The FDA granted orphan drug designation for HG004, a gene replacement therapy for the treatment of inherited retinal diseases caused by RPE65 mutations, HuidaGene Therapeutics announced in a press release.
The designation offers the company “the potential for certain benefits, including tax credits for clinical development, exemptions for certain FDA application fees, 7 years of post-approval market exclusivity, and assistance in the drug development process,” the release said.
In addition, drug development and registration action in the U.S. will be accelerated, with HG004 eligible