targeted therapy, has shown in the Phase 3 SAPPHIRE trial to statistically and clinically improve motor function in SMA patients receiving standard SMN-targeted treatments. The trial’s success, based on the Hammersmith Functional Motor Scale Expanded (HFMSE), was presented at the 2025 Muscular Dystrophy Association Conference.
Scholar Rock is preparing for a U.S. commercial launch in 2025, pending approval, with a European launch anticipated in 2026. Apitegromab has already received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, and Priority Medicines (PRIME) and Orphan Medicinal Product designations from the EMA.
The company’s focus on neuromuscular and other serious diseases is supported by a proprietary platform that develops selective monoclonal antibodies to modulate protein growth factors. Scholar Rock’s pipeline and approach to growth factor-targeted drugs are expected to advance the standard of care in various conditions.
This announcement is based on a press release statement and does not represent an endorsement of apitegromab’s efficacy or potential market success. The treatment’s approval and commercial availability remain subject to regulatory review outcomes. Analysts maintain an optimistic outlook, with price targets ranging from $45 to $57. For deeper insights into Scholar Rock’s financial health, growth prospects, and 10+ additional ProTips, visit InvestingPro, where you’ll find comprehensive analysis in the Pro Research Report.
In other recent news, Scholar Rock reported a significant earnings miss for Q4 2024, with earnings per share (EPS) of -$2.47, falling short of the forecasted -$0.58. Despite this, the company is preparing for a potential commercial launch of its spinal muscular atrophy (SMA) treatment in Q4 2025, indicating a focus on future product launches. Scholar Rock has also granted inducement equity awards to seven newly hired employees, consisting of stock options and restricted stock units for a total of 113,800 shares of common stock. These awards are part of the company’s strategy to attract new talent. Additionally, the company is planning to expand its neuromuscular franchise and explore new indications, with a focus on innovative, targeted therapies. Scholar Rock is preparing to file an Investigational New Drug (IND) application for SRK-439 in Q3 2025, aiming to address the cardiometabolic market. Analyst firms have shown interest in the company’s strategic initiatives, which include upcoming product launches and market expansion. These developments reflect Scholar Rock’s ongoing efforts to strengthen its market position and drive future growth.
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