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FDA Approves First PCSK9 Inhibitor

Indication given for heterozygous HF and secondary prevention…

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The FDA granted approval to alirocumab (Praluent) for heterozygous familial hypercholesterolemia (FH) and for patients with clinical atherosclerotic cardiovascular disease, making it the first in the new class of lipid-lowering PCSK9 inhibitors.

This is the first proprotein convertase subtilisin kexin 9 (PCSK9) inhibitor to be approved in the U.S., although the agency is widely expected to approve another PCSK9 inhibitor, evolocumab (Repatha, Amgen), within weeks. The approval was for use in addition to diet and maximally tolerated statin therapy in adults who require additional LDL cholesterol lowering.

Notably left out of the indications listed in the announcement was statin-intolerant patients without established clinical atherosclerotic cardiovascular disease, such as a prior heart attack or stroke.

Lead investigator on the pivotal trials, Christopher Cannon, MD, of Brigham and Women’s Hospital in Boston added, “I think that seems like a very prudent group of patients to treat,” noting that about 85% of patients in the clinical trial program fit in the secondary prevention bucket.

European regulators also recommended approval of alirocumab, although the European Commission still has to give the final okay as it did for a second PCSK9 inhibitor evolocumab last week.

That approval was broader, including primary hypercholesterolemia or mixed dyslipidemia alone or with other lipid-lowering therapy for patients unable to tolerate a statin and approval down to age 12 for homozygous familial hypercholesterolemia.

The injectable monoclonal antibody will be an important option for familial hypercholesterolemia (FH).

Patients with familial hypercholesterolemia and other very high risk conditions who cannot achieve optimal LDL levels with current therapies are going to be the direct beneficiaries of this revolutionary genetic approach to drug discovery.

The wholesale acquisition price for alirocumab on the U.S. market will be $1,120 every 28 days for both the 75 mg and 150 mg doses, Sanofi-Aventis and Regeneron Pharmaceuticals announced.

That price point makes alirocumab “the lowest priced patient-administered monoclonal antibody therapy on an annualized basis,” they said in a press release, pointing out that “the companies carefully considered the potential medical value that Praluent offers patients in determining the wholesale acquisition cost.”

Market research has suggested the price tag could be in the $7,000 to $12,000 per year range. The actual cost announced comes in well above the top of that range at about $14,600 per year ($40 per day).

The FDA approval for alirocumab is widely expected to be followed closely by approval of evolocumab (Repatha) by Aug. 27.

The decision followed last month’s 13-3 FDA advisory committee recommendation for approval for alirocumab and an 11-4 vote in favor of evolocumab approval. The five placebo-controlled clinical trials with alirocumab showed average LDL cholesterol reductions 36% to 59% over placebo.

Practice Pearls:

  • The first proprotein convertase subtilisin kexin 9 (PCSK9) inhibitor was approved in the U.S. with two more to follow shortly. The approval was for use in addition to diet and maximally tolerated statin therapy in adults who require additional LDL cholesterol lowering. It is not yet approved for those who cannot tolerate statins.
  • The wholesale acquisition price for alirocumab on the U.S. market will be $1,120 every 28 days for both the 75 mg and 150 mg doses.

FDA News Release July 2015.