EC Approves Amgen’s New Cholesterol Drug
The European Commission has granted marketing authorization for Amgen’s for Repatha (evolocumab), a proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor for treating uncontrolled cholesterol in patients that require additional intensive low-density lipoprotein cholesterol (LDL-C) reduction. Repatha is a human monoclonal antibody that inhibits PCSK9, a protein that reduces the liver’s ability to remove LDL-C, or “bad” cholesterol, from the blood.
Approval from the EC grants a centralized marketing authorization with unified labeling in the 28 countries that are members of the EU. Norway, Iceland, and Liechtenstein, as members of the European Economic Area (EEA), take corresponding decisions on the basis of the decision of the EC.
The EC approval marks the first regulatory approval for a PCSK9 inhibitor, a potential blockbluster class of drugs, for treating cholesterol. Repatha, developed by Amgen scientists, is designed to bind to PCSK9 and inhibit PCSK9 from binding to LDL receptors on the liver surface. In the absence of PCSK9, there are more LDL receptors on the surface of the liver to remove LDL-C from the blood.
Repatha is under regulatory review by the US Food and Drug Administration (FDA) with a target action date of August 27, 2015. Amgen is partnered with Astellas Pharma for Repatha in Japan as part of a multi-drug collaboration the companies formed in 2013. The long-term collaboration focuses on the co-development and co-commercialization in Japan of five Amgen pipeline medicines (which included the now approved Repatha). and also created a Tokyo-based joint venture company, Amgen Astellas BioPharma KK. Sanofi/Regeneron Pharmaceuticals also have a PCKS9 inhibitor under regulatory review and Pfizer has a drug in late-stage development. Regeneron/Sanofi recently received a recommendation for approval by a FDA advisory committee for their investigational anti-cholesterol drug, Praluent (alirocumab) injection. The BLA for Praluent was accepted for priority review by the FDA with a target action date of July 24, 2015. The marketing authorization application for Praluent in the European Union is currently under review by the European Medicines Agency.
A a recent Thomson Reuters analysis pegs both drugs as potential blockbusters. Based on estimates for 2019 sales, the analysis puts potential revenues at Regeneron Pharmaceuticals and Sanofi's Praluent (alirocumab) at $4.4 billion, and Amgen's/Astellas’ evolocumab at nearly $1.9 billion by 2019. Pfizer also has a late-stage PCSK9 inhibitor, bococizumab, which is now in a Phase III clinical trial program.