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Regeneron's BLA for Cholesterol Drug Accepted by the FDA

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Regeneron Pharmaceuticals, Inc. (REGN - Free Report) announced that the FDA has accepted for Priority Review a Biologics License Application (BLA) for pipeline candidate, evinacumab.

The company is seeking FDA approval for the candidate as an adjunct to other lipid-lowering therapies in patients with homozygous familial hypercholesterolemia (HoFH).

The agency has set a target action date of Feb 11, 2021.

The BLA was supported by positive results from a late-stage study, which evaluated the efficacy and safety of evinacumab in patients with HoFH.

Evinacumab is an investigational fully-human monoclonal antibody that binds to and blocks the function of ANGPTL3 and currently being studied in patients with HoFH (ongoing phase III extension trial), refractory hypercholesterolemia (phase II) and severe hypertriglyceridemia (phase II).

Meanwhile, regulatory submissions for evinacumab are also progressing in the European Union. In June 2020, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended an accelerated assessment for the candidate based on the high unmet medical need and therapeutic innovation demonstrated by the product.

In 2017, the FDA granted evinacumab a Breakthrough Therapy designation for the treatment of hypercholesterolemia in patients with HoFH. HoFH is an ultra-rare inherited disease that affects approximately 1,300 patients in the United States.

ELIPSE (Evinacumab LIPid StudiEs) HoFH was a multi-national phase III randomized, double-blind, placebo-controlled, parallel-group trial evaluating the efficacy and safety of evinacumab 15 mg/kg administered intravenously every four weeks in 65 patients aged 12 years or older with HoFH (43 evinacumab, 22 placebo).

A potential approval will boost Regeneron’s portfolio, although competition is stiff in this space from the likes of Amgen’s (AMGN - Free Report) Repatha.

Regeneron’s shares have surged 63% in the year so far compared with the industry’s growth of 3.9%.

 

We remind investors that Regeneron and Sanofi’s (SNY - Free Report) Praluent is also approved as an adjunct to diet, alone or in combination with other lipid-lowering therapies for the treatment of adults with primary hyperlipidemia (including heterozygous familial hypercholesterolemia) to reduce LDL-C. It is also being evaluated in patients with HoFH.

Approval of new drugs will reduce Regeneron’s dependence on its lead drug, Eylea, for which it has a collaboration with Bayer AG (BAYRY - Free Report) . The overall demand for Eylea in the United States was lower on a year-over-year basis in the recently reported second-quarter results.

Regeneron currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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