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Praluent and Repatha backed for approval in US

Sanofi and Amgen remain in fierce competition in PCSK9 inhibitors

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After two days of FDA advisory committee meetings, Sanofi and Amgen remain level pegging on their PCSK9 inhibitor programmes - both should be approved, but in narrow patient populations, according to panellists.

On Tuesday, the panel voted 13 to 3 in support of approval of Sanofi and Regeneron's Praluent (alirocumab), but most were in favour of a restricted label in high-risk patients with genetic conditions causing elevated cholesterol rather than wider use in the general elevated cholesterol population. 

For Amgen's Repatha (evolocumab), the panel voted unanimously in favour of approval in the genetic condition homozygous familial hypercholesterolaemia (hoFH) and 11 to 4 that it could be used in other indications such as the more common heterozygous familial hypercholesterolaemia (heFH). Once again however there were reservations about approving the drug in the broader cholesterol patient population.

The companies are hoping for approval for more general use in patients with elevated cholesterol who cannot tolerate statins or who cannot achieve their LDL-c targets on statin therapy alone. Amgen's drug has already been recommended for approval in Europe for hoFH as well as this larger target population.

Both Praluent and Repatha have achieved dramatic reductions in low-density lipoprotein-cholesterol (LDL-c) in clinical trials but - as expected given comments in the FDA's briefing documents - much of the discussion during the panel meetings focused on the validity of LDL-c as a surrogate marker for cardiovascular outcomes. 

On this topic panellists seemed to be split and this underpinned the diverging views on wider use of the drugs, and it was pointed out during voting that after approval on the basis of LDL-c there is currently no regulatory requirement to demonstrate an effect on outcomes.

Some panellists indicated they would prefer to wait for hard cardiovascular outcomes data from trials - which are ongoing but not due to report results for two years - before approval in patients at lower risk. 

The FDA still has to complete its review of both drugs and is not required to take the advice of its advisory committees, although it often does so. One possible scenario is initial approval in a limited population - likely hoFH and heFH - with broader use in the statin-intolerant and unresponsive population following after, once outcomes data is in hand.

A report from pharmacy benefits manager (PBM) Prime Therapeutics released ahead of the panel meetings said that the hoFH and heFH population is around 200,000 people in the US. In contrast, the number of people in the US who are statin intolerant or fail to achieve treatment goals on these drugs could be as high as 2.3 million.

Narrower approval would suggest that earlier projections of peak sales in the multibillion-dollar range for both products might at the very least take a little longer to come to fruition. GlobalData previously predicted that the PCSK9 inhibitor class as a whole could capture a 40% share of the $17.5bn acute coronary syndromes market by 2023.

The FDA's review date for Praluent is July 24 - accelerated thanks to Sanofi's purchase of a priority review voucher from BioMarin last year - while the agency is due to decide on Repatha by August 27. 

Article by
Phil Taylor

11th June 2015

From: Sales



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