The U.S. Food and Drug Administration announced on Tuesday, September 23, 2025, that it has declined to approve Scholar Rock’s apitegromab for the treatment of spinal muscular atrophy. The decision was formalized through a Complete Response Letter (CRL) that cited observations identified during a routine general site inspection of Catalent Indiana LLC, a third‑party fill‑finish facility acquired by Novo Nordisk in December 2024.
The CRL did not raise any additional approvability concerns beyond the manufacturing observations, and it did not reference apitegromab’s efficacy or safety data. The observations were not specific to apitegromab, but they were sufficient for the FDA to withhold approval until the issues are resolved. The letter effectively pauses the company’s priority‑review BLA and postpones the anticipated U.S. launch that was targeted for the end of 2025.
This regulatory setback delays Scholar Rock’s commercial entry into the SMA market, potentially postponing revenue generation and affecting investor expectations. The company’s ability to resume the approval process will depend on the resolution of the manufacturing issues at Catalent Indiana, underscoring the critical role of third‑party manufacturing compliance in the drug approval pathway.
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