Scholar Rock, Inc. – FDA CRL

The FDA issued a Complete Response Letter (CRL) to Scholar Rock, Inc. for its Biologics License Application (BLA) 761463 for SRK-015 (apitegromab-mstn), indicating the application cannot be approved in its current form due to several outstanding issues. A key concern stemmed from deficiencies identified during a Current Good Manufacturing Practice (CGMP) inspection of a manufacturing facility, requiring the facility to provide satisfactory responses to FDA Form 483 findings, potentially necessitating re-inspection.

Furthermore, the application requires substantial revisions to labeling. Scholar Rock must submit updated draft Prescribing Information and Carton and Container Labeling that conform to regulatory formats and content, including 21 CFR 201.56, 201.57, and submission in Structured Product Labeling (SPL) format. The proposed proprietary name, though conditionally acceptable, needs resubmission once all other deficiencies are resolved.

Additionally, a comprehensive safety update is mandated under 21 CFR 314.50(d)(5)(vi)(b). This update must detail significant changes in the safety profile, present new and combined safety data from all studies, provide case report forms for serious adverse events and discontinuations, and summarize worldwide safety experience. Critically, the FDA found inadequate data on developmental risks associated with apitegromab use during pregnancy. Therefore, if the BLA is approved, Scholar Rock will be required under Section 505(o)(3) of the FDCA to establish a 10-year worldwide single-arm pregnancy safety study to collect and analyze information on maternal, fetal, and infant outcomes.