FDA Approves First PCSK9 Inhibitor

Cholesterol-lowering drug is expected to cost between $7,000 and $12,000 annually.

The FDA today approved the first cholesterol-lowering Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) inhibitor alirocumab (Praluent), which could prove to be the latest budget-busting specialty drug.

Praluent was approved for use addition to diet and maximally tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia (HeFH) or for patients with clinical atherosclerotic cardiovascular disease such as heart attacks or strokes, who require additional lowering of low density lipoprotein (LDL) cholesterol.

“Praluent provides another treatment option for patients with HeFH or with known cardiovascular disease who have not been able to lower their LDL cholesterol enough on statins,” said John Jenkins, MD, director of the Office of New Drugs, Center for Drug Evaluation and Research. “The FDA strongly supports continued work to provide new and innovative options for the treatment and prevention of cardiovascular disease.”

PCSK9 inhibitors could cost the US health care system billions annually, according to pharmacy benefit manager Prime Therapeutics. The PCSK9 inhibitor Repatha by Amgen is also expected to be approved shortly by the FDA.

Unlike current statins that are taken orally and have less costly generic alternatives, PCSK9 inhibitors are self-injected monoclonal antibodies that block the PCSK9 protein from inhibiting removal of LDL from the liver. The drugs are expected to cost between $7,000 and $12,000 annually.

“While these new drugs offer hope to those who aren’t able to effectively manage their cholesterol with existing therapy, they need to be affordable,” said Patrick Gleason, PharmD, director of health outcomes at Prime. “Just to treat those with familial hypercholesterolemia alone, these drugs could cost the US health system billions of dollars per year, and as much as $23.3 billion to treat all potential people who could receive the drug.”

Prime estimates the drugs could add between $0.93 and $6.71 per member per month (PMPM) to commercial insurance coverage costs and up to $15.66 PMPM to Medicare coverage costs depending on the conditions PCSK9 inhibitors are approved to treat. If the drugs were prescribed to 40% of approximately 600,000 Americans with abnormally high cholesterol levels that statins don’t reduce, it could add an additional $2.1 billion annually in new costs.

“If not managed appropriately, the costs of PCSK9s could have a dramatic impact on overall drug spend starting later this summer,” said Pete Clagett, senior vice president of integrated care and specialty at Prime. “In a couple years, PCSK9s could cost America the equivalent of $73 per person per year.”

Express Scripts Chief Medical Officer Steve Miller told the Washington Post that PCSK9 inhibitors have the potential to be the biggest category in the history of pharmaceutical drugs. Miller added his company could pursue the same approach it took with high cost hepatitis C drugs, when it exclusively offered Viekira Pak after a discount from the manufacturer as a result of the cost for competitor drug Sovaldi.

“We have made it very clear to them that we’re willing to go exclusive with one drug or the other, if they don’t competitively price these drugs,” Miller told the Post.