Rocket Pharmaceuticals announces FDA complete response letter for Kresladi

Rocket Pharmaceuticals announced a regulatory update for Kresladi, a lentiviral vector-based gene therapy to treat severe leukocyte adhesion deficiency-I, or LAD-I. The FDA has issued a complete response letter, or CRL, in response to Rocket’s biologics license application for Kresladi wherein the FDA requested limited additional Chemistry Manufacturing and Controls, or CMC, information to complete its review. Rocket met with FDA senior leaders from Center for Biologics Evaluation and Research to align on the limited scope of additional CMC information needed to support the approval of Kresladi as quickly as possible. Positive top-line data from the global Phase 1/2 study of Kresladi demonstrated 100% overall survival at 12 months post-infusion for all nine LAD-I patients with 18 to 42 months of available follow-up. Data also showed large decreases compared with pre-treatment history in the incidences of significant infections, 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.