Amgen Inc.’s Repatha won the backing of a panel of U.S. regulatory advisers for some patients, making it the second in a new class of powerful cholesterol-lowering drugs to move closer to U.S. approval this week.
Repatha’s benefits outweigh its risks for some patients with difficult-to-treat high cholesterol, advisers to the Food and Drug Administration voted 11-4 on Wednesday. They also voted 15-0 that the medicine’s benefits outweigh its risks for patients with a rare genetic disorder that causes ultra-high cholesterol levels.
Panel members said Repatha’s use should be limited to patients with both forms of the genetic disorder and those who are at high risk of experiencing a heart attack until more study is done on the drug’s effect on heart health.
Repatha is part of a category of drugs known as PCSK9 inhibitors, designed to help patients who can’t get their LDL, or bad, cholesterol under control with widely used statins such as Pfizer Inc.’s Lipitor, or can’t tolerate the drugs. The FDA is scheduled to decide by Aug. 27 whether to approve Repatha and designate which patients should use it.
Another drug, Praluent from Sanofi and Regeneron Pharmaceuticals Inc., may be the first PCSK9 to market. The FDA is expected to decide on Praluent by July 24.
Advisers voted 13-3 on Tuesday that Praluent’s benefits outweigh its risks, though most members supported limiting approval to a more common genetic disease that affects about 1 in 500, called heterozygous familial hypercholesterolemia. Amgen is seeking to sell Repatha to patients with the rarer and more severe homozygous form of the disease as well.
The panel debated Tuesday and Wednesday whether the products’ ability to lower bad cholesterol is sufficient without proof that they will also reduce heart-related deaths. While the FDA has allowed cholesterol-lowering drugs, such as statins, to come to market based on the assumption that lower cholesterol leads to better heart health, the advisers were unconvinced the same could be presumed for PCSK9s.
Until studies on the drugs’ heart effects are completed in 2017, advisers recommended the drugs be limited to patients most in need. The panel raised concern that low-risk patients taking statins that are proven to improve heart health would too hastily attempt to make the switch to a PCSK9.
“On the one hand, cardiovascular outcomes trials are extremely important, and it is not yet proven that they will be successful,” said Kenneth Burman, a panel member and director of the endocrine section at Washington Hospital Center who voted in favor of approval. “On the other hand, it will take at least two years to establish whether LDL lowering is a surrogate, and in the meantime I support the use of this medicine.”
More study is need on the drugs to rule out risks such as developing diabetes or experiencing memory loss, panel members said.
Praluent may generate $1.9 billion in revenue for Sanofi in 2020, while Repatha may bring in $2.5 billion for Amgen, analysts estimate.
The drugs cut LDL, or bad, cholesterol levels by more than 60 percent in studies published in March in the New England Journal of Medicine. High cholesterol is linked to heart disease, the No. 1 killer of Americans.
Prime Therapeutics LLC, a pharmacy benefit manager owned by 13 Blue Cross and Blue Shield plans, expects about 2.5 million Americans will be eligible to take Praluent or Repatha, at a cost of as much as $23.3 billion a year, according to a statement. The drugmakers haven’t disclosed the price of the drugs, so Prime estimated the cost at $10,000 a year. The benefit manager called for controlling access to the drugs, limiting them to approved patients.