For many drug developers, Parkinson’s disease is a tricky disease to treat. However, developing a treatment ultimately could yield a huge reward as there are currently no therapies that effectively slow or reverse the progression of this disease. And after Thursday’s announcement, Voyager Therapeutics Inc. (NASDAQ: VYGR) could be poised to reap this reward.
Voyager shares made a solid gain early on Thursday after the company announced a key U.S. Food and Drug Administration (FDA) approval related to its Parkinson’s treatment. Specifically, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) 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.
Robert Pietrusko, Pharm.D., senior vice president of regulatory affairs and quality assurance at Voyager, commented:
The RMAT designation was based on our Phase 1b clinical data with VY-AADC and represents an important milestone for the program and recognition of this gene therapy as a potential treatment for Parkinson’s disease. We look forward to working closely with the agency through the benefits of the RMAT designation for guidance on the development of VY-AADC including advice to generate evidence needed to support its potential approval in an efficient manner.
Shares of Voyager closed Wednesday at $21.17, with a consensus analyst price target of $31.55 and a 52-week range of $8.10 to $31.91. Following the announcement, the stock was up about 15% at $24.24 in early trading indications Thursday.