Netherton syndrome is a rare autosomal recessive genodermatosis caused by SPINK5 mutations, characterized by congenital ichthyosis, hair-shaft defects, and severe atopic manifestations.
Japan's MHLW granted Orphan Drug Designation to three investigational therapies: gildeuretinol for Stargardt disease, QRX003 for Netherton Syndrome, and E2086 for narcolepsy, each offering potential 10-year market exclusivity.
The FDA granted Fast Track Designation to Quoin's QRX003 for Netherton Syndrome and supported NRx's NRX-100 NDA path under Fast Track for treatment-resistant depression, addressing serious unmet needs.
Quoin Pharmaceuticals has secured Orphan Drug Designations for QRX003 in the U.S. and Europe for Netherton Syndrome, while advancing regulatory pathways in Japan and Saudi Arabia. The company reported clinical progress including expansion of pediatric studies and potential for a Priority Review Voucher worth up to $200 million.
The FDA has granted fast track designation to QRX003 for Netherton Syndrome and IBI3003 for relapsed or refractory multiple myeloma, while also accepting regulatory applications for other therapies addressing serious conditions with unmet medical needs.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07538583 |
Phase 2/3 Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome |
RECRUITING | PHASE2/PHASE3 |
| NCT07280091 |
Study of Skin and Gut Microbiome in a Skin Condition Involving Skin Barrier Impairment and Allergic Symptoms: Netherton Syndrome |
RECRUITING | |
| NCT06953466 |
Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome |
RECRUITING | PHASE2/PHASE3 |
| NCT06539507 |
A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725 |
RECRUITING | PHASE1 |
| NCT06137157 |
Evaluation of Topical ATR12-351 in Adults With Netherton Syndrome |
COMPLETED | PHASE1 |
| NCT05979831 |
A Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in Patients With Netherton Syndrome |
TERMINATED | PHASE1/PHASE2 |
| NCT05902663 |
Natural History of Netherton Syndrome |
TERMINATED | |
| NCT05856526 |
A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome |
TERMINATED | PHASE2/PHASE3 |
| NCT05789056 |
Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome |
RECRUITING | PHASE2/PHASE3 |
| NCT05583669 |
A Study to Assess the Safety and Pharmacokinetics of Multiple Ascending Subcutaneous Doses of DS-2325a in Healthy Subjects |
COMPLETED | PHASE1 |
