Amgen has submitted a biologics license application (BLA) to the US Food and Drug Administration (FDA) for evolocumab to treat high cholesterol. Evolocumab is an investigational fully human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9), a protein that reduces the liver’s ability to remove low-density lipoprotein cholesterol (LDL-C), or “bad” cholesterol, from the blood.

In addition to Amgen, other companies have late-stage PCSK9 inhibitors for treating high cholesterol in development. Sanofi and Regeneron Pharmaceuticals, Inc. plan to submit their applications for alirocumab, their PCSK9 inhibitor to lower LDL-C, to US and European Union regulatory authorities by the end of this year. In June 2014, the companies acquired a rare pediatric disease priority review voucher in connection with the BLA submission for alirocumab in the US. The priority review voucher entitles the holder to designate a BLA for priority review, which provides for an expedited 6-month review from the filing date instead of the standard 10-month review. The companies purchased the voucher from BioMarin GALNS Ltd., a direct, wholly owned subsidiary of BioMarin Pharmaceutical, Inc., which had received it through the FDA's Rare Pediatric Disease Priority Review Voucher (PRV) Program.The Pediatric PRV was created by the 2012 Food and Drug Administration Safety and Innovation Act and is intended to encourage the development of treatments for rare pediatric diseases. Companies that receive a voucher may use it or transfer the voucher, including by sale, to other organizations. BioMarin had received the voucher in connection with FDA approval of its orphan drug, Vimizin (elosulfase alfa), an enzyme-replacement therapy for treating Mucopolysaccharidosis Type IVA (Morquio A). Pfizer is also developing a PCSK9 inhibitor, bococizumab, to treat hyperlipidemia, which is Phase III clinical development.

In other developments, Amgen has been granted priority review designation by the FDA for ivabradine, investigational oral cardiovascular drug that inhibits the I_f current (“funny” current) in the sinoatrial node, the body’s cardiac pacemaker. Ivabradine works to slow the heart rate without negative effects on myocardial contractility or ventricular repolarization. Developed by Les Laboratoires Servier, ivabradine was approved by the European Medicines Agency Procoralan in 2005 for the symptomatic treatment of stable angina and in 2012 for chronic heart failure in patients with elevated heart rates. Through a collaboration with Servier, Amgen has rights to commercialize ivabradine in the US.

Source: Amgen (evolocumab) and Amgen (ivabradine)