Scholar Rock (NASDAQ: SRRK) has received a complete response letter (CRL) from the U.S. Food and Drug Administration (FDA) for the apitegromab biologics license application (BLA) for the treatment of patients with spinal muscular atrophy (SMA). This 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.
The company intends to resubmit the apitegromab BLA upon the resolution of the observations related to Catalent Indiana LLC. It's worth noting that no other approvability issues were cited in the CRL, including concerns related to apitegromab’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. Following that submission, Catalent Indiana has continued to work rapidly to take corrective action and has kept the FDA apprised of that progress.
Once Catalent Indiana’s successful remediation of the FDA observations is achieved, Scholar Rock will 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.
It's important to note that apitegromab was granted orphan drug designation, rare pediatric disease designation, priority review, and fast track designation, recognizing its potential to treat a serious condition and fill an unmet medical need. The European Medicines Agency is also reviewing the apitegromab marketing authorization application, and a decision is anticipated near mid-2026, with a European launch expected in the second half of 2026.
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 SMA to demonstrate clinical success in a pivotal phase 3 clinical trial.
Spinal muscular atrophy 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. It's estimated that approximately 35,000 SMA patients globally have been treated with an SMN-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 is named for the visual resemblance of a scholar rock to protein structures and is committed to unlocking fundamentally different treatment approaches through its highly innovative anti-myostatin program.
Today the company's shares have moved 5.64% to a price of $32.58. If you want to know more, read the company's complete 8-K report here.
