FDA Gives MeiraGTx’s Achromatopsia Therapy Fast-Track Status

The FDA has granted MeiraGTx’s investigational gene therapy, AAV-CNGA3, Fast-Track designation for treating achromatopsia — an inherited retinal disease that causes severe vision impairment, sensitivity to light and other sight problems — caused by CNGA3 gene mutations.

The gene therapy is designed to treat the disease by restoring cone-photoreceptor function. The drug is delivered to the cone receptors at the back of the eye by subretinal injection.

MeiraGTx and Janssen Pharmaceuticals are collaborating on the development of AAV-CNGA3 as part of a larger partnership to develop and commercialize gene therapies for inherited retinal diseases. MeiraGTx is currently conducting a phase 1/2 trial of the candidate.