FDA Panel Recommends Approval of Praluent

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An FDA advisory panel voted 13-3 in favor of approval of Sanofi and Regeneron Pharmaceuticals' cholesterol-homeostasis therapy Praluent (alirocumab).


An FDA advisory panel voted 13-3 in favor of approval of Sanofi and Regeneron Pharmaceuticals' cholesterol-lowering therapy Praluent (alirocumab) on June 9, 2015. Its target action date is July 24, 2015.

Clinical data on Praluent published in March 2015 demonstrated that the drug significantly reduced the incidence of cardiovascular events in patients with hypercholesteremia and was associated with an approximately 60% reduction in low-density lipoprotein (LDL) cholesterol when compared with statins, the current standard of care.

While patients who do not respond well to statins, have adverse reactions to statins, or patients with a high risk of cardiovascular events could be good candidates for treatment with these mAbs, the advisory panel determined there needs to be more data on cardiovascular clinical trial outcomes to determine that the PCSK9 inhibitors are appropriate for this larger patient population. In briefing documents released ahead of the meeting, the panel wrote, “While cardiovascular events within this application are of interest, the number of adjudicated events is too small overall and within subgroups to make any reliable conclusions regarding the effect of alirocumab on risk of cardiovascular events. The ongoing event-driven cardiovascular OUTCOMES study with an estimated enrollment of 18,000 patients with acute coronary syndrome is designed to establish the effect of alirocumab on cardiovascular morbidity and mortality.” The panel concluded, however, that for patients with heterozygous familial hypercholesterolemia, Praluent’s benefits exceed its risks and the drug fulfills an unmet need.

"The discovery of PCSK9 as a powerful regulator of cholesterol levels and cardiovascular disease was one of the most important human genetic advances of the last decade," said George D. Yancopoulos, MD, PhD, chief scientific officer of Regeneron and president, Regeneron Laboratories, in a statement. "Today's outcome brings us one step closer to translating this genetics-based discovery into a treatment that may help the many patients in need of additional cholesterol lowering."


Sanofi and Regeneron essentially paid $67.5 million to have its drug known as the first-to-market PCSK9 monoclonal antibody. The companies got Priority Review status for Praluent through the purchase of a Rare Pediatric Disease Priority Review Voucher from BioMarin; BioMarin won the voucher when it received approval of Vimizim (elosulfase alfa) for Morquio A syndrome. As a result, the drug beat Amgen’s Repatha (evolocumab) in the race to FDA review. An FDA advisory committee is scheduled to review Repatha on June 10, with a target action date of August 27.

If prescribed to those with severe hypercholesteremia alone, CVS Health estimates that this class of therapies would cost $16 billion per year in the US. If the therapies are approved for patients with a history of coronary disease-a larger patient population-CVS Health predicts that PCKSK9 inhibitors would increase healthcare costs in the US by $150 billion per year. On June 9, Prime Therapeutics estimated that the approval of PCSK9 inhibitors could cost the US health system up to $23.3 billion per year to treat all the potential people who could be candidates to receive the drug. Regardless of the total cost to the US healthcare system, PCSK9 medications are expected to cost approximately $10,000 per year per patient.

Amgen announced in late 2014 that it is suing Sanofi and Regeneron to prevent the allegedly infringing manufacture, use, and sale of Sanofi and Regeneron's Praluent. Even though Amgen filed three patents for a mAb targeting PCSK9 first, on Aug. 28, 2014, Amgen’s product would be subject to the standard review, which lasts 10 months.

Source: PR Newswire