Homozygous familial hypercholesterolemia

HoFH · CVRM · 1 drugs · 1 indications

Ultra-rare genetic disorder with severely elevated LDL-C from birth.
Competitive Landscape (1 drugs)
DrugCompanyMechanismModalityRouteStage
EVKEEZAREGNANGPTL3 inhibitor (monoclonal antibody)Monoclonal antibodyIVAPPROVED
Indications (1)
Homozygous familial hypercholesterolemia
EVKEEZA APPROVED
Data from Supabase · Updated 2026-03-24