Amgen has filed its eagerly-watched cholesterol drug evolocumab in the USA, the first PCSK9 inhibitor to be submitted to regulators.

The filing with the US Food and Drug Administration contains data from 6,800 patients, including more than 4,500 patients with high cholesterol in ten Phase III trials. Evolocumab has shown high levels of efficacy in patients with elevated cholesterol on statins with or without other lipid-lowerers, those who cannot tolerate statins and those patients with heterozygous familial hypercholesterolemia (HeFH) and homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder.

Amgen R&D chief Sean Harper said the submission marks the first of several to regulators around the world “for our lipid-lowering programme and represents a critical milestone in our overall global development programme for evolocumab. He believes it will be a new treatment option for patients who, “despite currently available therapies, are unable to adequately reduce their LDL cholesterol levels.”

Along with the US filing, Amgen also unveiled positive top-line results from a Phase III study of evolocumab in combination with statins in Japanese patients with high cardiovascular risk and high cholesterol. The 404-patient trial met its co-primary endpoints of LDL cholesterol reduction.

Race with alirocumab

Evolocumab belongs to the closely-watched PCSK9 class, a protein that reduces the liver's ability to remove bad cholesterol, from the blood. It is in a race with Sanofi/Regeneron’s alirocumab to see which will first get to market.

In an investor note, UBS analyst Matthew Roden wrote that the timing of the filing, while in line with third-quarter guidance, was “slightly sooner than expectations and answers the question as to which company is first to file among PCSK9s”. He expects Amgen to get priority review status, adding that if first to market, the company “would be in the position to set the price and establish a nominal first mover advantage, although we expect…alirocumab to be a fast follower”.

Mr Roden concludes by saying “it is also plausible that if alirocumab is filed in the near term, the FDA could convene a single advisory committee meeting, in effect harmonising the timing of approvals”.