FDA Approves Praluent, the First PCSK9 Inhibitor in the US

A $14,600-a-year cholesterol-lowering drug was approved by FDA on July 24, 2016, and it was the first drug in its class to be approved in the US. The approval was for Regeneron and Sanofi’s Praluent (alirocumab), an injectable monoclonal antibody. “We don’t want the noise about these drugs to be price,” Leonard Schleifer, the chief executive officer and founder of Regeneron, told Forbes. “We have to make sure that people get access to the drug for a fair price if they’re insured, for free if they’re not insured, or at a discount if they’re underinsured.” A press release from Regeneron explains the price of Praluent, saying it is consistent with or even better than the price of other mAbs: “The US WAC [wholesale acquisition price] price of Praluent is $40 per day ($1120 every 28 days) for both the 75 mg and 150 mg doses, making Praluent the lowest priced patient-administered monoclonal antibody therapy on an annualized basis.”

"Praluent represents the culmination of more than a decade of tireless work to translate the genetic-based discovery of PCSK9 into an innovative medicine that brings meaningful value to patients," Schleifer said in a Regeneron press release.

Praluent was approved for many indications related to lowering of cholesterol for high-risk patients, but was not approved for the drug’s ability to lower low-density lipoprotein (LDL) alone in patients (i.e., primary prevention in statin-intolerant patients). A Praluent competitor, however, Amgen’s Repatha (evolocumab), was approved for a more broad indication in Europe on June 10, 2015. The broad approval overseas indicates that EMA accepts the control of LDL as a surrogate endpoint.

There is currently only limited information about how this class of medications will affect clinical outcomes and the prevention of cardiovascular events. Although early studies look promising, the lack of concrete proof at this stage may have prompted FDA to be more conservative with the indications covered by the Praluent approval. “Because more robust outcomes data, which could show whether these drugs reduce incidence of death and heart attacks, will not be available until 2017, the FDA has approved it for limited groups of patients at very high risk," said American College of Cardiology President Kim Allan Williams Sr, MD, FACC, in an emailed statement. "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."

Sources: FDA, Forbes, and Regeneron