Scholar Rock Advances Apitegromab with FDA After Setback
Company reports constructive FDA meeting for its lead SMA drug, signaling a clear path to a 2026 BLA resubmission despite recent manufacturing hurdles.
Scholar Rock (NASDAQ: SRRK) has signaled a clear path forward for its lead drug candidate, apitegromab, following a constructive meeting with the U.S. Food and Drug Administration. The discussions addressed a manufacturing-related setback that had previously delayed the drug's approval process, renewing optimism for a 2026 commercial launch for the novel Spinal Muscular Atrophy (SMA) therapy.
The Cambridge-based biopharmaceutical firm announced the successful Type A meeting with the FDA, which was held to resolve issues cited in a Complete Response Letter (CRL) from September 2025. That letter had stalled the Biologics License Application (BLA) for apitegromab, not due to concerns over the drug's safety or efficacy, but because of inspection findings at a third-party manufacturing facility operated by Catalent.
This development provides crucial clarity for investors and stakeholders. Despite the positive news, shares of Scholar Rock saw a slight downturn in morning trading, dipping 2.43% to $30.07. The response suggests a market that is cautiously optimistic, weighing the cleared regulatory pathway against the timeline to resubmission and eventual revenue generation.
Overcoming a Manufacturing Hurdle
The initial rejection was a significant but temporary obstacle. According to the company's Q3 update, the FDA's concerns were confined to the contract manufacturer, insulating the clinical data from apitegromab's robust Phase 3 SAPPHIRE trial. Scholar Rock has since moved proactively to mitigate these risks, securing a secondary U.S. fill-finish facility and reserving commercial capacity for the first quarter of 2026.
Management now anticipates resubmitting the BLA in 2026, which could position apitegromab for a U.S. market launch within the same year. A European launch is also anticipated in the second half of 2026. Apitegromab, a muscle-directed therapy, is designed to be the first of its kind to improve motor function in patients with SMA, a rare and serious genetic disease affecting muscle strength and movement.
Financial Health and Market Position
The company's strategic planning is supported by a strong balance sheet. Scholar Rock reported cash, cash equivalents, and marketable securities of $369.6 million as of September 30, 2025. This financial runway is projected to fund operations into 2027, providing ample resources to navigate the final stages of the regulatory process and prepare for commercialization without immediate financing pressures.
While the company reported a third-quarter earnings per share miss of $-0.90 against an estimated $-0.84, investors have largely focused on the positive regulatory progress for its lead asset. The potential of apitegromab has kept Wall Street sentiment broadly positive. The stock holds a consensus "Strong Buy" rating from 13 analysts, with an average price target of $46.55, implying significant upside from its current trading level. Some analysts viewed the September dip as a buying opportunity, confident that the manufacturing issues were solvable.
A De-Risked Path Forward
The resolution of the manufacturing overhang significantly de-risks the path to approval for what could be a transformative therapy for SMA patients. Apitegromab works by inhibiting myostatin, a protein that limits muscle growth, and is intended to be used in conjunction with existing foundational SMA therapies.
"The constructive engagement with the FDA provides us with a clear path forward to bring apitegromab to patients with SMA," the company stated in its press release. With the clinical efficacy and safety profile already established, the primary remaining hurdle is the successful reinspection of its manufacturing partner and the formal resubmission of its BLA.
For Scholar Rock, the coming months will be critical. The successful execution of its manufacturing and regulatory strategy could unlock substantial value and position the company as a key player in the rare disease market. Investors will be closely watching for updates on its BLA resubmission and the subsequent FDA review, which will be the ultimate catalyst for the company's transition from a clinical-stage to a commercial-stage enterprise.