Rocket Pharmaceuticals Announces European Medicines Agency Acceptance of RP-L102 Marketing Authorization Application for the Treatment of Fanconi Anemia
“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.
- “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 FA,” said Kinnari Patel, Pharm.D., MBA, President, Head of R&D and Chief Operating Officer, Rocket Pharma.
- The safety profile was highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, was well tolerated.
- 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.