What is the story about?
What's Happening?
The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for Scholar Rock's Biologics License Application (BLA) for apitegromab, a treatment for spinal muscular atrophy (SMA). The CRL is related to observations identified during a routine inspection of Catalent Indiana LLC, a third-party fill-finish facility. These observations are not specific to apitegromab itself, and the CRL did not cite any other approvability concerns regarding the drug's efficacy or safety data. Scholar Rock plans to resubmit the BLA once Catalent Indiana resolves the FDA's observations. Apitegromab is a monoclonal antibody targeting myostatin in skeletal muscle, and it has shown clinical success in Phase 3 trials.
Why It's Important?
The delay in the approval of apitegromab affects patients with SMA, a severe genetic neuromuscular disease characterized by muscle wasting and motor function decline. Apitegromab represents a novel muscle-targeted treatment that could significantly improve motor function and independence for SMA patients. The FDA's observations at the Catalent facility, while not related to the drug's efficacy, postpone the availability of this potentially transformative treatment. The situation underscores the importance of manufacturing compliance in the drug approval process, impacting the timeline for new therapies reaching patients.
What's Next?
Scholar Rock is working closely with Catalent Indiana to address the FDA's observations. Once resolved, Scholar Rock intends to resubmit the BLA for apitegromab, anticipating expedited FDA action. The company remains committed to pursuing approval for apitegromab, which has received multiple designations recognizing its potential to address unmet medical needs. Outside the U.S., the European Medicines Agency is reviewing apitegromab, with a decision expected by mid-2026. Scholar Rock plans to launch the drug in Europe, starting with Germany, in the latter half of 2026.
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