FDA Rejects Rocket Pharmaceuticals' Gene Therapy For Rare Immune Disorder, Asks For More Data, Stock Slides

Rocket Pharmaceuticals Inc (NASDAQ:RCKT) stock is trading down after the company announced a regulatory update for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral (LV) vector-based gene therapy to treat severe leukocyte adhesion deficiency-I (LAD-I).

The FDA has issued a Complete Response Letter (CRL) to Rocket’s Biologics License Application for Kresladi, in which the FDA requested limited additional Chemistry Manufacturing and Controls (CMC) information to complete its review.

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

Topline data from the global Phase 1/2 study of Kresladi demonstrated 100% overall survival at 12 months post-infusion (and for the entire duration of follow-up) for all nine LAD-I patients with 18 to 42 months of available follow-up.

Data also showed large decreases in the incidences of significant infections compared with the pre-treatment history, combined with evidence of resolution of LAD-I-related skin lesions and restoration of wound repair capabilities.

All primary and secondary endpoints were met, and Kresladi was well tolerated in all patients with no treatment-related serious adverse events.

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18.

CD18 is a key protein facilitating leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I suffer from recurrent bacterial and fungal infections.

Price Action: At last check on Friday, RCKT shares were down 9.01% at $19.50.