FDA panel backs Amgen's cholesterol drug for high-risk patients

[June 11, 2015]  By Toni Clarke

WASHINGTON (Reuters) - An advisory panel to the U.S. Food and Drug Administration recommended approval of Amgen Inc's cholesterol-lowering drug Repatha on Wednesday, but said it should be used only in patients at high risk of cardiovascular disease.

The panel voted unanimously to recommend approval of the drug, known also as evolocumab, for patients with homozygous familial hypercholesterolemia (HoFH), a hereditary disorder characterized by high LDL levels that can cause heart attacks in very young people.

The panel also recommended approval of Repatha in patients without HoFH who are at high risk of cardiovascular disease and are taking other cholesterol treatments, with a 11-4 vote.

The FDA is not obliged to follow the advice of its advisory panels but typically does so.

Repatha is one of a new class of cholesterol-lowering drugs known as PCSK9 inhibitors. Unlike statins such as Pfizer Inc's Lipitor, which are pills, PCSK9 inhibitors are biologic drugs given by injection. They are expected to be far more expensive than statins.

The panel on Tuesday recommended approval for another PCSK9 drug, Praluent, made by Regeneron Pharmaceuticals Inc and Sanofi SA.

The FDA is expected to makes its approval decisions this summer.

Repatha is designed to be given in biweekly doses of 140 mg or a monthly dose of 420 mg, on top of statins if patients can tolerate them.

Panelists raised questions about what physicians should do if Repatha, combined with statins, were to cause LDL, or so-called bad cholesterol, to drop too low. They fear that if physicians become alarmed by very low LDL levels they could cut back on the patient's statin, which would not be desirable since there is a large body of data showing that statins reduce the risk of heart attacks and strokes.

Panelists were reluctant to recommend the drug for a wider patient population until Amgen completes a large trial to assess whether Repatha's cholesterol-lowering impact translates into a reduction in cardiovascular risk.

Positive results from the outcomes study could eventually expand use of the medicines. If approved, Praluent and Repatha are expected to generate annual sales of more than $2.5 billion each by 2020, according to Thomson Reuters data.

However, pharmacy benefit managers, such as Express Scripts and CVS Health, are expected to demand steep discounts from the drugmakers in exchange for favoring use of one medicine or the other as they have with new hepatitis C treatments.

Amgen's shares were flat in after-hours trading after rising 0.5 percent to close at $155.55 on Nasdaq. Regeneron's shares declined 2.6 percent to close at $512.32 on Wednesday. They were halted during Tuesday's panel discussion.

(Reporting by Toni Clarke; Additional reporting by Bill Berkrot; Editing by Sandra Maler and Leslie Adler)

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