Regeneron's Evkeeza, carrying big price tag, wins FDA approval in ultra-rare cholesterol disease

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Regeneron won approval for Evkeeza to help treat patients with the ultra-rare disease HoFH.

Regeneron has been battling against rival Amgen with their mass-market PCSK9 cholesterol drugs, and now the company is underway with a new launch to match its rival in the ultra-rare disease homozygous familial hypercholesterolemia (HoFH).

Regeneron’s Evkeeza scored approval on Thursday as an add-on to other lipid-lowering therapies in patients 12 and older with HoFH, which affects approximately 1,300 patients in the U.S. The company has a "dedicated and experienced team in place" to support the rollout, a spokesman said. The drug is given once per month through an intravenous infusion.

It's Regeneron's latest cholesterol drug launch after the rollout of Sanofi-partnered Praulent in 2015. Unlike Praluent, which got off to a slow start due to its high price and payer restrictions, Evkeeza targets an ultra-rare disease, likely requiring a different go-to-market strategy.

But while Praluent doesn't carry an approval in HoFH, PCSK9 rival Repatha from Amgen does. Praluent is up for an FDA decision in HoFH later this year.

Still, Evkeeza is a "first of its kind therapy and works on a completely different pathway" from PCSK9 drugs, Regeneron's spokesman said. The new drug is dosed by weight and will carry a list price of about $450,000 per year on average, Regeneron said.

RELATED: Regeneron's Praluent sidekick nabs speedy FDA review, with February PDUFA date

Right now, though numerous therapies exist to lower LDL cholesterol, many HoFH patients can't get their levels to a healthy range, FH Foundation founder and CEO Katherine Wilemon said in an interview.

The inherited disease causes patients to produce “extraordinarily high” levels of cholesterol in the blood, which results in “very early and aggressive cardiovascular disease.” If untreated, patients often experience heart attacks in the first or second decade of their lives.  

Underdiagnosis is also an issue, and Wilemon said a new drug launch could help raise awareness about the disease. Many patients go years before reaching an accurate diagnosis, she said.

Last summer, Evkeeza snagged an FDA priority review on the heels of phase 3 data showing the medicine bested standard care.

In the trial, patients who took Evkeeza experienced LDL cholesterol reductions of 49% from baseline on average compared to placebo at week 24. Almost half of the Evkeeza patients experienced LDL-C reductions to under 100 mg/dL after entering with average levels of 260 mg/dL. Nearly all Evkeeza patients in the trial were on statins, and close to 80% were on a PCSK9 medicine, Regeneron said.  

RELATED: Regeneron's Praluent sidekick cuts cholesterol in half—even in hard-to-treat patients 

In addition to needing several drugs, many HoFH patients and their families routinely travel for lipid apheresis treatments to cleanse the cholesterol from their blood, Wilemon said. While that's an effective treatment, it doesn't last long and cholesterol levels quickly climb back up.

Regeneron is also studying Evkeeza in refractory hypercholesterolemia and severe hypertriglyceridemia.