Regeneron has been in an intense competition with Amgen with their mass-market PCSK9 cholesterol drugs, and now the firm has its firm set at launching a new product to match its rival in the ultra-rare disease homozygous familial hypercholesterolemia (HoFH).
Regeneron’s Evkeeza secured approval on Thursday as a supplement to other lipid-lowering therapies in patients 12 and older with HoFH, which affects about 1,300 patients in the U.S. The company has a “dedicated and experienced team in place” to back the rollout, a spokesman disclosed. The drug is administered once per month via an intravenous infusion.
It is Regeneron’s newest cholesterol drug launch after the launching of Sanofi-partnered Praulent in 2015. Unlike Praulent, which took off to a slow start as a result of its high cost and payer restrictions, Evkeeza targets an ultra-rare disease, possibly requiring a unique go-to-market strategy.
But while Praulent doesn’t hold an approval in HoFH, PCSK9 rival Repatha from Amgen does. Praulent is set for an FDA decision in HoFH later this year.
Still, Evkeeza boasts as a “first of its kind therapy and works on a completely different pathway” from PCSK9 drugs, Regeneron’s spokesman remarked. According to the company, the new drug is dosed by weight and will have a list price of approximately $450,000 per year on average.
At the moment, though several therapies exist to reduce LDL cholesterol, a lot of HoFH patients aren’t able to get their levels to a healthy range, FH Foundation founder and CEO Katherine Wilemon disclosed in an interview.
The inherited disease causes patients to have “extraordinarily high” levels of cholesterol in the blood, which brings about “very early and aggressive cardiovascular disease.” If untreated, patients are often subjected to heart attacks between ages 10-30.
Underdiagnosis is also a pertinent issue, and Wilemon disclosed that a new drug rollout could help promote awareness about the disease. A lot of patients go years before getting an accurate diagnosis, she said.
In 2020 summer, Evkeeza achieved an FDA priority review on the back of phase 3 data showing the medicine bested standard care.
In the trial, patients who were administered Evkeeza experienced a 49% decrease in LDL cholesterol from baseline on average compared to placebo at week 24. Nearly half of the Evkeeza patients underwent LDL-C reductions to below 100 mg/Dl after starting out with average levels of 260 mg/Dl. Almost all Evkeeza patients in the trial were on statins, and about 80% were on PCSK9 medicine, Regeneron disclosed.
The company is also studying the efficacy of the drug in refractory hypercholesterolemia and severe hypertriglyceridemia.