UPDATED: Amgen beats Regeneron, Sanofi to world's first big PCSK9 drug OK

Amgen's Sean Harper

Amgen ($AMGN) may be running a beat or two behind Regeneron ($REGN) and Sanofi ($SNY) in search of the first FDA approval for a PCSK9 cholesterol drug, but the Big Biotech scooped up the very first approval in Europe today for Repatha (evolocumab), giving the company boasting rights as first mover in what is widely seen as a new blockbuster market in the making.

Amgen ran a lineup of late-stage studies with more than 4,000 patients to prove that Repatha worked as billed, inhibiting the PCSK9 protein and blocking a mechanism that prevented the liver from flushing bad LDL cholesterol from the blood. The dramatic effects in clinical studies--mirrored in a rival therapy advanced by Regeneron and its close partner Sanofi--excited analysts with its prospects in a market in which large percentages of patients can't adequately control cholesterol levels with the cheap statins now on the market.

Notably, Amgen gets credit for winning a broad approval for their drug covering a large segment of the European market, including patients resistant to statins.

There's no such thing as a sure thing in drug development, a risky endeavor threatened constantly by unexpected adverse events or surprising regulatory setbacks, but all three big players have been playing this game with great expectations of the revenue to come. Amgen and Sanofi in particular badly need new blockbuster approvals to deliver the numbers investors are expecting.

The big regulatory news is expected later this week, with the FDA set to announce its decision on Praluent from Regeneron and Sanofi. Amgen is facing an August 27 PDUFA date in the U.S. Reuters is reporting that Praluent may well get a CHMP recommendation in Europe later in the week, putting Sanofi and Regeneron a couple of months behind Amgen in the hunt for a formal European Commission OK. Underscoring just how competitive a race this is, Sanofi and Regeneron bought a priority review voucher just to leapfrog ahead of Amgen in the U.S. 

Sanofi and Regeneron are already laying plans to launch their drug with low and high doses of 75 mg and 150 mg while Amgen has been prepping a 140-mg injection, with a 420-mg dose that can be injected once a month. That low dose is expected to be offered at a market-making discount that may be particularly attractive to patients. 

Amgen wasn't providing any clues about pricing today. A spokesperson for the company tells FierceBiotech that no price has yet been set.

"Many patients who are taking cholesterol-lowering therapies, including those with familial hypercholesterolemia, still struggle to control their LDL cholesterol levels," said John J.P. Kastelein, professor of medicine and chairman of the Department of Vascular Medicine at the Academic Medical Center of the University of Amsterdam, in a statement. "As the first in a new class of drugs in the European Union, evolocumab will offer physicians an important and innovative treatment option for patients with uncontrolled cholesterol who require additional LDL cholesterol reduction."

Outside experts at the FDA have been happy to support Repatha and Praluent for hypercholesterolemia, a relatively rare genetic condition that makes it extremely hard to control cholesterol. But there have been concerns that these new drugs could start to win over doctors and large numbers of mainstream patients before they fully exhaust an effort with statins. That was clearly on the FDA committee members' minds during their reviews last month, raising the prospect that the FDA could set up a regulatory roadblock that would limit these drugs' market potential.

"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 Dr. Sean Harper, executive vice president of R&D at Amgen.

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