FDA slaps CRL on Rocket Pharma’s Kresladi

Late-stage US biotech Rocket Pharmaceutical’s (Nasdaq: RCKT) shares slumped 12.5% in pre-market activity, but closed down just 2% at $21.10 on Friday after it announced a setback regarding approval of its gene therapy Kresladi (marnetegragene autotemcel; marne-cel).

Rocket revealed that the US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to Rocket’s Biologics License Application (BLA) for Kresladi wherein the FDA requested only limited additional Chemistry Manufacturing and Controls (CMC) information to complete its review. The FDA already extended the Priority Review period for the BLA in February tis year.

Rocket said it met with FDA senior leaders from Center for Biologics Evaluation and Research (CBER) to align on the limited scope of additional CMC information needed to support the approval of Kresladi as quickly as possible.