Quoin Pharmaceuticals Applies for Orphan Drug Designation for QRX003
Quoin Pharmaceuticals 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. If granted, QRX003 will have ODD status in Quoin's three core commercial territories of the US, EU and Japan.
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Quoin Pharmaceuticals Files for Breakthrough Designation for QRX003, Potential First Treatment for Netherton Syndrome
- Breakthrough Application: Quoin Pharmaceuticals has filed for Breakthrough Medicine Designation for QRX003 with the Saudi Food and Drug Authority, which, if granted, will expedite the review process and potentially allow early patient access by the second half of 2026, marking a historic milestone in the treatment of Netherton Syndrome.
- Clinical Trial Progress: QRX003 is currently undergoing two pivotal whole-body clinical trials, with enrollment expected to complete in the first half of 2026 and top-line data anticipated in the second half, providing critical evidence for subsequent drug applications and facilitating market entry.
- Significant Market Potential: If approved, QRX003 will be the first-ever treatment for Netherton Syndrome globally, and Quoin's distribution partnership with Genpharm will ensure its promotion in Saudi Arabia and other MENA countries, addressing the urgent need for rare disease therapies in the region.
- Regulatory Support Strengthened: QRX003 has received Orphan Drug and Pediatric Rare Disease Designations from the U.S. FDA and Orphan Drug Designation from the European Medicines Agency, indicating strong regulatory recognition worldwide and enhancing Quoin's competitive position in the rare disease market.
Quoin Pharmaceuticals Stock Rises 21% Following Topical Rapamycin Achievement
Stock Performance: Quoin Pharmaceuticals Ltd. (QNRX) saw a 21.15% increase in stock price, reaching $9.99, following the announcement of successful rapamycin loadings in their topical delivery platforms.
Clinical Development Plans: The company intends to manufacture clinical trial and stability batches in Q4 2025 and start clinical testing in the first half of 2026 for conditions like microcystic lymphatic malformations and venous malformations, which currently have no FDA-approved treatments.
Trading Activity: The announcement led to unusually high trading volume for QNRX, indicating strong investor interest in the company's developments.
Stock Range: The stock's 52-week price range is noted to be between $2.65 and $9.99.
