Quoin Pharmaceuticals Receives FDA Rare Paediatric Disease Designation for QRX003 in Netherton Syndrome
Wednesday, June 25, 2025
Quoin Pharmaceuticals Ltd, a late-stage clinical pharmaceutical company focused on rare and orphan conditions, has been granted Rare Paediatric Disease (RPD) Designation by the U.S. Food and Drug Administration (FDA) for its lead product candidate, QRX003, for the treatment of Netherton Syndrome.
This designation highlights the potential of QRX003 as a treatment option for a severely underserved paediatric group. It follows prior recognition from the European Medicines Agency (EMA), which granted Orphan Drug Designation to the same therapy in May 2025.
The FDA’s RPD programme aims to support the development of treatments for serious, life-threatening conditions that primarily affect individuals under the age of 18. Should the New Drug Application (NDA) for QRX003 be approved, and if the programme is reauthorised, Quoin may qualify for a Priority Review Voucher. This voucher can either be used to fast-track another application or be sold or transferred.
Netherton Syndrome is a rare inherited disorder estimated to affect around one in 200,000 newborns globally. Symptoms usually appear at birth or during infancy and tend to continue throughout life. Newborns often require extended hospital care due to severe skin problems, dehydration, and a heightened risk of infection. Early diagnosis is often difficult, as the condition can mimic other skin or immune-related diseases. The mortality rate in infancy is estimated at 10 to 20 percent, reflecting the seriousness of the disease.
Quoin remains focused on progressing its pivotal clinical studies and preparing for regulatory submissions, aiming to make QRX003 the first approved treatment for this condition.
Source: globenewswire.com
