Rare Daily Staff

The U.S. Food and Drug Administration has granted ProQR Therapeutics Rare Pediatric Disease designation for sepofarsen, its experimental treatment of Leber’s congenital amaurosis 10, the most common cause of blindness in children due to genetic disease.

Leber’s congenital amaurosis (LCA) consists of a group of diseases of which LCA10 is the most frequent and one of the most severe forms. LCA10 is caused by mutations in the CEP290 gene, of which the p.Cys998X mutation has the highest prevalence. LCA10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To date, there are no treatments approved or other products in clinical development that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA10 because of this mutation.

Sepofarsen (QR-110) is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of LCA10 due to the p.Cys998X mutation in the CEP290 gene.

The Rare Pediatric Disease designation provides priority review by the FDA to encourage treatments for rare pediatric diseases. Under the program, a sponsor who receives an approval for a drug or biologic for a rare pediatric disease may qualify for a voucher that can be redeemed to receive a priority review by the FDA of a subsequent marketing application for a different product. The vouchers are potentially lucrative because they can be sold. In August, Swedish Orphan Biovitrum sold its Priority Review Voucher to AstraZeneca for $95 million.

“This designation for sepofarsen underscores the significant unmet medical need for patients with this genetic disease-causing blindness,” said Daniel de Boer, CEO of ProQR. “Our goal is to advance a pipeline of programs that can treat inherited retinal diseases like LCA10 to bring medicines to patients as soon as possible.”

Photo: Daniel de Boer, CEO of ProQR

X