Rocket Pharmaceuticals, Inc. Announces Fda Acceptance of Biologics License Application with Priority Review for Rp-L201 (Marnetegragene Autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (Lad-I)
October 02, 2023 at 07:00 am EDT
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Rocket Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) and granted Priority Review for RP-L201 (marnetegragene autotemcel), a lentiviral vector (LV)-based investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I), a rare genetic immune disorder that predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant. The PDUFA date set by the FDA is March 31, 2024. Positive top-line data from the global Phase 1/2 study of RP-L201 demonstrated 100% overall survival at 12 months post-infusion (and for the entire duration of follow-up) for all nine LAD-I patients with 12 to 24 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 RP-L201 was very well tolerated in all patients with no treatment related SAEs. RP-L201 is an investigational gene therapy that contains autologous (patient-derived) hematopoietic stem cells that have been genetically modified with a lentiviral vector to deliver a functional copy of the ITGB2 gene,which encodes for the beta-2 integrin component CD18, a key protein that facilitates leukocyte adhesion and enables their extravasation from blood vessels to fight infection. Rocket holds FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric, and Fast Track designations in the U.S., PRIME and Advanced Therapy
Medicinal Product (ATMP) designations in the EU, and Orphan Drug designation in both regions for the program. RP-L201 was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras and Instituto de Investigación Sanitaria Fundación Jiménez Díaz. The lentiviral vector was developed in a collaboration between UCL and CIEMAT.
Rocket Pharmaceuticals, Inc. is a fully integrated, late-stage biotechnology company. The Company is advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Its lentiviral (LV) vector-based gene therapies target hematologic diseases and consist of late-stage programs for Fanconi Anemia, a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. It also has two clinical stages and one pre-clinical stage in vivo adeno-associated virus (AAV) programs, including programs for Danon disease, Plakophilin-2 Arrhythmogenic Cardiomyopathy and BAG3 Dilated Cardiomyopathy.
Rocket Pharmaceuticals, Inc. Announces Fda Acceptance of Biologics License Application with Priority Review for Rp-L201 (Marnetegragene Autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (Lad-I)
ROCKET PHARMACEUTICALS, INC. 
