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 Orphan Drug Designation (ODD) to its lead product candidate, QRX003, for the treatment of Netherton Syndrome. The designation follows previously granted Orphan Drug Designation by the European Medicines Agency (EMA) in May 2025.
The FDA’s Orphan Drug Designation program provides orphan status to therapies intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. This designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved. QRX003 is on track to potentially become the first approved treatment for Netherton Syndrome.
“Receiving Orphan Drug Designation from the FDA is yet another important milestone in our mission to bring QRX003 to patients suffering with Netherton Syndrome,” said Dr. Michael Myers, CEO of Quoin Pharmaceuticals. “Together with the EMA designation granted earlier in the year, this latest recognition by the FDA could potentially help facilitate the pathway of QRX003 to approval in the US whilst providing significant data protection to the product, if approved. 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 Q1 2026, top-line data is anticipated in the second half of 2026, and NDA submission is planned later in the year.
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