MeiraGTx Holdings plc announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV-GAD. This designation is for the treatment of Parkinson’s disease in patients not adequately controlled with anti-Parkinsonian medications.
The RMAT designation is based on positive data from three clinical studies, including a Phase 1 dose-escalating study, a double-blind, sham-controlled Phase 2 study, and a second randomized, double-blind, sham-controlled clinical bridging study. These studies demonstrated the potential benefit of AAV-GAD as a one-time treatment administered via stereotactic infusion.
RMAT designation includes the benefits of Fast Track and Breakthrough Therapy designations, offering rolling review and potential Priority Review of a Biologics License Application (BLA). It also allows for increased interaction with the FDA and comprehensive discussions to expedite the development and manufacturing strategy for this program, which addresses a serious neurodegenerative disorder affecting millions worldwide.
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