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


Regeneron has taken a step closer to getting a green light for its experimental high cholesterol drug as the FDA allows a swift review of evicanumab.

Patients with high cholesterol from a rare genetic disorder struggle to keep their cholesterol levels in check, despite taking multiple lipid-busting drugs.

A new drug from Regeneron may change that—even for the most difficult-to-treat patients, and now it could see approval early next year after nabbing a speedy review.

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The pharma has been handed a priority review for evicanumab as an adjunct to other lipid-lowering therapies in patients with homozygous familial hypercholesterolemia (HoFH), an ultra-rare inherited disease that affects approximately 1,300 patients in the U.S. The PDUFA date is February 11, 2021.

The drug could sit next to Regeneron’s already approved fatty blood drug Praluent, which works by blocking PCSK9. Evicanumab works differently, binding to angiopoietin-like protein 3 (ANGPTL-3), which inhibits lipoprotein lipase, an enzyme that breaks down triglycerides and other lipids.

Earlier this year Regeneron published detailed phase 3 data for its drug when pitted against placebo in 65 patients with HoFH who struggled to keep their cholesterol levels in check despite taking multiple cholesterol-busting medicines.

Patients taking evinacumab alongside their usual lipid-lowering meds saw their “bad” cholesterol levels drop 49% from baseline compared to the placebo group: patients taking their regular drugs alone.

Of the 65 patients, 21 (one-third) had the most severe form of HoFH, which often does not respond to other treatments. The 15 patients in this group who received evinacumab saw their “bad” cholesterol levels fall 47.1%, while the six hard-to-treat patients who got placebo saw their cholesterol increase 16.2%—that’s an average relative reduction of 59.6% for evinacumab.

Nearly half of patients taking evinacumab saw their “bad” cholesterol dive to below 100 mg/dL, the level recommended by the Centers for Disease Control and Prevention. It’s this data which has been handed to the FDA for its review.

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