Quoin Pharmaceuticals announced that the FDA has granted Orphan Drug Designation to QRX003 for the treatment of Netherton Syndrome, a rare and potentially life-threatening genetic skin disorder. The designation provides Quoin with development incentives, including potential tax credits, user fee exemptions, and seven years of market exclusivity upon approval.
QRX003 is a once-daily topical lotion formulated with a broad-spectrum serine protease inhibitor utilizing proprietary Invisicare delivery technology. It is designed to mimic the function of LEKTI, a protein missing in patients with Netherton Syndrome, which plays a critical role in regulating skin shedding and maintaining barrier integrity. By restoring balance to overactive kallikrein enzymes, QRX003 aims to strengthen the skin barrier, reduce inflammation, and improve overall skin health.
Clinical data from ongoing open-label studies have demonstrated clinically meaningful improvements in skin condition, itch reduction, and patient quality of life, with no significant safety issues observed. However, results also showed that discontinuation of treatment led to a reversion of symptoms within four weeks, underscoring the chronic nature of the disease and the need for consistent therapy.
If approved, QRX003 could become the first FDA-approved treatment for Netherton Syndrome. Quoin’s Chief Executive Officer, Dr. Michael Myers, stated that the designation marks an important milestone in advancing a long-awaited therapy for an underserved patient population and reaffirms the company’s commitment to rare dermatologic diseases.
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