FDA grants rare pediatric disease designation to HG004 for inherited retinal diseases
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Key takeaways:
- HG004 received rare pediatric disease designation.
- The designation supports development of treatments for serious diseases that affect fewer than 200,000 children in the U.S.
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 review for any subsequent marketing application.
HG004 was granted orphan drug designation earlier this year.
“We are pleased to have received another significant regulatory feedback from the U.S. FDA underscoring the dire need for a treatment option of this devastating inherited blindness,” Linyu Shi, PhD, co-founder and chief scientific officer of HuidaGene, said in the release. “Data from our preclinical studies have shown that HG004 demonstrates significant superiority than AAV2-hRPE65 in the recovery of retinal function of the Rpe65-/- mice.”
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