RMAT and fast track status for Rocket's Fanconi anemia therapy

US gene therapy company Rocket Pharmaceuticals (Nasdaq: RCKT) yesterday announced that the US Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) and fast track designations to RP-L102, the company’s lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi anemia (FA).

RMAT designation was granted based on the positive efficacy and safety results from the ongoing Phase I/II clinical trial of RP-L102 being conducted in Europe. The news lifted Rocket’s share 1.18% to $16.35 by close of trading Tuesday.

"The RMAT designation, which is the CBER equivalent of Breakthrough designation with the same benefits, and fast track designations represent important milestones for the FA program and recognition of RP-L102 as a potential treatment for this devastating disease,” said Dr Gaurav Shah, chief executive and president of Rocket. “Achieving these milestones further underscores the urgent need to develop new treatments for young children affected by FA that would otherwise lead to bone marrow failure and death in the absence of a hematopoietic stem cell transplantation. We look forward to working closely with the FDA on our development plan to rapidly advance RP-L102 towards potential approval,” he added.