FDA accepts priority review for Regeneron’s evinacumab, which could join Praluent in cholesterol market

Almost a year ago to the day, Regeneron announced solid Phase III testing results for evinacumab, a compound that’s designed to lower LDL cholesterol in patients who need treatment beyond a PCSK9 inhibitor. As that anniversary approaches, the biotech is potentially one step closer to putting it on the market.

The FDA has granted priority review and accepted a BLA for evinacumab as a supplement to other lipid-lowering therapies in individuals with homozygous familial hypercholesterolemia, Regeneron announced Wednesday morning. Regeneron’s target action date is next February 11.

Evinacumab is still under review in Europe, with the EMA having recommended an accelerated assessment.

Regeneron already has a PCSK9 inhibitor on the market in Praluent, originally approved back in 2015. Praluent and Amgen’s Repahta, the other big PCSK9 drug on the market, engaged in a sparring match with insurers after lower-than-expected adoption due to sticker shock. Their prices was slashed to $5,850 in October 2018 after insurers won that battle.

HoFH is a rare condition in which patients have mutations in both copies of either PCSK9, LDLR or APOB genes. This can increase their risk for premature atherosclerotic disease and cardiac events as early as their teenage years.

Those with the disease typically do not respond well to the standard PCSK9 inhibitors and other lipid-lowering treatments like statins. The Phase III results from last year showed that individuals on evinacumab showed a 49% reduction in LDL cholesterol from baseline compared to just 2% in the placebo group, which was still taking other medications to lower cholesterol.

At the time, analysts noted that evinacumab could be a boon for individuals who tried drugs like Praluent or Repatha and saw little effect. Cowen analyst Yaron Werber wrote that peak sales could reach $200 million to $400 million in the US with similar numbers in Europe.

Evinacumab itself targets angiopoietin-like protein 3, or ANGPTL3, which inhibits lipoprotein lipase and endothelial lipase and plays a key role in lipoprotein metabolism. The compound is also being studied in patients with refractory hypercholesterolemia and severe hypertriglyceridemia, both in Phase II.

Should the compound be approved, it could feasibly join a long line of Regeneron’s effective antibody treatments. Drugs such as Dupixent, Kevzara, Libtayo and Praluent have all come from the same genetically-engineered mouse platform that the company used to develop evinacumab. Regeneron noted, however, that the compound has yet to be fully evaluated for safety and efficacy by any regulatory authority.

Those four drugs pulled in a combined $1 billion-plus for the biotech in the second quarter of 2020, though the vast majority came from Dupixent sales. The eczema drug, which was recently approved for children ages six to 11 with moderate-to-severe atopic dermatitis, netted the company $945 million in the quarter.