FDA Approves Another PCSK9 Inhibitor

The US Food and Drug Administration last night announced it has approved evolocumab (Repatha/Amgen), an injectable drug for some patients who are unable to get their LDL cholesterol under control.

Statins are a cheaper and generally effective treatmen for high LDL cholesterol, but the new class of drugs is expected to help people who cannot tolerate them, for whom they do not work, and to benefit people with heterozygous familial hypercholesterolemia.

The FDA said the Amgen drug’s efficacy and safety were confirmed in a 52-week placebo-controlled trial and in eight 12-week placebo-controlled trials in patients with primary hyperlipidemia. Participants taking the drug had an average reduction in LDL cholesterol of approximately 60% compared to placebo.

Evolocumab was approved in Europe last month.

It is the second PCSK9 inhibitor to get FDA approval.

The agency gave the green light to alirocumab (Praluent/Sanofi-Regeneron) for use in addition to diet and maximally tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia (HeFH) or patients with clinical atherosclerotic cardiovascular disease such as heart attacks or strokes, who require additional lowering of LDL cholesterol. Cardiologists have been eagerly awaiting these drugs.

In an interview last month, MD Magazine asked New York City cardiologist David Vorchheimer, MD, of the Montefiore Einstein Center for Heart and Vascular Care what PCSK9 approvals could mean for physicians and patients. Vorchheimer has offices on Manhattan’s Upper Eastside, and at the Bronx-based Montefiore system’s main campus.

Vorchheimer said “For most patients PCSK9 inhibitors will not be first-line agents,” and added, “But for the person who is not tolerating a statin, or who is not at goal with LDL lowering, for me, a cardiologist, this is very exciting.”

Vorchheimer estimated that about 10% to 15% of his own patients might be candidates for PCSK9 inhibitors. Others have estimated that as many as 50% of patients now taking statins might instead take the new drugs.

The fact that the drugs have to be injected is seen as a drawback, compared to statins.

Cost is a big factor compared to statins, Vorchheimer said.

But for patients with HeFH, the cost of PCSK9 inhibitors could be far lower than that of two existing drugs according to Rare Disease Report. Those drugs are lomitapide (Juxtapid/Aegerion) and mipomersen (Kynamro/Genzyme)