FDA Rejects Scholar Rock’s SMA Drug Due to Catalent Indiana Manufacturing Issues

The U.S. FDA issued a Complete Response Letter (CRL) declining approval of Scholar Rock's apitegromab for the treatment of spinal muscular atrophy (SMA) due to inspection findings at the Catalent Indiana manufacturing facility123.

The issues cited by the FDA were related to manufacturing practices at Catalent Indiana, a third-party fill-finish facility, and were not specific to apitegromab itself or its safety and efficacy profile1236.

No concerns were identified regarding the drug's efficacy, safety, or the primary drug substance manufacturer236.

Scholar Rock stated that Catalent has submitted corrective responses to the FDA and is implementing further actions to address the agency's observations23.

Scholar Rock plans to resubmit its Biologics License Application (BLA) for apitegromab once the FDA confirms resolution of the manufacturing issues123.

The decision has delayed the potential approval of apitegromab, but the company remains committed to pursuing its approval after remediation3.

Sources:

1. https://trial.medpath.com/news/10916d86f3904dfb/fda-issues-complete-response-letter-for-scholar-rock-s-apitegromab-due-to-manufacturing-issues

2. https://smanewstoday.com/news/fda-denies-apitegromab-approval-sma-cites-issues-facility/

3. https://www.neurologylive.com/view/fda-hands-scholar-rock-crl-sma-agent-apitegromab

6. https://www.contemporarypediatrics.com/view/fda-issues-crl-for-apitegromab-for-sma-citing-facility-inspection