HuidaGene Therapeutic announced the U.S. Food and Drug Administration (FDA) had granted Rare Pediatric Disease Designation (RPDD) to HG004 for the treatment of inherited retinal disease caused by RPE65 mutations(RPE65-IRDs).
The FDA grants RPDD 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.
PRVs have historically commanded prices in excess of $100 million USD with the highest price paid for a PRV was $350 million USD.
For a large company launching a billion-dollar drug, the six-month acceleration in regulatory review can be of substantial economic value. Most recently, Sarepta Therapeutics sold a PRV for $102 million USD.
“This Rare Pediatric Disease designation from the US FDA highlights the significant unmet medical need that HuidaGene is seeking to address with HG004 for RPE65-IRDs,” said Alvin Luk, PhD, MBA, CCRA, co-founder and CEO of HuidaGene. “HG004 was granted both RPDD and orphan drug designation (ODD) by the US FDA in March, allowing tax credits for clinical development, exemptions for certain FDA application fees, 7-year of market exclusivity, and assistance in the drug development process. We look forward to advancing HG004 in the clinic and remain steadfast in our commitment to answering patients worldwide.”
“We are pleased to have received another significant regulatory feedback from the US FDA underscoring the dire need for a treatment option of this devastating inherited blindness,” said Linyu Shi, PhD, co-founder and CSO of HuidaGene. “Data from our preclinical studies have shown that HG004 demonstrates significant superiority than AAV2-hRPE65 (LUXTURNA like) in the recovery of retinal function of the Rpe65-/- mice. We are committed to delivering transformative genetic medicines for rare genetic diseases globally.”
About 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.