FDA Grants Orphan Drug Designation to AGTC for Achromatopsia Gene Therapy

The FDA has awarded Applied Genetic Technologies orphan drug designation for a candidate intended to treat achromatopsia caused by mutations in the CNGA3 gene.

Evidence in animal models suggests the therapy — administered as a one-time injection — has the potential to provide long-lasting vision improvement in patients affected by achromatopsia, an inherited retinal disease.

Recent studies of sheep affected by achromatopsia caused by mutations in the CNGA3 gene showed that delivery of an adeno-associated virus vector with a normal copy of CNGA3 restored their vision, as measured by their ability to navigate an obstacle maze.

The company also received orphan drug designation from the European Medicines Agency in October for the same indication.