High-cost PCSK9 cholesterol drugs inch closer to the market

Here come the PCSK9s! The term refers to the mechanism of action of proprotein convertase subtilisin/kexin type 9, a monoclonal antibody that has now been approved for commercialization by Sanofi-Aventis and Regeneron Pharmaceuticals in the US. On the same day, the European Medicines Agency voiced a positive opinion for the same drug; last May, the same agency approved Amgen’s PCSK9, Repatha (evolocumab), and on July 21, the European Commission gave Repatha full market authorization. Both are expected to be offered at much higher cost than the widely used statin therapies, most of which are now generic.

7/27 UPDATE: According to several press reports, Sanofi-Aventis and Regeneron have settled on a price for Praluent that works out to $14,600 per year, or about $560 per biweekly dose. Amgen is reportedly preparing a biweekly and a monthly version of Repatha.

Drug approval is a three-step process in Europe: first, the EMA, then the European Commission, then each member nation of the EU. So, in the race to commercialization, it appears that Sanofi-Aventis’ product, Praluent (alirocumab), is first to cross the line in the US, while Amgen’s Repatha will be first across the line in Europe. An FDA review committee approved Repatha in June, and final approval is pending.

Both drugs target patients with high, uncontrolled cholesterol, but payers fear that they will be prescribed indiscriminately to the millions of patients currently taking statins. A just-released report from Prime Therapeutics, a pharmacy benefit manager owned by most of the Blue Cross/Blue Shield health plans, posited that if the drugs have pricing of $7,000-12,000 per year, and if used by 2.3 million patients, the therapy could cost the US health system $23 billion per year, every year. “If used by only 40% of the more than 600,000 Americans with a rare genetic condition leading to abnormally high cholesterol levels for which statins aren’t always effective, they could still add an additional $2.1 billion per year in new costs,” the report adds. Similar concerns have been voiced by other PBMs since the beginning of this year—a highly unusual situation of a lively pricing and cost debate well before the drugs were approved, and before any of the pharma companies have actually issued a price sheet.

But that’s a lot of ifs. The Prime Therapeutics report notes that adherence to generic statin pills is a continuing problem for the 42 million Americans diagnosed with high cholesterol. PCSK9s require a biweekly or monthly injection, presumably with a self-administered pen. The drugs are specifically targeted for patients with dhypercholesterolemia and mixed dyslipidemia, and for homozygous familial hypercholesterolemia—conditions not well controlled by statins. In any case, Prime has already prepared an entry in its drug cost calculator to help health plans estimate the cost impact of PCSK9s. How’s that for the drugs’ reception in the market!