Rocket Pharmaceuticals has withdrawn its biologics license application (BLA) for RP-L201, an experimental gene therapy targeting Leukocyte Adhesion Deficiency-I (LAD-I), a rare inherited blood disorder. The application was under review by the FDA, with hopes for RP-L201 to become the first approved gene therapy for this severe immune deficiency. The company cited ongoing discussions with the FDA regarding additional data requirements and clinical protocol clarifications as the reason for the withdrawal.
Leukocyte Adhesion Deficiency-I, affecting only a handful of patients globally, is marked by recurrent and life-threatening infections due to an inability of white blood cells to migrate normally. RP-L201 uses a lentiviral vector to deliver the corrected gene to patients’ blood-forming stem cells, aiming for a one-time curative approach. Rocket Pharmaceuticals emphasized that no new safety concerns had arisen, and intends to resubmit the application after addressing regulatory queries and supplementing trial data.
Analysts note that the withdrawal highlights challenges facing first-in-class gene therapies, including regulatory uncertainties and high data standards. Despite the setback, Rocket maintains optimism for RP-L201’s eventual approval, citing durable clinical benefit observed in treated patients.
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox!
Sign up now!