BLA for Evinacumab, a Treatment for HoFH, Receives Priority Review Status


This week, Regeneron Pharmaceuticals announced that their Biologics License Application (BLA) for evanicumab, a treatment for homozygous familial hypercholesterolemia (HoFH), was granted Priority Review status by the FDA. The investigational therapy could be used in conjunction with other lipid-lowering treatments, providing relief for patients.


Familial hypercholesterolemia (FH) is an inherited form of extremely high cholesterol. Currently, it has two main subtypes: heterozygous (HeFH) and homozygous (HoFH). Patients with HoFH require two LDLR, APOB, or PCSK9 gene mutations; those with HeFH require only one. Approximately 1,300 American citizens have HoFH. People with Afrikaan, French Canadian, Lebanese, and Finnish backgrounds have a higher risk of inheriting this condition.

According to the American Heart Association, cholesterol is:

a waxy substance [that comes from] your liver [and] foods derived by animals.

This substance plays a role in hormone and cell membrane development, as well as fat digestion. But too much cholesterol, warns the AHA, can:

form a thick, hard deposit on the inside of the arteries, [narrowing the arteries and making them less flexible], – a condition known as atherosclerosis. If a blood clot forms and blocks one of these narrowed arteries, a heart attack or stroke can result.

Patients with HoFH often have excessively high levels of a “bad” form of cholesterol called low-density lipoprotein cholesterol, or LDL-C. This is because receptors in their blood stream are defective or missing. As a result, cholesterol accumulates in blood. Generally, HoFH is resistant to treatments, like statins, which are normally used to treat high cholesterol.

Symptoms include:

  • Coronary artery disease
  • Chest pain
  • Xanthomas, or collections of cholesterol buildup, on hand, finger, and heel tendons
  • Cholesterol buildup around the eyelids
  • Heart attacks

Learn more about HoFH here.


To create evinacumab, Regeneron used their proprietary VelocImmune® platform. Through this, Regeneron creates human monoclonal antibodies through a genetically-modified mouse platform with humanized immune systems. The therapy, an investigational monoclonal antibody, inhibits angiopoietin-like 3 (ANGPTL3). Previously, reducing ANGPTL3 function was shown to also reduce lipid levels, such as LDL-C.

The efficacy of evinacumab is currently being studied in a Phase 3 extension trial, following positive data from the Phase 3 ELIPSE trial. During that trial, 65 patients with HoFH received either a placebo or intravenous injections of 15mg/kg evinacumab every 4 weeks for a 5.5 month period. Trial results are published here.

Ultimately, evinacumab was shown to reduce LDL-C levels in patients better than prior treatment options. The FDA aims to respond to the BLA in full by February 2021.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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