Regulatory update on Kresladi (marnetegragene autotemcel; marne-cel) to treat severe leukocyte adhesion deficiency-I.- Rocket Pharma

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

Rocket met with FDA senior leaders from Center for Biologics Evaluation and Research (CBER) to align on the limited scope of additional CMC information needed to support the approval of Kresladi as quickly as possible. “It is reassuring to have the FDA as a close collaborator who understands the high unmet medical need, clear clinical benefit and importance of timely patient access,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. “CBER leadership’s direct involvement and commitment to working expeditiously to deliver this therapy to patients gives us great hope on behalf of the primary immunodeficiency community.”

Positive top-line data from the global Phase 1/II 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 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.