Quoin Pharmaceuticals Receives FDA RPD Designation for QRX003

Quoin Pharmaceuticals' lead asset, QRX003, has been granted Rare Pediatric Disease designation by the FDA for the treatment of Netherton Syndrome, reinforcing its potential as a therapeutic candidate for a profoundly underserved pediatric population. This follows earlier recognition by the European Medicines Agency, which granted Orphan Drug Designation to QRX003 in May 2025.

ASHBURN, Va., June 19, 2025 — Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) has received a significant boost for its lead asset, QRX003, with the U.S. Food and Drug Administration (FDA) granting Rare Pediatric Disease (RPD) Designation for the treatment of Netherton Syndrome. This designation underscores the potential of QRX003 as a therapeutic candidate for a profoundly underserved pediatric population. The designation follows earlier recognition by the European Medicines Agency (EMA), which granted Orphan Drug Designation to QRX003 in May 2025 [1].

Netherton Syndrome is a rare genetic disorder affecting approximately 1 in 200,000 newborns worldwide. Symptoms typically present at birth or in early infancy and persist throughout life, often requiring prolonged hospitalization due to severe skin inflammation, dehydration, and increased risk of infection. The condition is challenging to diagnose early and has a mortality rate estimated at 10 to 20 percent in infancy, highlighting the urgent need for effective treatments [1].

Dr. Michael Myers, Chief Executive Officer of Quoin Pharmaceuticals, expressed his optimism about the designation. "We are very pleased to announce the receipt of Rare Pediatric Disease Designation for QRX003 for Netherton Syndrome, a severe and underserved genetic disease," he said. "The Quoin team is fully focused on completing our pivotal clinical studies and advancing QRX003 towards a New Drug Application as the first potential treatment for this terrible disease. We are highly encouraged by the promising efficacy data and clean safety profile seen to date."

The FDA’s Rare Pediatric Disease Designation program is designed to encourage the development of new therapies for serious and life-threatening diseases that primarily affect individuals under 18 years of age. If a New Drug Application (NDA) for QRX003 is approved, Quoin may be eligible to receive a Priority Review Voucher (PRV), which can be redeemed to receive priority review for another marketing application or may be sold or transferred [1].

Quoin Pharmaceuticals is a late clinical-stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases. Their innovative pipeline comprises four products in development that collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa, and others [1].

Quoin Pharmaceuticals cautions that statements in this press release that are not a description of historical facts are forward-looking statements. Actual results may differ materially from those expressed or implied by such forward-looking statements due to various risks and uncertainties, including the Company’s ability to pursue its regulatory strategy, complete clinical trials on time, and achieve desired results and benefits as expected [1].

References:
[1] https://www.biospace.com/press-releases/quoin-pharmaceuticals-announces-fda-grants-rare-pediatric-disease-designation-for-qrx003-in-netherton-syndrome