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ProQR's genetic vision loss drug gets US FDA fast-track status

ProQR Therapeutics NV said the U.S. Food and Drug Administration granted the fast-track designation to its investigational drug QR-1123 to treat a rare genetic disorder that causes progressive vision loss.

The Dutch biopharmaceutical company is developing QR-1123 to counter the underlying causes of autosomal dominant retinitis pigmentosa, or adRP, caused by the P23H mutation in the rhodopsin gene. The disorder starts off as night blindness during childhood and can end in complete blindness by the time one is an adult.

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It is estimated that the P23H-altered adRP affects about 2,500 people in the U.S.

"There are no current treatment options available for patients to improve vision or prevent vision loss due to adRP. We look forward to beginning enrollment in phase 1/2 (Aurora) clinical trial for QR-1123 in the coming months," ProQR CEO Daniel de Boer said in a statement.

The U.S. regulator grants fast-track designations to expedite the review of drugs that treat serious conditions and fill unmet medical needs. Incentives include frequent meetings and communications with the FDA and potential accelerated approval, priority review or rolling review.

The Dutch company acquired the exclusive worldwide license to QR-1123 from Ionis Pharmaceuticals Inc. in 2018 and recently secured approval to study it in humans.