Sepofarsen has received rare pediatric disease designation from the FDA for the treatment of Leber’s congenital amaurosis 10, according to a press release from ProQR Therapeutics.
Sepofarsen, an RNA-based oligonucleotide, is designed to address the underlying cause of LCA10 in the CEP290 gene.
With the designation, the drug will receive priority review by the FDA, and it could qualify ProQR for a voucher redeemable for priority FDA review of a subsequent marketing application for a different product, the release said.
“This designation for sepofarsen underscores the significant unmet medical need for patients with this genetic disease causing blindness. 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,” ProQR CEO Daniel de Boer said in the release.
A previous press release from the company detailed 12-month topline results of a phase 1/2 trial, which showed the target registration dose, 80 µg with a 160 µg loading dose, was associated with statistically significant, clinically meaningful vision improvement as well as a favorable benefit-risk profile. In addition, 6-month dosing frequency was associated with durable vision improvements.
At 12 months, the target registration dose had an equal to or greater response than the response reported in the 3-month interim analysis. Patients with baseline vision better than light perception were more likely to respond to sepofarsen treatment.
Eleven study subjects received between one and four doses of sepofarsen. Eight cataracts were observed, three in the target registration dose cohort and five in a high-dose cohort. Two cases of mild cystoid macular edema and two cases of subclinical retinal thinning occurred in three subjects in the now-retired 320 µg/160 µg dose group.
“We are very pleased with the data reported from the phase 1/2 study, in which LCA10 patients treated with sepofarsen experienced a rapid and durable improvement in vision. The topline data from this study strengthen our confidence in the design of the ongoing phase 2/3 trial, which could be the sole registration trial for the sepofarsen program,” David Rodman, MD, executive vice president of research and development at ProQR, said in the release.