Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (“Quoin” or the “Company”), a late clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the Company’s lead asset, QRX003, for the treatment of Netherton Syndrome.
The designation reinforces the potential of QRX003 as a therapeutic candidate for a profoundly underserved pediatric population. It follows earlier regulatory recognition by the European Medicines Agency (EMA), which granted Orphan Drug Designation to QRX003 in May 2025.
“We are very pleased to announce the receipt of Rare Pediatric Disease Designation for QRX003 for Netherton Syndrome, a severe and underserved genetic disease,” said Dr. Michael Myers, Chief Executive Officer of Quoin Pharmaceuticals. “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 intended 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, upon reauthorization of the program 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.
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