Scholar Rock's Apitegromab BLA for SMA Faces FDA Complete Response Letter Due to Fill-Finish Facility Issues

Scholar Rock has received a Complete Response Letter from the FDA for the apitegromab Biologics License Application due to issues at a third-party fill-finish facility, Catalent Indiana LLC. The company plans to resubmit the application once manufacturing issues are resolved, and the FDA will review it expeditiously. Apitegromab is a treatment for spinal muscular atrophy and has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, and Priority Medicines and Orphan Medicinal Product designations from the EMA.

Scholar Rock, a biopharmaceutical company focused on rare neuromuscular diseases, has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for its apitegromab Biologics License Application (BLA). The CRL is related to observations identified during a routine general site inspection of Catalent Indiana LLC, a third-party fill-finish facility acquired by Novo Nordisk A/S in December 2024 FDA Issues Complete Response Letter (CRL) for ...^[1].

The CRL did not cite any other approvability concerns, including apitegromab’s efficacy and safety data or the third-party drug substance manufacturer. The observations are not specific to apitegromab and were discussed during Scholar Rock’s second quarter 2025 earnings announcement. Catalent Indiana submitted a comprehensive response in early August 2025 to address the FDA observations and has continued to work rapidly to take corrective action FDA Issues Complete Response Letter (CRL) for ...^[1].

Upon Catalent Indiana’s successful remediation of the FDA observations, 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. David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock, stated, "We are continuing to work closely with Catalent Indiana on the FDA’s manufacturing observations so that we can resubmit the apitegromab BLA as soon as possible" FDA Issues Complete Response Letter (CRL) for ...^[1].

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 in spinal muscular atrophy (SMA) to demonstrate clinical success in a pivotal Phase 3 clinical trial. The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations to apitegromab for the treatment of SMA FDA Issues Complete Response Letter (CRL) for ...^[1].

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. 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 FDA Issues Complete Response Letter (CRL) for ...^[1].

Scholar Rock is committed to developing and commercializing apitegromab for children and adults with SMA and other rare, severe, and debilitating neuromuscular diseases. The company’s proprietary platform focuses on developing novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity FDA Issues Complete Response Letter (CRL) for ...^[1].

Outside of the U.S., the apitegromab marketing authorisation application (MAA) is under review by the European Medicines Agency (EMA) and a decision is anticipated near mid-2026. European launch is anticipated in the second half of 2026, with Germany expected to be the first European market with patient access FDA Issues Complete Response Letter (CRL) for ...^[1].