FDA advisory panel backs approval of Sanofi, Regeneron's Praluent

An FDA advisory panel on Tuesday voted 13-3 in support of approval of Sanofi and Regeneron Pharmaceuticals' cholesterol-lowering therapy Praluent (alirocumab). However, many of the panellists suggested that use of the PCSK9 inhibitor should be limited to certain patients, such as those with familial hypercholesterolaemia. Some members of the advisory committee additionally questioned whether the data submitted by the drugmakers confirm the effect of the therapy on cardiovascular risk.

In briefing documents released ahead of the panel meeting, FDA staff concluded that Praluent significantly reduces LDL cholesterol levels and is well tolerated, while questions were raised regarding whether the risks of the drug exceeded its benefits. Meanwhile, clinical data published in March in the NEJM showed that Praluent, as well as Amgen's PCSK9 inhibitor Repatha (evolocumab), reduced the risk of cardiovascular events by around half, while cutting LDL cholesterol levels by more than 60 percent. Further data from large-scale studies clarifying whether the two PCSK9 inhibitors alter cardiovascular outcomes are expected by 2017.

Panel member Nancy Geller suggested that Praluent should be approved for patients with heterozygous familial hypercholesterolaemia. "I don't want to deprive them of this drug for another two years," Geller remarked. However, Geller and other panel members said that for the majority of potential patients, they would first want to see data from the cardiovascular outcomes trial (for further analysis, see ViewPoints: How Sanofi and Regeneron looked to improve chances of PCSK9 AdCom success).

In addition, several panel members noted that while statin drugs have been shown to lower LDL cholesterol and reduce cardiovascular disease, it's not clear if PCSK9 inhibitors will do the same. "I don't believe we have enough data today," commented committee member William R. Hiatt. Meanwhile, panel chairman Robert J. Smith also expressed the concern held by a number of others that the effect of LDL cholesterol "may be different for different patient groups."

Sanofi and Regeneron obtained an FDA priority review voucher from BioMarin Pharmaceuticals last year for $67.5 million to hasten the regulatory assessment of Praluent, which was awarded priority review status in January for patients with hypercholesterolaemia. A final decision on approval of the drug is expected by July 24.

Meanwhile, an FDA advisory committee is scheduled to review Repatha on June 10, with a final decision on whether or not to approve the therapy expected by August 27. For related analysis, see ViewPoints: FDA briefing documents reveal notable differences between anti-PCSK9 mAbs.

Analysts have suggested that Praluent, which is also under regulatory review in Europe, could be priced at about $1000 per month, generating revenue of $1.9 billion in 2020. Meanwhile, Repatha is predicted to garner sales of $2.5 billion by 2020. Earlier this year, CVS Health estimated that the PCSK9 inhibitor class could increase US healthcare costs by as much as $150 billion per year (for related analysis, see Spotlight On: How are PBMs preparing for the impending anti-PCSK9 bonanza?).

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