Amgen ($AMGN) is likely to come in second in the race to launch new cholesterol treatments in the U.S., but the Big Biotech is leading the charge in Europe, securing a crucial regulatory recommendation that clears the way for approval.
The company's treatment, evolocumab, is an antibody designed to block the protein PCSK9 and thereby help the body clear excess LDL, or bad, cholesterol from the blood. The European Medicines Agency's Committee for Medicinal Products for Human Use, poring over data from Amgen's expansive Phase III program, has recommended the injection be approved to treat severe high cholesterol. That recommendation now goes to the European Commission, which typically hands down a final approval within about three months.
That puts Amgen on track be the first PCSK9 contender in Europe, leading rival partners Sanofi ($SNY) and Regeneron ($REGN) and their alirocumab, submitted to the EMA in January.
In the U.S., however, Sanofi and Regeneron leapfrogged Amgen by buying a voucher for a speedy FDA review, lining up for a likely July approval. Amgen's antibody is in line for a final FDA decision by Aug. 27. Pfizer ($PFE), still working through Phase III with its similar bococizumab, is angling for third place in both markets.
Each treatment has the potential to bring in as much as $3 billion a year at its peak, analysts have said, but such lofty projections likely hinge on success in some ongoing outcomes trials. Each PCSK9 antibody has demonstrated marked effects on lowering bad cholesterol, but whether doing so can substantially reduce the risks of heart attack and other deadly events remains the subject of debate. Now, evolocumab, alirocumab and bococizumab are in the midst of huge long-term studies to suss out their overall benefits, and the ultimate success of the PCSK9 class likely depends on how well they hold up.
In the meantime, the two leading PCSK9 factions are preparing for a near-term fight to differentiate themselves as they march toward the market. Express Scripts ($ESRX), the pharmacy benefit manager that successfully spurred a price war among competing hepatitis C therapies last year, has identified PCSK9 blockers as its next target, figuring it can squeeze some savings by pitting fairly comparable drugs against one another. None of the companies has explicitly discussed expected pricing for the injected therapies, but it's sure to be contentious topic come July.
- read the statement (PDF)