FDA Rejects Rocket's (RCKT) Gene Therapy BLA for Rare Disease

Rocket Pharmaceuticals (RCKT) announced that the FDA issued a complete response letter (CRL) to its biologics license application (BLA) seeking approval for its gene therapy Kresladi (marnetegragene autotemcel) to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder. Per the CRL, the FDA sought 'limited' additional information on the therapy's Chemistry Manufacturing and Controls (CMC), which is a part of the BLA submission. The goal of CMC is to ensure that every batc ...