So Praluent Is Approved. The Question Now Is, Who Gets It?

Forty dollars a day.

That’s the price that Sanofi and Regeneron announced late Friday for alirocumab, the cholesterol-fighting PCSK9 inhibitor they will sell as Praluent, which won FDA approval Friday afternoon.

The amount, which comes to $14,600 a year, was higher than the estimates of $7000 to $12,000, and it quickly drew the attention off the approval itself, which was neither as broad as the drug’s supporters once hoped nor as narrow as some had feared.

The companies announced the price in a press release, saying it was based on the drug’s value. The amount, the statement said, was "the lowest priced patient-administered monoclonal antibody therapy on an annualized basis,” and that “the companies carefully considered the potential medical value that Praluent offers patients in determining the wholesale acquisition cost.”

Pharmacy benefit managers, who have been bracing themselves for the arrival of this drug class, did not sound impressed with that logic. Troyen Brennan, executive vice president of CVS Health Corp., told The Wall Street Journal, “This could become the most expensive medication that we use.”

Sanofi and Regeneron said health plans and PBMs will see discounts, there will be programs for those without insurance, and the effect on the healthcare system that some fear will not materialize. There was disagreement over just how broad FDA’s approval would turn out to be, with 1 analyst suggesting the approval could capture up to 20% of the high-cholesterol population.

Among cardiologists who are eager to prescribe the drug, some think alirocumab’s availability in 2 doses (75 mg and 150 mg) may offer advantages because it will allow clinicians to try the lower dose first. Pricing for the drug, which is given by injection, is the same at either dose, according to the press release issued Friday.

Following the FDA advisory committee meeting of June 9 on alirocumab, followed by the meeting the next day on the drug’s chief rival, evolocumab, some wondered if approval would be limited to a small group of patients with heterozygous familial hypercholesterolemia (FH) hypercholesterolemia, a genetic disorder, leaving out the much larger group of patients with high cholesterol and heart disease that cardiologists hoped to treat.

As recently as this spring, during the meeting of the American College of Cardiology (ACC) and in a literature review of studies of both PCKS9 inhibitors, the most ardent supports of this new class saw it as a therapy for those who cannot tolerate statins, despite the lack of long-term safety data.

FDA did not go that far on Friday. For now, regulators approved the drug only for those with HeFH hypercholesterolemia and for those with heart disease who have had heart attacks or strokes and who have been unable to bring low-density lipoprotein (LDL) cholesterol down to safe levels on maximally tolerated statins.

FDA took a more cautious approach than that of European regulators, who approved evolocumab for broader use last Tuesday and recommended alirocumab for approval Friday. This is typical, and FDA has been especially cautious with diabetes and cardiovascular medications in the years since the furor surrounding Avandia, which became a blockbuster only to have FDA sharply restrict its sales years late.

Trading of Regeneron shares halted Friday afternoon when news of the FDA action was pending, and shares fell 2.62% when trading resumed.

Health plans and PBMs are expected to work aggressively to prevent the PCSK9 class from shocking their balance sheets the way Sovaldi, the first cure for hepatitis C, did in early 2014. Besides negotiating aggressively for discounts, which will be easier with evolocumab facing an August 27, 2015, approval deadline, health plans are expected to scrutinize the FDA approval to ensure that only those who meet the criteria gain access.

The price of the new therapy class will be reviewed by the Institute Clinical and Economic Review (ICER), which received a grant to study cost-effectiveness of new therapies and announced that PCSK9 inhibitors would be first on its list. ICER has already announced a scoping document for this process.

ACC President Kim Allan Williams, MD, FACC, said the cautious approach makes sense while the cardiology community awaits long-term safety studies that are due in 2017. Once that evidence is available, a broader indication may be warranted.

“The ACC eagerly awaits the results of the clinical trials that are in progress. In the meantime, we continue to recommend physicians limit prescribing to the very high risk, hard-to-treat groups approved by the FDA and otherwise follow the current guidelines, which recommend lifestyle change and, if needed, statins for most patients with or at risk of heart disease,” Williams said.