Sanofi ($SNY) and Regeneron ($REGN) may be on pace to win the first U.S. approval for a new type of cholesterol treatment, but the pair trails rival Amgen ($AMGN) in Europe in a race to make good on blockbuster sales predictions.
The European Medicines Agency has recommended approval for Sanofi and Regeneron's Praluent (alirocumab), an antibody designed to block the protein PCSK9 and thereby help the body clear excess LDL, or bad, cholesterol from the blood. That recommendation now goes to the European Commission, which typically hands down a final approval in two to three months.
Amgen, maker of the similar Repatha (evolocumab), won continental approval earlier this week, giving it first-mover status in Europe. Each treatment is slated to carry a label indicating it for use in patients who can't get to their target cholesterol levels on cheap statins alone, patients who can't or won't take those generic drugs, and patients suffering from a pair of genetic diseases that lead to uncontrollable LDL.
Meanwhile, in the U.S., Sanofi and Regeneron have leapfrogged Amgen by buying a priority review voucher that moved up their expected FDA decision date. The agency has promised to hand down a yes or no on Praluent by Friday, while Amgen's Repatha isn't up for approval until Aug. 27.
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.
PCSK9 antibodies have 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. Both Repatha and Praluent are in the midst of large, long-term studies to determine out their overall benefits, and the ultimate success of the PCSK9 class likely depends on how well they hold up.
And uncertainties about the ultimate utility of PCSK9 therapies could have a major effect on market uptake, especially in the U.S.
In a pair of panel meetings last month, a group of FDA advisers voiced serious concerns about how little is known about PCSK9 treatments. Many cardiologists are skeptical about whether the so-called statin-intolerant population is as large as it might seem, and the panel was iffy about recommending approval in that indication out of fear that it could lead to over-prescription of antibodies the long-range effects of which are yet to be established. The FDA, unlike its European counterparts, may leave those patients off of its PCSK9 labels, which could have a read-through to peak sales.
- read the statement (PDF)