In a setback for Rocket Pharmaceuticals, the FDA has issued a Complete Response Letter rejecting its gene therapy, Kresladi, designed to treat severe leukocyte adhesion deficiency-I (LAD-I). The agency’s decision comes with a request for additional chemistry, manufacturing, and controls (CMC) information to complete their review process.
Initially slated for a March 2024 decision, Kresladi’s fate was delayed by the FDA, pushing the Prescription Drug User Fee Act (PDUFA) date to June 30. Rocket Pharmaceuticals expressed readiness to provide the necessary CMC data following discussions with FDA’s Center for Biologics Evaluation and Research (CBER), aiming to expedite the therapy’s approval.
Rocket CEO Gaurav Shah emphasized the FDA’s understanding of the therapy’s crucial medical necessity and clinical benefits, despite the setback causing an 11% drop in Rocket’s stock price during premarket trading on Friday.
Kresladi, utilizing a lentiviral vector, targets LAD-I, a rare genetic condition affecting pediatric patients. The therapy’s Biologics License Application (BLA) was accepted last October, supported by promising Phase I/II data demonstrating significant survival rates and clinical improvements in treated patients.
This development places Rocket among several biotech firms encountering FDA hurdles this week. Merck and Daiichi Sankyo received a CRL for their lung cancer treatment, and AbbVie faced an FDA denial for its Parkinson’s disease candidate, all related to manufacturing issues at third-party facilities.
Rocket Pharmaceuticals remains optimistic about addressing FDA concerns swiftly to bring Kresladi to patients grappling with LAD-I, underscoring the critical need for timely approval in the rare disease space.