Voyager Therapeutics announces FDA RMAT designation granted for VY-AADC
Voyager Therapeutics announced that the U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation for Voyager's VY-AADC gene therapy treatment for Parkinson's disease in patients with motor fluctuations that are refractory to medical management. Recently established under the 21st Century Cures Act, RMAT designation is an expedited program for the advancement and approval of regenerative medicine products, including gene therapy products. The designation includes all the benefits of the FDA's Fast Track and Breakthrough Therapy designation programs, with the ability for early interactions with the agency to discuss any surrogate or intermediate endpoints to support the potential acceleration of approval and satisfy post-approval requirements. An investigational drug or therapy is eligible for RMAT designation if it meets the definition of a "regenerative medicine therapy" as defined in the 21st Century Cures Act, it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the therapy has the potential to address unmet medical needs for such disease or condition. RMAT designation for VY-AADC was granted based on clinical data from the ongoing Phase 1b trial with VY-AADC in patients with Parkinson's disease. During this trial, one-time administrations of VY-AADC demonstrated robust and durable improvements in patients' motor function along with substantial reductions in use of daily oral levodopa and other Parkinson's disease medications. Infusions of VY-AADC have been well-tolerated in this trial with no vector-related serious adverse events reported to date.