VEXAS syndrome

VEXAS · Hematology · 1 drugs · 1 indications

Recently discovered (2020) autoinflammatory disease caused by UBA1 gene mutations in blood stem cells. Causes blood clots, lung inflammation, recurrent fevers, and bone marrow failure. Predominantly affects men over 50. Mean survival ~5 years from diagnosis. No approved therapies.
Competitive Landscape (1 drugs)
DrugCompanyMechanismModalityRouteStage
VonjoSOBIJAK2/IRAK1 inhibitorSmall moleculePOAPPROVED
Indications (1)
VEXAS syndrome
Vonjo PHASE2
Upcoming Catalysts
Vonjo - MF (severe thrombocytopenia) - Ph3 - Confirmatory (PACIFICA)CLINICAL
SOBI2026-2027
Data from Supabase · Updated 2026-03-24