Rocket Pharmaceuticals, Inc. (RCKT) announced that the FDA has issued a Complete Response Letter concerning the Biologics License Application for Kresladi. The letter requests additional information on Chemistry Manufacturing and Controls (CMC) to finalize the review.
The company has engaged with senior officials from the FDA’s Center for Biologics Evaluation and Research to clarify the specific CMC details required to expedite Kresladi's approval.
The global Phase 1/2 study of Kresladi has successfully met all primary and secondary endpoints. The results demonstrate a 100% overall survival rate at 12 months post-infusion, significant reductions in infection rates compared to pre-treatment levels, and no serious adverse events related to the treatment.
Kresladi is a lentiviral vector-based gene therapy aimed at treating severe leukocyte adhesion deficiency-I, a rare pediatric disease with autosomal recessive inheritance.
