USFDAJun 12, 2026, 06:36 AM
GSK's Momelotinib Gets US & EU Orphan Drug Status for VEXAS Syndrome
AI Summary
GSK announced that its drug momelotinib has been granted Orphan Drug Designation by both the US Food and Drug Administration and the European Medicines Agency for the treatment of VEXAS syndrome. VEXAS syndrome is a rare, life-threatening haemato-inflammatory condition for which there are currently no approved treatments. This designation supports the ongoing development efforts and regulatory evaluations for momelotinib, which is already approved for myelofibrosis. A phase II/III ATLAS trial evaluating momelotinib's efficacy and safety in VEXAS syndrome is underway, with its design to be presented at the 2026 European Hematology Association Congress.
Key Highlights
- GSK's momelotinib received Orphan Drug Designation from US FDA and EU EMA for VEXAS syndrome.
- VEXAS syndrome is a rare, life-threatening haemato-inflammatory condition with no approved treatments.
- The syndrome has a poor prognosis with a 30-40% five-year mortality rate.
- A phase II/III ATLAS trial for momelotinib in VEXAS syndrome is currently underway.
- The study design for the ATLAS trial will be presented at the 2026 European Hematology Association Congress.
- Momelotinib is already approved in the US, EU, UK, and Japan for myelofibrosis.
- Orphan Drug Designations support development and regulatory evaluations for rare disorders.
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