Quoin Pharmaceuticals Submits QRX003 For Orphan Drug Designation To Japan’s MHLW To Treat Rare Netherton Syndrome

Quoin Pharmaceuticals Ltd., a late clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, announced that it has submitted an application for Orphan Drug Designation (ODD) in Japan for its lead product candidate, QRX003, aimed at treating Netherton Syndrome. This follows a successful meeting with the Japanese Ministry of Health, Labour and Welfare (MHLW), where it was confirmed that QRX003 qualifies for both Orphan Drug Designation and Fast Track regulatory review in Japan. If approved, QRX003 would hold ODD status across Quoin’s three core commercial markets: the United States, Europe, and Japan.

Japan’s Orphan Drug Designation program grants special status to therapies intended for rare diseases affecting fewer than 50,000 people in the country. Benefits of the designation include research and development subsidies, tax credits for qualified clinical testing, reduced MHLW application fees, priority regulatory review, and ten years of market exclusivity once approved. QRX003 is on track to potentially become the first approved treatment for Netherton Syndrome, addressing a significant unmet medical need.

“Following a productive meeting with MHLW, we are optimistic that QRX003 will be granted Orphan Drug Designation in Japan,” said Dr. Michael Myers, CEO of Quoin Pharmaceuticals. “We are also encouraged that QRX003 qualifies for Fast Track review. Our plans to establish a commercial presence in Japan, one of our three key markets, remain on track. Quoin is fully committed to advancing the development of QRX003 as quickly as possible to benefit patients and families affected by this rare and challenging disease.”

QRX003 lotion (4%) is currently being evaluated in two late-stage whole-body pivotal clinical trials for Netherton Syndrome. Enrollment in these studies is expected to be completed in the first half of 2026, with top-line results anticipated in the second half of the year. Quoin plans to submit a New Drug Application (NDA) for QRX003 later in 2026 or early 2027. The product has already received Orphan Drug Designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in 2025, reinforcing its potential to become a first-in-class therapy for this rare condition.