New cholesterol products carry high stakes for drugmakers

Approval by the Food and Drug Administration of the new PCSK9 inhibitors would be an enormous boon to the drugmakers that developed them.

According to Karen Andersen, a senior analyst covering biotechnology for Morningstar, a Chicago-based independent investment-research firm, here's what's at stake for the manufacturers seeking FDA approval of the new drugs.

Paris-based Sanofi is partnering with Tarrytown, N.Y.-based Regeneron Pharmaceuticals to develop alirocumab. Sanofi has been “struggling for growth,” Andersen said. Its patent on Lantus, a top-selling insulin with more than $7 billion in sales worldwide in 2014, runs out this year, with makers of biosimilars itching to challenge its franchise.

“They're seeing less ability to raise prices on their diabetes drugs and more competition,” she said. “Alirocumab would be a great growth driver for their branded-drug division.”

Regeneron faces less pressure than Sanofi. Its flagship drug Eylea, injected to treat age-related macular degeneration, a major cause of blindness, as well as macular edema, has been a winner with worldwide sales of $1.7 billion in 2014. Moreover, Regeneron has “a very strong pipeline and Eylea is gaining market share,” she said.

Thousand Oaks, Calif.-based Amgen sees evolocumab as a major revenue replacement for Neupogen, which is used to prevent infections during chemotherapy and also faces competition from biosimilars. Last month, the FDA and a U.S. District Court in San Francisco both ruled against Amgen's efforts to block Swiss drugmaker Sandoz from launching a biosimilar competitor, Zarxio.

“PCSK9 inhibitors are definitely an important market for Amgen,” Andersen said. “They need to see growth from their pipeline to counter some of that biosimilars competition.”

Amgen initially had the lead in the race to market a PCSK9 cholesterol-reduction drug. But the Sanofi/Regeneron team closed the gap after paying $67.5 million to buy an FDA priority review voucher for a rare pediatric disease review from BioMarin Pharmaceutical of San Rafael, Calif., which had developed a biologic product to treat a rare pediatric disease known as mucopolysaccharidosis type IVA. Once the FDA issues such vouchers for rare pediatric diseases, they can be resold to be used for priority reviews for other products.