MeiraGTx Holdings PLC said the U.S. Food and Drug Administration has granted orphan drug designation to its gene therapy AAV-CNGA3.
The gene therapy is a potential treatment of achromatopsia, a hereditary visual disorder that causes absence of color vision, decreased vision, light sensitivity and involuntarily movement of the eyes. The therapy will receive seven-year market exclusivity if approved with an orphan drug designation, the company said Aug. 13. Orphan designation facilitates and expedites the development of drugs for rare diseases.
MeiraGTx has previously received a positive recommendation for orphan drug status from the European Medicines Agency for the therapy.
New York-based MeiraGTx is a clinical-stage gene therapy company with four ongoing clinical programs and a broad pipeline of preclinical and research programs for the treatment of a range of inherited and acquired disorders.