REGULATORY NEWS | EMA Accepts Rocket Pharma’s Gene Therapy Application for Fanconi Anemia
The European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102, Rocket Pharma’s lentiviral (LV) vector-based investigational gene therapy for Fanconi Anemia.
RP-L102 is a gene therapy containing autologous hematopoietic stem cells that have been modified to contain a functional copy of the FANCA gene. Mutations in FANCA hinder DNA repair and occur in 60% to 70% of Fanconi Anemia cases. Patients with this disorder experience bone marrow failure, congenital malformations, and a predisposition to cancer.
RP-L102 has the Priority Medicines and Advanced Therapy Medicinal Product designations in the European Union and the Regenerative Medicine Advanced Therapy, Rare Pediatric Disease, and Fast Track designations in the United States.
“The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to patients impacted by this devastating childhood disorder. Currently, there are no existing options to potentially prevent BMF for patients with Fanconi Anemia (FA)” said Kinnari Patel, Pharm.D., MBA, President, Head of R&D and Chief Operating Officer, Rocket Pharma.
MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial. RP-L102 demonstrated sustained genetic correction, comprehensive phenotypic correction, and hematologic stabilization. The safety profile was highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, was well tolerated. There were no signs of bone marrow dysplasia, clonal dominance, or insertional mutagenesis related to RP-L102.
In the absence of allogeneic hematopoietic stem cell transplant (HSCT), the primary cause of death among patients with FA is bone marrow failure (BMF), which typically occurs during the first decade of life. Although allogeneic transplants can cure the hematologic component of FA, they confer significant side effects and substantially increase the risk of solid organ malignancies, which have become the most frequent cause of FA-related death.
The Biologics License Application (BLA) for FA remains on track for submission to the U.S. Food and Drug Administration (FDA) in the first half of 2024.
Source: Rocket Pharma Press Release
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