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ProQR's eye drug wins US FDA's rare pediatric disease tag

ProQR Therapeutics NV's drug sepofarsen was granted a rare pediatric disease designation by the U.S. Food and Drug Administration for the treatment of an inherited eye disease that causes blindness in children.

The Dutch biopharmaceutical company is evaluating sepofarsen, or QR-110, to treat Leber's congenital amaurosis 10, a genetic eye disorder that appears in infancy and is the most common cause of blindness due to genetic disease in children. People with this disorder typically have severe visual impairment. The disorder affects about two to three per 100,000 newborns.

The rare pediatric disease tag is granted by the FDA to encourage the development of treatments for rare pediatric diseases. Under the program, the company may qualify for a voucher that can be redeemed to receive a priority review by the regulator for a future marketing application for a different product.

An interim analysis of sepofarsen's early-stage clinical study showed rapid and sustained improvement in vision in patients. The company is currently recruiting patients for a phase 2/3 trial called Illuminate.

Sepofarsen, which is administered through intravitreal injections in the eye, has previously been granted orphan drug designation in the U.S. and Europe and fast-track designation from the FDA. The therapy also received access to the PRIME scheme by the European Medicines Agency, which aids and speeds the review process for medicines that may provide a therapeutic advantage over existing drugs.