FDA Panel Backs Amgen's Repatha, But With Strings Attached

Published: Jun 15, 2015

FDA Panel Backs Amgen's Repatha, But With Strings Attached
June 11, 2015
By Mark Terry and Riley McDermid, BioSpace.com Breaking News Staff

The U.S. Food and Drug Administration (FDA)’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted unanimously to approve the use of Amgen ’s Repatha (evolocumab) yesterday, but then backpedaled by indicating it should only be used for a rare type of high-risk disease.

The panel voted 15-0, recommending that Repatha should be approved to treat homozygous familial hypercholesterolemia (HoFH). HoFH is a rare genetic disorder that results in extremely high levels of cholesterol. It has been known to cause heart attacks in children.

In addition, the panel voted 11-4, recommending Repatha be approved for other uses, primarily heterozygous familial hypercholesterolemia (FH), a similar, but less rare genetic disorder.

Analysts project that Praluent could generate $1.9 billion for Sanofi in 2020 and Repatha could generate $2.5 billion for Amgen in the same period. You can see our analyst wrap-up on the decision here.

A major concern of the panel is apparently that, even though the drug appears very effective at lowering the so-called bad cholesterol, LDL, they want evidence that the drug also shows evidence that using the drug will decrease incidents of heart attacks and strokes. This would typically require a larger outcomes trial.

Quoted in a Forbes live blog by Matthew Herper, panelist Martha Nason said, “I, like everyone else around the table, believe the cardiovascular trial is very important. My concern would be that if this were available fairly widely that really would hamper the ability to follow those people and to really get your answer.”

The FDA doesn’t have to follow the advice of its advisory panels, but usually does. It is expected to make a final decision this summer.

Repatha is a class of drugs calls PCSK9 inhibitors. PCSK9 is a protein that targets LDL receptors for degradation, which decreases the liver’s ability to remove LDL-C, the bad cholesterol. Repatha binds to PCSK9 and inhibits it from binding to LDL receptors. As a result, there are more LDL receptors on the surface of the liver to help remove LDL-C from the blood.

The drug is an injectable, and is designed to help patients who can’t lower their LDL with statins, such as Pfizer Inc. ’s Lipitor.

Repatha may be beaten to the market by another PCSK9 drug, Praluent, from Sanofi and Regeneron Pharmaceuticals, Inc. . The FDA will make a decision on Praluent on July 24. The FDA will not decide on Repatha until Aug. 27.

Overall, the FDA advisory panel voted yesterday 13-3 on Praluent, indicating that its benefits outweighed its risks. In the case of Praluent, the panel suggested limiting approval to heterozygous familial hypercholesterolemia, which affects about 1 in 500 people.

Geoffrey Porges, an analyst with Sanford Bernstein, said the FDA appeared to follow the road map it used earlier this week when approving Regeneron Pharmaceuticals, Inc. (REGN)’s similar drug Praulent.

Porges said the criticism Amgen faced from the panel has also put Wall Street on notice that safety data needs to be flawless before approval is sought.

Amgen did sustain some criticism from the agency and its advisor about the conduct and duration of their Phase III trials, allegedly lacking sufficient safety data beyond the 12 week endpoint used in most of the company's trials,” he wrote in a note to investors.

FDA staff and advisers still appear to be enamored with REGN's 2-dose step up offering, and criticized Amgen for their failure to develop and study a lower dose. We expect both stocks to be volatile at least until their late summer launches”

In a statement made by pharmacy benefit manager Prime Therapeutics LLC, which is owned by 13 Blue Cross and Blue Shield plans, PCSK9 inhibitors could cost the U.S. health care system up to $23.3 billion annually.

“Two PCSK9 inhibitors—Praluent from Regeneron & Sanofi, and Repatha from Amgen—are expected to be approved by the U.S. Food and Drug Administration this summer. Unlike traditional statins, which are oral medicines and have inexpensive generic alternatives, PCSK9 inhibitors are self-injected monoclonal antibodies.

These drugs act by preventing the protein PCSK9 from interfering with the liver LDL removal process, allowing more LDL cholesterol removal from the blood.”

Although pricing is speculative, Prime Therapeutics estimated that prices could range from $7,000 to $12,000 per year.

When Will Pfizer's Breakup Happen?
Speculation that the revamping of Pfizer Inc. ’s internal business structure could happen as soon as this year has biotech wondering just when this Big Pharma company could see changes.

Last week an analyst with J.P. Morgan said he thinks there will be a much faster timeline than most of Wall Street had predicted for Pfizer’s stated mission to refocus its efforts on new medicines.

Pfizer initially announced in 2012 that it would be shedding units that were non-essential to that goal. It then promptly sold its nutrition silo to Nestle for $11.85 billion, which was rapidly accompanied by a public spin-off of its animal health business for $2.2 billion.

“While a Pfizer break-up would likely be a 2017 event, we see potential catalysts in 2015-2016," said Chris Schott, an analyst at J.P. Morgan. "Three years of audited financial statements (2014-2016) are required before any part of Pfizer can be spun off, and we also see 2017 as an attractive time for action as investors see Pfizer’s innovative pipeline clearly contributing to growth and the established business having transitioned to a more stable profile."

BioSpace wants to know what you think: Will Pfizer be a changed company by the end of 2015?

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