Scholar Rock (NASDAQ: SRRK) has reported that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the apitegromab Biologics License Application (BLA) for the treatment of patients with spinal muscular atrophy (SMA). The CRL is related to observations identified during a routine general site inspection of Catalent Indiana LLC, a third-party fill-finish facility. The observations are not specific to apitegromab, and no other approvability concerns were cited in the CRL, including the drug's efficacy and safety data or the third-party drug substance manufacturer.
Catalent Indiana submitted a comprehensive response in early August 2025 to address the observations noted by the FDA. Upon successful remediation of the FDA observations by Catalent Indiana, Scholar Rock intends to resubmit the apitegromab BLA. The company believes that the FDA will be able to act expeditiously on the application once the manufacturing issues have been resolved.
Apitegromab, an investigational fully human monoclonal antibody inhibiting myostatin activation, has been granted orphan drug designation, rare pediatric disease designation, priority review, and fast track designation by the FDA, recognizing its potential to treat a serious condition and fill an unmet medical need. The marketing authorization application (MAA) for apitegromab is also under review by the European Medicines Agency, with a decision anticipated near mid-2026. The European launch is expected in the second half of 2026, with Germany anticipated to be the first European market with patient access.
Scholar Rock remains focused on working hand-in-hand with the FDA to pursue approval of the first and only muscle-targeted treatment for people living with SMA. The company is committed to continuing to work closely with Catalent Indiana to resolve the manufacturing observations and resubmit the apitegromab BLA as soon as possible.
Spinal muscular atrophy (SMA) is a rare, severe, genetic neuromuscular disease characterized by the irreversible loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk, and progressive muscle wasting. The disease causes continuous motor function decline throughout life and can diminish the independence of both children and adults. It is estimated that approximately 35,000 SMA patients globally have been treated with an SMA-targeted treatment.
Scholar Rock is a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for children and adults with SMA and other rare, severe, and debilitating neuromuscular diseases. The company's commitment to unlocking 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. Following these announcements, the company's shares moved 2.23%, and are now trading at a price of $33.305. For more information, read the company's full 8-K submission here.
