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Rocket Pharma gets FDA review extension review of gene therapy

EditorEmilio Ghigini
Published 13/02/2024, 13:14
© Reuters.
RCKT
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CRANBURY, N.J. - Rocket Pharmaceuticals, Inc. (NASDAQ: NASDAQ:RCKT) announced that the U.S. Food and Drug Administration (FDA) has extended the Priority Review period for its Biologics License Application (BLA) for KRESLADI™, a gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I). The new Prescription Drug User Fee Act (PDUFA) date is now set for June 30, 2024, to provide the FDA with additional time to review supplementary Chemistry, Manufacturing, and Controls (CMC) information provided by the company.

Gaurav Shah, M.D., CEO of Rocket Pharma, expressed the company's commitment to working with the FDA to expedite the availability of KRESLADI™ for patients with severe LAD-I, a rare genetic disorder that severely compromises the immune system. He emphasized the importance of the therapy and the company's confidence in its potential.

The therapy has shown promising results in a global Phase 1/2 study, with a 100% overall survival rate at 12 months post-infusion for all nine LAD-I patients and significant reductions in infection rates and healing of LAD-I-related skin lesions. The study met all primary and secondary endpoints, and KRESLADI™ was well tolerated with no serious adverse events related to the treatment.

KRESLADI™ is an investigational gene therapy that uses a patient's own genetically modified stem cells to treat LAD-I, a disease characterized by mutations in the ITGB2 gene. This gene is crucial for leukocyte adhesion, which is essential for fighting infections. Currently, the only potential cure for severe LAD-I is a bone marrow transplant, which has significant risks and may not be available in time for affected children.

Rocket Pharmaceuticals has received several designations for KRESLADI™, including FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric, and Fast Track designations, as well as PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU, and Orphan Drug designations in both the U.S. and EU.

The company's research into LAD-I is supported by a grant from the California Institute for Regenerative Medicine, and its pipeline includes other gene therapies targeting various rare genetic disorders.

This news is based on a press release statement from Rocket Pharmaceuticals, Inc.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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