Quoin Pharmaceuticals Wins Second Rare Pediatric Disease Designation for Lead Skin Therapy
核心洞察
The FDA granted Rare Pediatric Disease designation to Quoin Pharmaceuticals' topical candidate QRX003 for Peeling Skin Syndrome (搜索), an inherited disorder with no approved treatment.
It is the second such designation for QRX003, which previously received the same status for Netherton Syndrome (搜索) and Orphan Drug status from the European Medicines Agency.
Quoin plans to start a Phase 2 study before the end of 2026, enrolling up to 12 pediatric and adult patients in the U.S. and Europe under a cleared IND.
Quoin Pharmaceuticals announced on September 8, 2026 that the FDA has granted Rare Pediatric Disease designation to its lead candidate QRX003 for Peeling Skin Syndrome (搜索), a rare inherited disorder in which the skin's outer layer sheds excessively, causing significant pain and persistent itching. No treatment is currently approved for the condition. It is the second Rare Pediatric Disease designation for QRX003, following an earlier one for Netherton Syndrome (搜索).
The company said its IND for the program has been cleared and it expects to begin a Phase 2 study before the end of 2026, enrolling up to 12 pediatric and adult patients with Peeling Skin Syndrome (搜索) in the U.S. and Europe. Quoin described it as the first formal study ever conducted in the U.S. for the disease under an open IND. If QRX003 later wins full approval, the designation could make Quoin eligible for a Priority Review Voucher, a transferable credit that accelerates review of a future application and can be sold to another company.
QRX003 is a topical lotion that blocks kallikrein proteases (搜索), targeting the mechanism believed to underlie the damaged skin barrier in Netherton Syndrome (搜索). Quoin sees comparable underlying biology in Peeling Skin Syndrome (搜索), which is why it is pursuing the same drug across both conditions. The company's pipeline, built around QRX003 and a second platform asset, QRX009 (搜索), targets additional rare skin and vascular disorders including Palmoplantar Keratoderma (搜索), Pachyonychia Congenita (搜索), Gorlin Syndrome (搜索) and several types of vascular malformations.
Source: BioBuzz