Japan's Ministry of Health, Labour and Welfare has granted orphan drug designation to QRX003 for treating Netherton Syndrome, Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) announced. The designation provides development incentives including up to 10 years of market exclusivity upon approval.

The ministry grants orphan drug designation to medicines for rare diseases affecting fewer than 50,000 patients in Japan with high unmet medical need. Benefits include prioritized consultation, reduced fees, tax incentives, and priority review of applications.

QRX003 has received multiple regulatory recognitions globally, including orphan drug designation, pediatric rare disease designation, and fast track designation from the U.S. Food and Drug Administration, plus orphan drug designation from the European Medicines Agency. The company has also filed for breakthrough medicine designation with the Saudi Food and Drug Authority.

"Japan is a strategically important market for QRX003, and, along with the US and Western Europe, is one of three core territories in which we plan to self-commercialize QRX003 and our other pipeline products, once approved," said CEO Michael Myers.

QRX003 lotion (4%) is currently in Phase 2 clinical trials for Netherton Syndrome patients. The company expects to initiate a pivotal Phase 3 study in the second half of 2026, with potential new drug application filing in 2027.

Netherton Syndrome is a rare inherited skin disorder caused by mutations in the SPINK5 gene. No FDA-approved therapies currently exist for the condition, with treatment limited to supportive care and off-label therapies.