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AGTC granted FDA orphan drug designation for gene therapy to treat achromatopsia

Achromatopsia is an inherited retinal disease caused by mutations in the CNGA3 gene.
AGTC granted FDA orphan drug designation for gene therapy to treat achromatopsia
Products receiving orphan drug designation are eligible to receive market exclusivity for a period of seven years

Applied Genetic Technologies (NASDAQ:AGTC), a biotechnology company focused on rare eye diseases, said the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation for its gene therapy product candidate for the treatment of achromatopsia, an inherited retinal disease, caused by mutations in the CNGA3 gene.

AGTC is developing products for achromatopsia based on mutations in the CNGA3 and CNGB3 genes, which together account for approximately 75% of the total achromatopsia patient population.

The Alachua, Florida-based company previously announced that the European Medicines Agency (EMA) granted orphan designation for the same indication in October.

"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," said Sue Washer, CEO of AGTC.

AGTC presented early preclinical findings in a naturally occurring dog model of the CNGB3 form of the disease, and previously received orphan drug designation from the FDA and the EMA for its investigational gene therapy product for the treatment of an additional variant of achromatopsia caused by mutations in the CNGB3 gene.

Orphan drug designation, covered by the U.S. Orphan Drug Act of 1983, is granted to drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 individuals.

Products receiving orphan drug designation are eligible to receive market exclusivity for a period of seven years, an accelerated regulatory review process, an exemption from certain taxes and user fees and additional clinical support from FDA.

 

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