UPDATED: NICE, in reversal, OKs Amgen's Repatha while snubbing Sanofi and Regeneron's Praluent

Amgen's ($AMGN) Repatha is in a market brawl with Sanofi ($SNY) and Regeneron's ($REGN) Praluent as the competitors rush to sew up as many OKs around the world as possible for the new-gen cholesterol fighters following their FDA approval last year within weeks of one another. Now Amgen has pulled ahead in England after the country's drug pricing watchdog has both changed course to cover Repatha for some patients, while dealing its competitors a blow with a rejection for Praluent.

The so-called PCSK9 meds are expected to be big sellers worldwide as a new approach to lowering persistently high bad cholesterol but England's National Institute for Health and Care Excellence (NICE) is not making it easy for them to break into the market there. Today NICE approved Amgen's Repatha for use against a number of specific conditions in which patients have persistently high LDL cholesterol, PharmaTimes reports. While the decision was a reversal of last year's complete rejection of Repatha for use in the U.K, it was not as broad as Amgen had hoped.

Tony Patrikios, Amgen's executive medical director in the U.K. and Ireland, urged NICE to think again about expanding the drug's patient pool, according to PharmaTimes. "NICE has reviewed the technical responses to its initial appraisal, listened to patients and healthcare professionals and proposed a recommendation for the use of Repatha by the NHS in England and Wales," Patrikios said. "However, beyond these draft recommendations, another priority will be patients with existing cardiovascular disease who fail to reduce their LDL-C to a satisfactory level despite the use of statins and who remain at high risk. For them, access to new treatment options is critically important to help avoid a heart attack or stroke."

NICE will allow Repatha's use in patients with primary non-familial hypercholesterolaemia or mixed dyslipidemia with progressive cardiovascular disease (CVD), who also have persistently high LDL-C concentrations; in patients with primary heterozygous-familial hypercholesterolaemia or mixed dyslipidemia with progressive, symptomatic CVD and persistently high LDL-C; and in those with severe primary heterozygous-familial hypercholesterolaemia or mixed dyslipidemia without CVD, if they continue to have high levels of cholesterol despite maximum lipid-lowering therapy and can't take statins. Today's approval is contingent on Amgen offering a discount to patients through its patient access program.

Last November, in preliminary guidance, the gatekeeper had rejected Repatha, based both on cost and effectiveness. At the time, NICE said that while the drug was effective in reducing bad cholesterol levels, there was no proof that it can actually prevent angina, heart attacks and strokes. Amgen hopes to have data available later this year that will show that link.

While Amgen didn't get all that it wanted, Sanofi and Regeneron got nothing at all from the price gatekeeper. It was preliminary draft guidance, so there could yet be a reversal, but today NICE said no to Praluent's use. It based its decision in part on the fact that the drugmakers have evaluated the drug "in combination with use of ezetimibe plus a statin, in the large population of people with non-familial hypercholesterolaemia." Ezetimibe is Merck's ($MRK) Zetia. Evaluators also were concerned about the drug's price.

In an emailed statement, Sanofi said it was disappointed in NICE's decision but that it was committed to working with the agency during a reconsideration to make Praluent available in England. It said it was pleased that NICE recognized Sanofi's intention to focus on the patient subgroups at risk for further cardiovascular events. Sanofi even gave a nod to its competitor. Noting that NICE had approved Repatha in a second consideration, Sanofi said it "is pleased that the Institute has acknowledged the value of the PCSK9 inhibitor class and the potential health and economic value these medicines may provide to the NHS."

It is a tough blow for Sanofi and Regeneron as they duke it out with Amgen in the new category. Last month, the partners saw Amgen's Repatha get the jump as the first PCSK9 approved in Japan.

In the U.S., where both are approved, the fight has been playing out as each has been trying to nail down preferred deals with pharmacy benefit managers who have pitted the drugmakers against one another to extract discounts for the PCSK9 cholesterol fighters. Express Scripts ($ESRX) agreed to offer both Praluent and Repatha on its formulary in exchange for discounts from both, while Praluent will be UnitedHealth Group's ($UNH) "preferred" drug for treating superhigh LDL cholesterol. Amgen nailed a formulary exclusive with CVS ($CVS).

- read the PharmaTimes Repatha story
- more from PharmaTimes on Praluent
- get NICE documents on Repatha here
- here's NICE's draft guidance on Praluent

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