The Marketing Authorization Approval (MAA) for RP-L102, a lentiviral vector-based investigational gene therapy for Fanconi anemia has been approved by the European Medicines Agency (EMA), according to a release by the drug’s developer, Rocket Pharmaceuticals.
RP-L102 is a gene therapy containing autologous hematopoietic stem cells that have been modified to contain a functional copy of the FANCA gene. 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,” said Kinnari Patel, PharmD, the Chief Operating Officer of Rocket Pharmaceuticals. “We look forward to partnering closely with the EMA throughout the review process to make RP-L102 available to patients with [Fanconi anemia] who are in need of new treatment options.”
The MAA approval was informed by positive findings in a global phase I/II trial on RP-L102 wherein the agent showed sustained genetic correction, comprehensive phenotypic correction, and hematologic stabilization.
The use of RP-L102 may allow patients with Fanconi anemia to avoid allogeneic hematopoietic stem cell transplantation and the related side effects while still preventing bone marrow failure, which is the primary cause of death among patients with Fanconi anemia.
The Biologics License Application for RP-L102 for Fanconi anemia remains on track for submission to the US Food and Drug administration for the first half of 2024, according to Rocket Pharmaceuticals.
Related: What Are the Outcomes of Patients With Fanconi Anemia Undergoing HSCT?
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