London and New York based gene therapy company, MeiraGTx declared that the European Medicines Agency has extended Priority Medicines (PRIME) designation to MeiraGTx’s gene therapy product candidate A002 for the treatment of patients with achromatopsia (ACHM) due to mutations in the CNGB3 gene.
Achromatopsia is a chronically debilitating inherited eye disorder that severely limits a person’s sight. Patients are legally blind from birth with very poor visual acuity as well as extreme light sensitivity, involuntary eye movement, and complete color blindness. The PRIME application was based on non-clinical in vivo data and early clinical safety data from MeiraGTx’s ongoing Phase I/II dose escalation study.
Zandy Forbes, Ph.D., President and CEO of MeiraGTx said “Having just received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) earlier this month, we are very pleased with the momentum of our ACHM program and the recognition by regulatory agencies of its potential to help those in need of effective treatment options.”
A002 is an adeno-associated virus (AAV) investigational gene therapy designed to deliver a codon-optimized CNGB3 cDNA under the control of the cone arrestin (CAR) promoter to photoreceptors in the back of the eye. A002 is delivered via a sub-retinal injection to cover the central region of the retina, including the fovea where the majority of cones are located. MeiraGTx has completed dosing of eight CNGB3 patients in the Phase I/II study and is currently treating patients in the third dose escalation cohort.