A key regulatory achievement has been secured by Quoin Pharmaceuticals , a late clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, with the announcement that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead product candidate, QRX003, intended for the treatment of Netherton Syndrome. , a late clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, with the announcement that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead product candidate, QRX003, intended for the treatment of Netherton Syndrome.

This follows a similar ODD previously granted by the European Medicines Agency (EMA) in May 2025, cementing the product's status as a therapy for a disease affecting fewer than 200,000 people in the United States.

The FDA's designation provides Quoin with critical benefits, including tax credits for qualified clinical testing, fee waivers, and, most significantly, a guarantee of seven years of market exclusivity for QRX003 in the U.S. upon approval of its New Drug Application (NDA). Quoin's CEO, Dr. Michael Myers, highlighted that this designation is a vital milestone in the company's urgent mission to deliver QRX003 to patients, as it helps facilitate the pathway to approval and provides significant data protection.

The product, QRX003 lotion (4%), is currently being evaluated in two late-stage whole body pivotal clinical trials, with the company anticipating completion of enrollment in the first quarter of 2026, top-line data in the second half of 2026, and NDA submission planned for later that same year, positioning QRX003 to potentially become the first approved treatment for Netherton Syndrome.