FDA approves first PCSK9 inhibitor to lower cholesterol

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The FDA has approved alirocumab, an injectable cholesterol-lowering medication and the first commercially available proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitor.

Alirocumab (Praluent, Regeneron Pharmaceuticals and Sanofi Aventis) is approved as an adjunct to diet and statins to treat patients with heterozygous familial hypercholesterolemia (HeFH) or patients with clinical atherosclerotic cardiovascular disease who have not been able to lower their low-density lipoprotein (LDL) cholesterol.

The wholesale acquisition cost of alirocumab is $40 per day for the 75 mg and 150 mg doses or nearly $15,000 per year, according to the companies.

“Actual costs to patients, payers and health systems are anticipated to be lower as WAC pricing does not reflect discounts or rebates,” they said in a news release. “Out-of-pocket costs to patients will vary depending on insurance status and eligibility for patient assistance.”

The approval was based on results of five trials that included 2,476 patients who received alirocumab. The patients had HeFH or were at high risk for stroke or heart attack. Compared with the placebo group, the alirocumab group had a 36 percent to 59 percent reduction in LDL cholesterol.

Patients who received alirocumab had more local injection site reactions as well as symptoms of the common cold or flu-like symptoms.

Regeneron and Sanofi Aventis said that alirocumab’s effect on cardiovascular morbidity and mortality has not been determined.

On June 10, an FDA advisory panel voted in favor of evolocumab (Repatha, Amgen), another PCSK9 inhibitor. The FDA is expected to make an approval decision on evolocumab by Aug. 27.

In June, two members of the FDA advisory panel who did not vote in favor of either PCSK9 inhibitors spoke with Cardiovascular Business about their concerns. They mentioned they had issues with using LDL cholesterol as a surrogate markers and would have liked to have seen proof that the drugs prevented or delayed major adverse cardiovascular events such as MI, ischemic stroke and death due to cardiovascular disease.

“We don’t have outcomes; it’s hard to recommend approval without outcomes,” Peter Wilson, MD, of Emory University and the Emory Clinical Cardiovascular Research Institute in Atlanta, said. “I’m on the conservative side of assessing these types of new molecules. We have a pretty good portfolio of lipid medications… I’m data driven, and especially when we have expensive new products, we’ve got to be data driven. We cannot just be anecdotal because once we’re there, the products get overprescribed and we don’t even really know how much benefit we’re getting out of these.”