July 21, 2015
By Mark Terry, BioSpace.com Breaking News Staff
Thousand Oaks, Calif.-based Amgen announced today that its drug, Repatha (evolocumab), for uncontrolled cholesterol, has been given marketing authorization by the European Commission (EC).
Repatha is the first proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor to be approved anywhere. The drug is a human monoclonal antibody that inhibits PCSK9, which is a protein that slows down the liver’s ability to remove LDL-C, or “bad” cholesterol.
The approval will allow Amgen to market the drug in 28 countries in the European Union. As members of the European Economic Area (EEA), Norway, Iceland and Liechtenstein will follow along with the EC’s decision, typically.
“We are proud that our cholesterol-lowering medication, Repatha, is the first PCSK9 inhibitor to be approved by any regulatory agency in the world,” said Sean Harper, executive vice president of Amgen’s research and development in a statement. “High LDL cholesterol is a major global health burden and many patients are unable to appropriately control their LDL cholesterol with the maximum tolerated dose of a statin, or are unable to take statins due to intolerance or contraindications. We are excited to make this new cholesterol-lowering medication available for patients in Europe.”
Repatha is a bit on the fence in terms of approval in the U.S. On July 11, 2015, the U.S. Food and Drug Administration (FDA)‘s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted unanimously to approve Repatha, but then waffled by indicating it should only be used for a rare type of high-risk disease.
The panel voted 15-0, recommending Repatha should be approved to treat homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder. It also voted 11-4, recommending Repatha be approved for other uses.
Repatha is a new class of drugs called PCSK9 inhibitors. Repatha is injected, and is designed to assist patients who can’t lower their LDL with statins, such as Pfizer Inc. ’s Lipitor.
This approval in Europe means Amgen’s Repatha beat competitors Sanofi and Regeneron Pharmaceuticals, Inc. to market — but not necessarily in the U.S. The FDA is expected to make a decision on those companies’ product, Praluent, on July 24. The FDA is not expected to make a decision on Repatha until Aug. 27.
Analysts believe Praluent could create $1.9 billion for Sanofi in 2020 and Repatha could generate $2.5 billion for Amgen in the same period.
Geoffrey Porges, an analyst with Sanford Bernstein, indicated in a research note that Amgen took some criticism from the FDA over certain aspects of its Phase III trials and some of its safety data. “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.”
Emphasizing just how important the race for this market is, Sanofi and Regeneron spent $67.5 million on a priority review voucher. The voucher was held by BioMarin Pharmaceutical Inc. , which was awarded the voucher early in 2014 by the FDA to encourage development of drugs for rare pediatric diseases. The holder of the voucher gets priority review of a drug application by the FDA, which can shorten the review process by six months as opposed to the typical 10 months.
BioMarin received the voucher related to Vimizim, a drug for a rare pediatric condition called Morquio A syndrome. The program takes into account that companies may prefer the cash created by selling the voucher to other companies, which is what happened in this situation. Regeneron indicated it was using it to attempt to stay in the race with Amgen, and possibly beat them to market.
Amgen has declined to discuss pricing or marketing plans for Repatha.
