Is Evkeeza Approved for Homozygous Familial Hypercholesterolemia?
Yes, Evkeeza (evinacumab-dgnb) is FDA-approved to treat homozygous familial hypercholesterolemia (HoFH) in patients 5 years and older as an adjunct to other low-density lipoprotein cholesterol (LDL-C)-reducing therapies.[1] It targets ANGPTL3, a protein that inhibits lipoprotein lipase, reducing LDL-C by 47-51% in clinical trials when added to standard care.[2]
How Does Evkeeza Work in HoFH?
Evkeeza is a monoclonal antibody given intravenously every 4 weeks. In HoFH patients—a genetic condition causing extremely high LDL-C due to absent or defective LDL receptors—it lowers cholesterol independently of the LDL receptor pathway, addressing a key limitation of statins and PCSK9 inhibitors.[1][2] Phase 3 ELIPSE HoFH trial showed average LDL-C drops of 47% versus 2% placebo at week 24.[2]
Who Qualifies for Evkeeza Treatment?
Pediatric approval covers ages 5+; adults were approved first in February 2021.[1] It's for HoFH confirmed by genetic testing or clinical criteria, used with diet, statins, ezetimibe, or apheresis. Not for heterozygous FH or primary prevention in non-HoFH patients.[3]
What Results Show in Clinical Trials?
| Trial | Patients | LDL-C Reduction (Evkeeza vs Placebo) |
|-------|----------|-------------------------------------|
| ELIPSE HoFH (Phase 3) | 65 adults/children | 47% vs 2% at 24 weeks [2] |
| Open-label extension | Up to 96 weeks | Sustained 40-50% reductions [4] |
No head-to-head trials versus competitors like lomitapide or mipomersen.
Common Side Effects and Risks
Most frequent: upper respiratory infections (45%), flu-like symptoms (27%), fatigue (19%). Serious risks include hypersensitivity reactions and infusion issues; monitor lipids during treatment.[1][3] Long-term safety data limited to ~2 years.
How Does Evkeeza Compare to Other HoFH Drugs?
| Drug | Mechanism | LDL-C Drop | Dosing | Key Drawback |
|------|-----------|------------|--------|-------------|
| Evkeeza | ANGPTL3 inhibitor | 47-51% | IV every 4 weeks | Infusion required |
| Juxtapid (lomitapide) | MTP inhibitor | 40-50% | Oral daily | Liver toxicity, GI issues |
| Kynamro (mipomersen) | ApoB antisense | 25-36% | SubQ weekly | Flu-like symptoms, liver risks |
| Praderol (olpasiran)* | siRNA Lp(a) reducer | Emerging (not HoFH primary) | SubQ every 12 weeks | Phase 3 ongoing |
Evkeeza offers receptor-independent action, better tolerated than orals.[2][5] *Not yet approved for HoFH.
Cost and Access Issues
List price ~$450,000/year; patient assistance via Regeneron covers copays for eligible insured patients.[6] Covered by most Medicare plans but prior authorization required. No generics; patent exclusivity to ~2035.[7]
When Does Evkeeza's Exclusivity End?
Orphan drug exclusivity until February 2028; composition-of-matter patent expires 2034-2036 per DrugPatentWatch.[7] No active Paragraph IV challenges listed.
[1]: FDA Label - Evkeeza (2021, updated 2023). Link
[2]: Regeneron ELIPSE HoFH Trial (NEJM, 2021). Link
[3]: American Heart Association HoFH Guidelines (2023). Link
[4]: Regeneron Data (EAS Congress, 2023). Link
[5]: Comparative Review (JACC, 2022). Link
[6]: Regeneron Co-Pay Program. Link
[7]: DrugPatentWatch - Evinacumab Patents. Link