Rocket Pharmaceuticals: FDA Grants Priority Review For RP-L201 - Quick Facts

(RTTNews) - Rocket Pharmaceuticals, Inc. (RCKT) announced the FDA has accepted the Biologics License Application and granted Priority Review for RP-L201, a lentiviral vector-based investigational gene therapy for severe Leukocyte Adhesion Deficiency-I. The PDUFA date is March 31, 2024.

Severe Leukocyte Adhesion Deficiency-I is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. This rare disorder predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant. LAD-I is estimated to impact an estimated 800 to 1,000 individuals in the U.S. and Europe.

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