MeiraGTx announces FDA granted RMAT designation to AAV-GAD

MeiraGTx (MGTX) Holdings announced that the U.S. Food and Drug Administration, FDA, has granted Regenerative Medicine Advanced Therapy, RMAT, designation to AAV-GAD for the treatment of Parkinson’s disease not adequately controlled with anti-Parkinsonian medications. This RMAT was awarded following the presentation to the FDA of positive data from 3 clinical studies demonstrating the benefit of AAV-GAD when administered in a one-time stereotactic infusion to the subthalamic nucleus in the brain. A Phase 1 dose escalating clinical study was conducted, followed by a double-blind, sham-controlled Phase 2 study and a second randomized, double-blind, sham-controlled dose ranging clinical bridging study.