Quoin Pharmaceuticals' QRX003 Receives EMA Orphan Drug Status for Netherton Syndrome

Quoin Pharmaceuticals Ltd., a specialty pharmaceutical company in its late clinical stages, is making significant strides in the treatment of rare and orphan diseases. Recently, the company announced a pivotal achievement: the European Medicines Agency (EMA) has granted Orphan Drug Designation to its leading product, QRX003, specifically for Netherton Syndrome. This recognition not only highlights the potential of QRX003 as a breakthrough treatment but also provides the company with several incentive benefits. These benefits include scientific advice on study protocols, fee reductions, and access to EU grants. Upon approval, QRX003 will enjoy a decade of market exclusivity in Europe for treating Netherton Syndrome.

Netherton Syndrome is a challenging and life-altering condition that currently lacks approved treatment options. QRX003 is being developed as a topical, non-systemic treatment, aiming to address this significant unmet medical need. The company's CEO, Dr. Michael Myers, emphasized the progress Quoin Pharmaceuticals is making with QRX003, noting the promising clinical and regulatory advancements achieved so far. Dr. Myers highlighted the encouraging results from ongoing studies, particularly pointing out the efficacy signals observed and the favorable safety profile QRX003 has demonstrated to date. The extent of skin healing and the near-complete elimination of severe chronic itching in pediatric patients underscore the product’s potential efficacy as a broad-spectrum kallikrein inhibitor, which may offer a safe and effective therapy for this debilitating disease.

QRX003 is currently undergoing evaluation in several late-stage clinical trials. The product is designed to provide a much-needed therapeutic option for individuals affected by Netherton Syndrome, a condition that significantly impacts the quality of life of patients and their families. Quoin Pharmaceuticals is steadfast in its mission to develop and commercialize therapeutic solutions for rare and orphan diseases, addressing the unmet medical needs of patients, their families, and healthcare providers.

Beyond QRX003, Quoin Pharmaceuticals' innovative pipeline includes multiple products targeting a diverse range of rare conditions. These conditions include Peeling Skin Syndrome, SAM Syndrome, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa, Microcystic Lymphatic Malformations, Venous Malformations, and Angiofibroma, among others. The company is committed to advancing its pipeline with the potential to make a significant impact on these rare diseases, offering hope and potential relief to patients worldwide.

This recent regulatory milestone in Europe marks a significant step forward for Quoin Pharmaceuticals as it continues to advance QRX003 in late-stage clinical trials. The promise of extended market exclusivity upon approval provides a strong incentive for the company to continue its efforts in bringing this novel treatment to patients in need.

In conclusion, Quoin Pharmaceuticals Ltd. is at the forefront of developing transformative therapies for rare and orphan diseases. The Orphan Drug Designation for QRX003 by the EMA is a testament to its potential as a viable treatment for Netherton Syndrome. With a strong commitment to addressing unmet medical needs and a robust pipeline targeting a wide array of rare conditions, Quoin Pharmaceuticals is poised to make significant contributions to the field of rare disease treatment.