FDA grants fast track designation to AAV-CNGA3 gene therapy for achromatopsia

The U.S. Food and Drug Administration has granted fast track designation to AAV-CNGA3 (MeiraGTx Holdings Plc) gene therapy for the treatment of achromatopsia (ACHM) caused by mutations in the CNGA3 gene, according to a press release.

AAV-CNGA3 is designed to restore cone function, delivered to the cone receptors at the back of the eye via subretinal injection.

AAV-CNGA3 is currently being evaluated in an open-label, dose-scalation phase 1/2 clinical trial in patients with ACHM due to mutations in the CNGA3 gene.

“We are very pleased to have received fast track designation for AAV-CNGA3 and that the FDA has recognized a significant need exists to quickly advance new therapies for those with ACHM,” said Alexandria Forbes, PhD, president and chief executive officer of MeiraGTx in the press release. “ACHM is a serious and debilitating disease and we look forward to communicating closely with the FDA as we continue the clinical advancement of AAV-CNGA3.”

Head the full press release here.