ICYMI: Good Results From Evinacumab for Rare Form of Familial Hypercholesterolemia


A press release issued recently by Regeneron Pharmaceuticals heralded the acceptance of a Biologics License Application by the FDA for Priority Review of its investigational drug, evinacumab.

The Priority Review will expedite the FDA’s decision but does not guarantee the FDA’s approval. The developer of the drug also pays a substantial fee for the expedited review.

The FDA’s decision, according to the Priority Review, is due as of February 11, 2021. In 2017, evinacumab received the designation of Breakthrough Therapy which expedites the development of drugs intended to treat life-threatening disorders.

The drug is intended to be used as an adjunct to other therapies that are designed to lower LDL in patients who have homozygous familial hypercholesterolemia.

About Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia (HoFH), is an extremely rare genetic disease affecting approximately 1300 people in the United States. It is the result of a defect on chromosome 19.

The disorder causes substantially elevated levels of low-density lipoprotein cholesterol (LDL). Patients may experience atherosclerotic disease or may have cardiac events in their teens.

The guidelines for treating HoFH suggest lowering the LDL-C soon after the disease is discovered. However, patients are partially or even entirely unresponsive to the lipid-lowering therapies that are currently available.

About Evinacumab

Evinacumab is known to be the first drug in its class that has a positive effect on HoFH patients. That would include patients who have either minimal or no receptor function. Evinacumab binds to ANGPTL3 and blocks its function.

Research that was published in a 2017 issue of the NEJM indicated that patients who lost function in their ANGPTL-3 gene exhibited substantially fewer blood lipids including LDL-C.

This discovery led to the creation of evinacumab which blocks the ANGPTL3 protein. Evinacumab compensates for the loss-of-function mutation by lowering LDL-C in HoFH patients.

A Forty-Nine Percent Reduction in LDL

Compared to lipid-lowering treatment, evinacumab reduced LDL cholesterol in HoFH patients by forty-nine percent.

The Phase 3 Elipse Trial

The Biologics License Application was submitted based on the results of a positive Phase 3 Trial involving sixty-five HoFH patients with the minimum qualifying age set at twelve years.

Patients were administered treatment intravenously every four weeks. Of that total, forty-three patients were administered evinacumab and twenty-two patients received a placebo.

The reduction of LDL-C in HoFH patients treated with evinacumab versus the placebo was the primary endpoint. The trial period lasted twenty-four weeks with all patients completing the entire course of treatment. The therapy was well tolerated.

Evinacumab’s reduction of LDL-C from the recorded baseline in comparison to the placebo was the study’s primary endpoint.

Trial results were presented at the March 2020 annual session of the American College of Cardiology.

The Evinacumab Story

The developer, Regeneron, used technologies for evinacumab that it had created for the treatment of the Ebola virus. The FDA is currently reviewing the treatment for use with COVID-19 patients.

Evinacumab was invented using a genetically-engineered mouse model. The model was created with an immune system that was genetically-humanized to produce monoclonal antibodies. These antibodies act like human antibodies in the immune system.

In addition to the ongoing Phase 3 trial, Regeneron is conducting a Phase 2 trial for patients with refractory hypercholesterolemia that is resistant to treatment.

A third clinical trial conducted by Regeneron is a Phase 2 study of patients with severe hypertriglyceridemia.

EU Submissions

Evinacumab is also being considered for regulatory submissions in the European Union. The EU’s Agency CHMP recommended that the drug’s assessment be accelerated due to the therapeutic benefits it has displayed and also the great unmet need for such a product.

Note that evinacumab has not yet undergone a full evaluation by a regulatory authority.

About Regeneron

As a leader in biotechnology, Regeneron’s focus is on the development of medicine for patients who have serious diseases.

The company was founded over thirty years ago. Seven of its treatments have been approved by the FDA. It currently has numerous products in development by its physician-scientists that have been created in its labs.

Regeneron’s Genetics Center is currently conducting one of the world’s largest genetics sequencing studies. The scientists are looking for genetic alterations in patients who have cancer and inherited conditions such as cystic fibrosis.


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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