Scholar Rock receives CRL from FDA for apitegromab BLA

Scholar Rock (SRRK) announced that the FDA has issued a complete response letter for the apitegromab biologics license application for the treatment of patients with spinal muscular atrophy. The CRL is related to observations identified during a routine general site inspection of Catalent Indiana, a third-party fill-finish facility which was acquired by Novo Nordisk (NVO) in December 2024. The observations are not specific to apitegromab. The CRL did not cite any other approvability concerns, including apitegromab’s efficacy and safety data or the third-party drug substance manufacturer. The Catalent Indiana observations were discussed during Scholar Rock’s second quarter earnings announcement. Catalent Indiana submitted a response in early August to address the observations noted by the FDA. Following that submission, Catalent Indiana has continued to work to take corrective action and has kept the FDA apprised of that progress. Upon Catalent Indiana’s successful remediation of the FDA observations, Scholar Rock will resubmit the apitegromab BLA. Scholar Rock believes that the FDA will be able to act expeditiously on the application once the manufacturing issues have been resolved.