US regulators have approved Sanofi and Regeneron’s PCSK9 inhibitor Praluent for the treatment of high cholesterol, but for a narrower patient population than initially hoped.

Specifically, the US Food and Drug Administration has waved through the jab’s use alongside diet and maximum statin therapy in adults with heterozygous familial hypercholesterolaemia, or those with atherosclerotic cardiovascular disease such as heart attacks or strokes, who need additional lowering of LDL cholesterol.

But the regulator stopped short of clearing Praluent’s (alirocumab) use for patients deemed intolerant to statin therapy, because of uncertainty over the long-term benefit-risk equation for this group, heeding the concerns of its advisory committee.

Nevertheless, bagging first approval of a PCSK9 inhibitor in the country means that Praluent is also likely to be the first launched onto the market, beating Amgen’s rival biologic Repatha (evolocumab) to the post. Repatha is, however, hot on its tail, having already secured the first European approval (including statin intolerant patients) and expecting a US nod next month.

Analysts are largely expecting peak sales of around $2 billion-$3 billion for each of the top PCSK9 inhibitors coming onto the market, although uptake could dampened by pricing. According to FiercePharma, Sanofi is looking to charge around $40 a day for Praluent, or $14,000 a year (wholesale), which, given that patients will be switching from cheap generics and taking the drug over a lifetime, could have quite a financial impact.

EU recommendation

Meanwhile, European regulators advisors have also backed the use of Praluent for the treatment of hypercholesterolaemia.

However, unlike its US peers, the Committee for Medicinal Products for Human Use has given its blessing for use of the drug to treat those intolerant to statin therapy as well as those with primary hypercholesterolaemia and mixed dyslipidaemia, thereby opening the door to a much, much larger patient pool.