HG004, a gene replacement therapy, received rare pediatric disease designation from the FDA for the treatment of inherited retinal diseases caused by RPE65 mutations, according to a press release from HuidaGene.
Rare pediatric disease designation is granted to incentivize “development of new treatments for serious or life-threatening diseases that primarily affect children ages 18 years or younger with fewer than 200,000 people affected in the U.S.,” the release said.
The designation allows a company to qualify for a priority review voucher, which can expedite a 6-month priority