[Updated and corrected 8/27/15, 7:28pm, see below.] The new race to fight cholesterol is on. For the second time in a month, the U.S. Food and Drug Administration has approved a drug for people with dangerous levels of cholesterol who aren’t getting enough help from statins, which have long been among the world’s most prescribed medicines.
The agency said Thursday that doctors can prescribe evolocumab (Repatha), made by Thousand Oaks, CA-based Amgen (NASDAQ: AMGN), for certain people whose high cholesterol is not being lowered enough by statins and a change in diet.
Just as it did one month ago for a similar drug, alirocumab (Praluent), the FDA has given doctors a wide berth to decide which patients among potentially millions are eligible for the drug. In its press release this afternoon, the FDA said evolocumab could be used in patients who have inherited a condition that gives them dangerously high levels of cholesterol, as well as in patients with a history of heart disease who aren’t being helped enough by statins.
[This paragraph updated to include payment language and Amgen comments.] Amgen has set an annual price of $14,100. But it also said in a press release that the net price of evolocumab would be linked to cholesterol reductions and “anticipated appropriate patient utilization.” Amgen officials did not hold a conference call to discuss the news. When asked whether the language meant Amgen would be paid on a sliding scale based on how well evolocumab worked, or how well patients stayed with their injection schedule, a spokeswoman said the company had no comment beyond what was in the press release. She also declined to say when more details would be available.
When alirocumab gained approval in July, its owners Regeneron Pharmaceuticals (NASDAQ: REGN) of Tarrytown, NY, and France’s Sanofi (NYSE: SNY), announced it would cost slightly more, about $14,500 a year. Patients are meant to self-inject the drugs every two weeks for life. (Evolocumab will also have a monthly option, due out next year.)
Both approvals—and the leeway doctors seem to have to prescribe the drugs—are almost certain to generate pushback from insurance companies and their drug-buying middlemen, who have issued dire warnings about the costs of alirocumab and evolocumab if widely prescribed.
European regulators approved evolocumab in July and are likely to approve alirocumab, as well.
The competition between alirocumab and evolocumab began well before the approvals. In 2014, Regeneron and Sanofi paid $67.5 million in 2014 for a voucher that let them speed up the FDA review of alirocumab by a few months, which is why their approval date arrived one month before Amgen.
Both drugs block a protein known as PCSK9 (proprotein convertase subtilisin/kexin Type 9), that makes it harder for the body to get rid of the bad form of cholesterol, LDL-c. By blocking PCSK9, alirocumab and evolocumab help the liver do its job to flush LDL-c out of our arteries. Both are monoclonal antibodies.
Another PCSK9 inhibitor antibody, from Pfizer, is in Phase 3 trials. Farther down the road is a an RNA-based drug from Alnylam Pharmaceuticals (NASDAQ: ALNY) that should produce Phase 1 data next weekend at a medical conference in London.
The approval of both PCSK9 drugs is good news first and foremost for people who have inherited hypercholesterolemia, a dangerously high level of cholesterol. The so-called homozygous form, often known by the shorthand HoFH, is rare and severe; children with it can die of heart attacks. It afflicts anywhere from 1 in 160,000 to 1 in 1 million people worldwide, according to the FH Foundation. Those people will prime candidates for PCSK9 drugs. They are specifically noted on the FDA’s label for evolocumab, but not on the label for alirocumab, because Regeneron and Sanofi did not test their drug in that population. (If doctors adhere to the label, that puts alirocumab out of reach for the estimated 2,000 people in the U.S. with HoFH.)
[A previous version of this story misstated the U.S. HoFH number.]
The FDA approved both drugs for people with heterozygous hypercholesterolemia, or HeFH, which is inherited from only one parent. One in 200 to 500 people worldwide have HeFH, according to the FH Foundation.
But they could end up in a gray area from an insurer’s point of view, says FH Foundation president Katherine Wilemon, who herself has HeFH—and had a heart attack at age 39.
Insurers might want doctors to exhaust all other possibilities, including more … Next Page »