Selected approval record (2021-02-11): FDA approved evinacumab as an add-on treatment for adults and children aged 12 years and older with HoFH. This is the original indication and age range at approval.
LDL-C: 49 percentage-point reduction vs placebo · 24 weeks
15 mg/kg every 4 weeks
Homozygous familial hypercholesterolemia; 63 adults and 2 adolescents
ITT least-squares means from mixed-model repeated-measures analysis.
Safety context
Label risks include infusion reactions, serious hypersensitivity and fetal harm.
Limitations: Rare-disease population: not representative of usual elevated LDL. TG was outside the testing hierarchy. HDL excluded because the label uses a different safety population.
Status in this atlas: Selected approval record (2021-02-11): FDA approved evinacumab as an add-on treatment for adults and children aged 12 years and older with HoFH. This is the original indication and age range at approval. Status source
Binds and inhibits ANGPTL3, relieving its inhibition of lipoprotein lipase and endothelial lipase.
Uses, doses and availability depend on the formulation, indication and country. Study doses below describe research; they are not prescribing instructions.
The molecule
Human IgG4 monoclonal antibody directed against ANGPTL3.
Structure reference
Evinacumab
Human IgG4 monoclonal antibody directed against ANGPTL3.
Shown: Recombinant antibody protein.
Protein class is verified from the label. No exact whole-antibody atomic structure has been verified for this bundle; no substitute protein model is included.
A next milestone is not summarized for this therapy. This does not mean development has ended.
Development history4 selected milestones
Evinacumab · compound history across indications
Selected sourced milestones, not a complete development history. Dates distinguish study starts, publications, announcements and approvals; the first listed date is not necessarily when development began.
Publication
First-in-human ANGPTL3 inhibition results
The publication combined human genetic evidence with a phase 1 study of evinacumab in volunteers with elevated lipids. The clinical experiment established early lipid-lowering activity, not a cardiovascular-event benefit of treatment.
An open-label study in nine adults with homozygous familial hypercholesterolemia reported the effects of adding evinacumab to intensive lipid-lowering regimens, providing disease-specific evidence before the pivotal randomized trial.
The randomized phase 3 trial showed substantial LDL-C lowering when evinacumab was added to existing therapy in HoFH. This trial supplies the endpoint and time-course evidence plotted here; online publication preceded the August 20 journal issue.
FDA approved evinacumab as an add-on treatment for adults and children aged 12 years and older with HoFH. This is the original indication and age range at approval.