FDA Grants RMAT Designation for VY-AADC for Parkinson Disease

In the phase 1b trial, one-time administrations of VY-AADC demonstrated robust and durable improvements in patients’ motor function.

Voyager Therapeutics announced that the US Food and Drug Administration (FDA) granted regenerative medicine advanced therapy (RMAT) designation for VY-AADC gene therapy for treatment of Parkinson’s disease in patients with motor fluctuations 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,” Robert Pietrusko, PharmD, senior vice president, regulatory affairs and quality assurance, Voyager, said in a statement. “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.”

The designation was granted based on clinical data from an ongoing phase 1b trial in patients with Parkinson’s disease.

In the trial, 1-time administrations of VY-AADC demonstrated robust and durable improvements in patient’s motor function, in addition to substantial reductions in use of daily oral levodopa and other Parkinson’s disease medications.

Infusions of the medication has been well-tolerated, with no vector-related serious adverse effects yet reported.

Comprised of the adeno-associated virus-2 capsid and a cytomegalovirus promoter to drive AADC transgene expression, VY-AADC is designed to deliver the AADC gene directly into neurons of the putamen where dopamine receptors are located, enabling the neurons of the putamen to express the AADC enzyme to convert levodopa into dopamine.

This approach has the potential to durably enhance the conversion of levodopa to dopamine, providing clinically meaningful improvements by restoring motor function and improving symptoms after a single administration.

Currently, there are no therapies that effectively slow or reverse the progression of the disease.