Primary hemophagocytic lymphohistiocytosis

pHLH · Immunology · 1 drugs · 1 indications

Ultra-rare hyperinflammatory syndrome where immune cells (macrophages, T-cells) become overactivated and attack the body own tissues. Think of it as the immune system "friendly fire" that won't stop — cytokine storm destroys organs. Fatal without treatment. ~1 in 100,000 births.
Competitive Landscape (1 drugs)
DrugCompanyMechanismModalityRouteStage
GamifantSOBIAnti-interferon-gamma monoclonal antibodyMonoclonal antibodyIVAPPROVED
Indications (1)
Primary HLH
Gamifant APPROVED
Upcoming Catalysts
Gamifant - IFN-gamma-driven Sepsis - Ph2b/3 Design DiscussionCLINICAL
SOBI2026
Data from Supabase · Updated 2026-03-24