Achromatopsia treatment wins orphan status

The U.S. FDA has granted orphan drug designation to AGTC’s gene therapy product candidate for the treatment of achromatopsia caused by mutations in the CNGA3 gene.

“Receiving U.S. orphan drug designation is another significant milestone as we continue to advance our pipeline of novel gene therapies to treat rare inherited eye disorders,” Sue Washer, president and CEO of AGTC, said in the release.

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