FDA approves Sanofi, Regeneron's PCSK9 inhibitor Praluent

Sanofi and Regeneron Pharmaceuticals announced that the FDA approved Praluent (alirocumab) for certain patients with high cholesterol, making it the first PCSK9 inhibitor cleared in the US. Specifically, the drug is indicated as an adjunct to maximally tolerated statin therapy for adults with heterozygous familial hypercholesterolaemia or clinical atherosclerotic cardiovascular disease (ASCVD), who require additional lowering of LDL cholesterol. Last month, an FDA advisory panel backed approval of the cholesterol therapy, although some committee members suggested that the drug should be limited to certain patients such as those with familial hypercholesterolaemia.

Approval of Praluent was supported by findings from the Phase III Odyssey clinical trial programme. In documents released ahead of the advisory panel vote, FDA researchers determined that the drug significantly lowered LDL cholesterol levels, while questions concerning the risk-benefit ratio of the therapy were raised. Meanwhile, research data published earlier this year in the NEJM illustrated that Praluent, as well as Amgen's anti-PCSK9 therapy Repatha (evolocumab), decreased the risk of cardiovascular events by about 50 percent while reducing LDL cholesterol levels by more than 60 percent. Moreover, a large study aiming to demonstrate whether reductions in LDL cholesterol levels improve patient outcomes for both therapies is ongoing and scheduled for completion in 2017.

Sanofi CEO Olivier Brandicourt stated "we look forward to working with other regulatory authorities to make Praluent available to patients worldwide." Also on Friday, the European Medicines Agency's Committee for Medicinal Products for Human Use recommended approval of the therapy for cholesterol reduction in patients intolerant or unresponsive to statins.

Meanwhile, authorities in Europe approved Repatha earlier this week, marking the first clearance of a PCSK9 inhibitor anywhere in the world. An FDA panel recommended approval of the therapy last month, with the US agency expected to issue a final decision regarding whether to authorise the drug by August 27.

Some pharmacy benefit managers have expressed concerns regarding the potential prices of PCSK9 inhibitors, with Sanofi and Regeneron planning to price the therapy at $14 600 a year. "This could become the most expensive medication that we use," said Troyen Brennan, chief medical officer at CVS Health, which previously estimated that PCSK9 inhibitors could cost the healthcare system $150 billion annually. Conversely, Sanofi research head Elias Zerhouni countered "it's not going to be the big shock to the system that other people are predicting", also nothing "we came to a price that is reflective of value, not what the market will bear," (for additional analysis, see Spotlight On: How are PBMs preparing for the impending anti-PCSK9 bonanza?).

Analysts estimate Praluent may generate sales of $1.1 billion in 2018 while Repatha may garner $1.36 billion.

For related analysis on the PCSK9 inhibitor class, see ViewPoints: Europe provides positive regulatory momentum for PCSK9 inhibitor class, but regional cardiologists likely to prescribe less than US counterparts and ViewPoints: Label for newly approved Repatha in EU will have US payers holding their breaths.

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