21 June 2018 - Voyager Therapeutics today announced that the U.S. FDA 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.
“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,” said Robert Pietrusko, Pharm.D., senior vice president of regulatory affairs and quality assurance at Voyager. “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.”