FDA staff consider suitable patients groups for Amgen's PCSK9 inhibitor Repatha

In documents released Monday ahead of an FDA advisory committee meeting on June 10, agency staff suggested that Amgen's PCSK9 inhibitor Repatha (evolocumab) can significantly lower LDL cholesterol. However, the reviewers questioned which patient groups are suitable for the injectable therapy and whether to approve it based on currently-available data.

Last week, FDA staff released a similar review on Sanofi and Regeneron Pharmaceuticals' PCSK9 inhibitor Praluent (alirocumab) ahead of an advisory panel meeting on June 9. The regulator is expected to issue a final decision on approval of Praluent by July 24 and on Repatha by August 27.

In study data published in March, Repatha was shown to lower LDL-C by 61 percent compared to placebo, as well as reduce the risk of cardiovascular (CV) events by 50 percent. Amgen has studied the drug in several different patient groups, including those already taking statins, those who cannot take statins due to side effects and in patients with heterozygous familial hypercholesterolaemia. At the advisory committee meeting, the panel will consider which patients are most likely to benefit from the drug, considering potential risks seen in studies, including higher rates of pancreatitis and kidney problems.

Both Repatha and Praluent are being investigated in large-scale efficacy trials designed to determine whether the drugs' effects on LDL-C levels translate into improve CV outcomes, with results anticipated in 2017. FDA staff said that preliminary results from a CV outcomes study of Amgen's therapy showed adverse side effects on the liver, although patients taking placebo also experienced them, which reviewers noted was "reassuring."

In the US, Amgen filed a lawsuit last year accusing Sanofi and Regeneron of infringing its patents for Repatha. Sanofi and Regeneron have claimed that the allegations lack merit. Meanwhile, Repatha has also been recommended for approval by European regulators for patients with high cholesterol.

Analysts at Sanford C. Bernstein & Co. suggest that PCSK9 inhibitors could be priced at around $1000 per month, with Goldman Sachs analysts estimating that the drug class could generate as much as $10 billion in annual revenue. Meanwhile, CVS Health has previously suggested that the drugs could increase healthcare costs in the US by as much as $150 billion per year (for related analysis, see Spotlight On: How are PBMs preparing for the impending anti-PCSK9 bonanza?).

For additional insight 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 Physician Views Poll Results: How will EU5 cardiologists use the PCSK9 inhibitors?

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