Familial chylomicronemia syndrome
FCS · CVRM · 1 drugs · 2 indications
Ultra-rare genetic disorder (~1 in 1 million) where the body cannot break down dietary fat particles (chylomicrons). Triglycerides can exceed 10x normal levels. Like a backed-up drain that can't clear fat from blood — leads to devastating pancreatitis attacks, fatty liver, and chronic debilitating symptoms.
Competitive Landscape (1 drugs)
| Drug | Company | Mechanism | Modality | Route | Stage |
|---|
| Tryngolza | SOBI | ApoC-III antisense oligonucleotide | Antisense oligonucleotide | SC | APPROVED |
Indications (2)
Familial chylomicronemia syndrome
Severe hypertriglyceridemia
Upcoming Catalysts
Tryngolza - Severe Hypertriglyceridemia - EMA SubmissionREGULATORY
SOBI2026
Data from Supabase · Updated 2026-03-24