Quoin Pharmaceuticals Granted Orphan Drug Designation by EMA for QRX003 in Netherton Syndrome
Ashburn, VA — May 2025 — Quoin Pharmaceuticals Ltd., a late clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, has announced that its lead product, QRX003, has been granted Orphan Drug Designation (ODD) by the European Medicines Agency (EMA) for the treatment of Netherton syndrome.
The EMA's Orphan Drug Designation provides several key benefits:
• Scientific guidance on clinical study protocols
• Reductions in regulatory fees
• Access to European Union grants
• 10 years of market exclusivity in the EU upon product approval
“We continue to make important clinical and regulatory progress for QRX003 as a potential treatment for Netherton syndrome, and this milestone marks yet another significant step forward for Quoin,” said Dr. Michael Myers, CEO of Quoin Pharmaceuticals.
“As we continue to generate positive clinical results from our ongoing studies, we are very encouraged both by the efficacy signals we are seeing and the clean safety profile of the product to date. In particular, the extent of skin healing observed coupled with the almost complete elimination of severe chronic pruritus in our investigator paediatric study underscores QRX003’s mechanism of action as a broad-spectrum kallikrein inhibitor that has the potential to be a safe and effective treatment for this disease.”
• Mechanism: Topical, non-systemic, broad-spectrum kallikrein inhibitor
• Target Indication: Netherton syndrome, a rare and life-altering skin disorder
• Current Status: Undergoing evaluation in multiple late-stage clinical trials
• Significance: No currently approved treatments for Netherton syndrome exist
Netherton syndrome is a severe genetic skin disorder characterized by:
• Persistent skin inflammation
• Chronic pruritus (itching)
• High risk of infections
• Significant impact on quality of life, particularly in pediatric patients
Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company developing and commercializing innovative therapeutic products for rare and orphan diseases. The company's lead candidate, QRX003, represents a promising potential treatment for patients suffering from unmet medical needs such as Netherton syndrome.