FDA Grants Rare Pediatric Disease Designation for Huidagene’s Therapy to Treat Inherited Blindness

Rare Daily Staff

The U.S. Food and Drug Administration had granted Rare Pediatric Disease designation to Huidagene Therapeutics’ HG004 for the treatment of inherited retinal disease caused by RPE65 mutations.

Inherited retinal disease (IRD) is a group of rare blinding conditions caused by mutations in any 1 of more than 250 genes. Leber’s congenital amaurosis (LCA), severe early childhood-onset retinal dystrophy (SECORD), early-onset severe retinal dystrophy (EOSRD), and retinitis pigmentosa (RP), which may all be grouped under the heading of inherited retinal disease caused by RPE65 mutations (RPE65-IRD), are considered to represent a phenotypic continuum of the same disease. The RPE65-IRD with a typical onset between birth and five years of age exhibits several common clinical findings, chiefly night blindness, progressive loss of visual fields, and loss of central vision. Given the often severe and early visual loss associated with RPE65-IRD, other areas of development, including speech, social skills, and behavior, may also be delayed.

HG004 is a CRISPR-based therapeutic in development for the one-time, direct-to-RPE treatment of inherited retinal disease caused by mutations in the RPE65 gene. Huidagene says data from preclinical studies have shown that HG004 demonstrates significant superiority compared to AAV2-hRPE65 (similar to Luxturna) in the recovery of retinal function of the Rpe65-/- mice.

“This Rare Pediatric Disease designation from the U.S. FDA highlights the significant unmet medical need that HuidaGene is seeking to address with HG004 for RPE65-IRDs,” said Alvin Luk, co-founder and CEO of HuidaGene.

The FDA grants Rare Pediatric Disease designation to incentive 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 RPDD program allows for a sponsor who receives an approval to qualify for a priority review voucher (PRV) that can be deemed to receive an expedited six-month priority review for any subsequent marketing application or may be sold or transferred. Most recently, Sarepta Therapeutics sold a PRV for $102 million.

Photo: Alvin Luk, co-founder and CEO of HuidaGene.