FDA approves Regeneron’s Evkeeza for HoFH

pharmafile | February 12, 2021 | News story | Medical Communications FDA, Regeneron 

The FDA has approved Regeneron’s Evkeeza (evinacumab-dgnb) as an adjunct to other low-density lipoprotein cholesterol (LDL-C) lowering therapies to treat adult and paediatric patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH).

Evkeeza is the first FDA-approved treatment that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3), a protein that plays a key role in lipid metabolism.

HoFH, also known as homozygous FH, is an ultra-rare inherited condition that affects approximately 1,300 patients in the US. HoFH occurs when two copies of the familial hypercholesterolemia (FH)-causing genes are inherited, one from each parent, resulting in dangerously high levels of LDL-C. Patients with HoFH are at risk for premature atherosclerotic disease and cardiac events as early as their teenage years.

The FDA approval is based on results from the Phase III ELIPSE HoFH trial, in which 65 patients were randomised to receive either Evkeeza 15mg/kg intravenously every four weeks plus other lipid-lowering therapies or lipid-lowering therapies alone. The mean baseline LDL-C level of patients in both groups was 255 mg/dL.

The trial met its primary endpoint, with Evkeeza-treated patients reducing their LDL-C from baseline by 49% on average compared to placebo at week 24. Reductions in LDL-C seen with the drug were observed as early as week two, and maintained throughout the double-blind treatment period and open label trial period.

Dr George D Yancopoulos, President and Chief Scientific Officer at Regeneron, said: “Evkeeza is the first FDA-approved ANGPTL3 inhibitor and the latest example of the promise of Regeneron’s development approach that harnesses genetic insights and pioneering technology to deliver new treatment options for patients who need them.

“We are proud to bring Evkeeza to patients with HoFH, and Regeneron is grateful to the patients and doctors who participated in our trials to make this a reality.”

Katherine A Wilemon, Founder and CEO of the FH Foundation, also commented: “The FDA’s approval of Evkeeza is a watershed moment for individuals born with HoFH, a severe form of familial hypercholesterolemia.

“Those living with HoFH have faced devastatingly high LDL-C levels and an uncertain future. Evkeeza significantly lowered LDL-C levels in clinical trials and this new treatment offers an important new option for people living with HoFH.”

Darcy Jimenez

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