[Corrected 6/10/15, 10:14pm. See below.] Katherine Wilemon had a celebratory glass of wine a couple hours ago, which some say is good for the heart. Even better for Wilemon, however, is the reason she’s celebrating: In a two-day discussion about two new drugs aimed at high cholesterol, a panel of outside advisors recommended that the FDA approve both because they dramatically reduce the levels of LDL-c, the so-called bad cholesterol.
The drugs under review were alirocumab (Praluent) from Sanofi (NYSE: SNY) and Regeneron Pharmaceuticals (NASDAQ: REGN) and evolocumab (Repatha) from Amgen (NASDAQ: AMGN). Both now await a final FDA decision.
Wilemon is founder and president of the FH Foundation, a Pasadena, CA-based advocacy for people with an inherited form of dangerously high cholesterol called familial hypercholesterolemia. The version of FH inherited from both parents is classified as a rare disease; the form inherited from one parent is quite common, affecting at least 1 in 500 people. The panel’s recommendations yesterday and today, although peppered with reservations, make it clear that for both forms of FH, the drugs have an excellent chance of approval. (FDA isn’t obliged to follow such advice, but it often does. The decision on alirocumab is due July 24, the decision on evolocumab August 27.) [This paragraph was corrected to clarify that one of the two versions of FH is a rare disease.]
“I’m more relieved than I have ever been in my whole life,” said Wilemon right after the meeting. She has FH, too. She suffered a heart attack at 39, and after years of taking all manner of lipid-lowering treatments, her cholesterol level was a stubbornly high 167.
Her cholesterol level now? “Forty-one,” she said. Wilemon (pictured) disclosed that she is taking part in a trial of one of the new class of drugs, known as PCSK9 inhibitors because they block a protein that makes it hard for the liver to clear LDL-c—the bad cholesterol—from the blood stream. (She wouldn’t say if she has been taking one of the drugs under review this week, or a third one, bococuzimab, that’s not ready for FDA review.)
On both days, the advisors’ votes were positive, but there were significant differences. On Tuesday, 13 of 16 panel members gave a thumbs-up to alirocumab. It was a general “yes”—they could not formally vote on the drug’s use in various patient subtypes. But they made their views fairly clear.
In comments before and after the vote, there was little dissent about approving the drug for familial hypercholesterolemia, or FH, but the panelists raised serious questions about using the drugs to treat other patient groups—such as people with very high cholesterol (not the genetically inherited form), and people who cannot tolerate statins, which are the current standard of care.
Those same reservations came up again today in the discussion about Amgen’s evolocumab. One difference, however, came in the formal vote. The panel specifically cast ballots for FH. First, they agreed 15-0 that homozygous carriers, who inherited the FH genetic mutation from both parents, should be eligible for the drug—““a clear no-brainer,” as one panelist put it. (For a description of the vote and discussion around it, see the live blog Matthew Herper of Forbes wrote during the event.)
They also voted 11-4 that heterozygous carriers, who inherited the mutation from only one parent, should be eligible. Despite the genetic nature of FH, the disease is still mainly diagnosed by piecing together other clues, including a family history of early-onset heart disease, a high cholesterol count impervious to statins, and blemishes called xanthomas—collections of lipids that appear as welts on the skin.
There are genetic tests, but it’s unclear how good they are. Wilemon called genetic testing for FH “the gold standard but not the perfect standard,” and noted that in the U.S., the tests aren’t covered by insurance and thus are barely used.
That leads to a bigger question about these new PCSK9 inhibitors: Will insurers pay for them? As I noted in my column Tuesday, the drug-buying middlemen who aim to keep costs down have already vowed … Next Page »