The FDA has Announced Their Approval of Evkeeza for Homozygous Familial Hypercholesterolemia

The FDA has just announced their approval of Evkeeza as a therapy for those living with homozygous familial hypercholesterolemia. The therapy is owned by Regeneron Pharmaceuticals. It was evaluated with FDA Priority Review. In 2017, Evkeeza was granted Breakthrough Therapy Designation, and it is currently undergoing accelerated review in the European Union.


HoFH is an ultra-rare condition. It occurs when an individual inherits one FH causing gene from each parent. It leads to dangerously high levels of LDL-C, also known as bad cholesterol.

The condition can cause premature atherosclerotic disease as well as cardiac events. These can occur when patients are as young as teenagers.


Evkeeza is a monoclonal antibody that binds to ANGPTL3 and blocks its function.

Scientists at Regeneron first found the gene family it belongs to over two decades ago. In 2017, a study was published explaining that when ANGPTL3 doesn’t function as it should, the body produces less blood lipids. This includes LDL-C. When fewer of these lipids exist, patients are at a significantly decreased risk of coronary artery disease.

Regeneron researchers used their VelocImmune technology to produce Evkeeza. This technology uses genetic engineering to produce fully human antibodies in mice.

This therapy is meant to be used in conjunction with other LDL-C lowering therapies. It has been approved for patients at least 12 years of age who are diagnosed with HoFH.

It is the very first FDA approved therapy that blocks ANGPTL3 function.

Phase 3 ELIPSE Trial

This therapy was approved based on results from a Phase 3 trial called ELIPSE. The results from this investigation have been published in the New England Journal of Medicine.

This trial included a total of 65 patients who were randomized to either receive the therapy or placebo (lipid-lowering therapies on their own). Those who received Evkeeza were given 15 mg/kg by IV every 4 weeks in addition to other therapies that lower lipids.

The primary endpoint of the trial was reduction in LDL-C from baseline. The average baseline for patients in the trial was 255 mg/dL. The trial met its endpoint with those patients who were given Evkeeza experiencing a reduction in their baseline LDL-C of 47% on average. For those on placebo, LDL-C increased by 2%. Patients given Evkeeza experienced the reduction as early as two weeks into the trial. All patients maintained their outcomes during the treatment period and open label period.

Notably, patients who had not responded to other therapies due to their limited LDL receptor function experienced the same degree of lowering as other patients receiving Evkeeza.

Secondary endpoints of reductions in apolipoprotein B, non-high density lipoprotein cholesterol, as well as total cholesterol were also documented.

The most common AEs in the trial were nasopharyngitis, flu like symptoms, nausea, dizziness, rhinorrhea, asthenia, and pain in the extremities. AEs caused withdrawal from the trial from two patients taking Evkeeza. In total, less than 3% of all patients documented experiencing AEs.

You can read more about this newly approved therapy here.

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