Quoin Pharmaceuticals Ltd. announced that following a successful meeting with the Japanese MHLW that it has submitted an application for Orphan Drug Designation (ODD) for its lead product candidate, QRX003, for the treatment of Netherton Syndrome. At the meeting, the MHLW confirmed that QRX003 qualifies for both ODD and Fast Track regulatory review in Japan. Quoin remains steadfastly committed to completing the clinical development of QRX003 with a high degree of urgency on behalf of patients and families living with this devastating disease." QRX003 lotion (4%) is being evaluated in two late-stage whole body pivotal clinical trials for Netherton Syndrome.
Enrollment in both pivotal studies is expected to be completed in 1H 2026, top-line data is anticipated in the second half of 2026, and NDA submission is planned later in the year or early 2027. In 2025, QRX003 was granted Orphan Drug Designation for the treatment of Netherton syndrome by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
