Sanofi and Regeneron Pharmaceuticals Inc. won the backing of U.S. regulatory advisers for their powerful cholesterol-lowering drug Praluent, moving it one step closer to approval for sale this summer.
The drug may end up being used in a smaller population than the companies are targeting, however.
Praluent’s benefit outweighs its risks, advisers to the Food and Drug Administration voted 13-3 Tuesday during a meeting in Gaithersburg, Maryland. Yet many wanted to limit sales to those who have a genetic disease causing ultra-high levels of bad cholesterol. The therapy would be the first in a new class of drugs, known as PCSK9 inhibitors, for patients with difficult-to-treat cholesterol.
The panel debated whether the product’s ability to lower bad cholesterol is sufficient without proof it 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 drug’s heart effects are completed in 2017, advisers suggested that Praluent be limited to those with the genetic disorder, known as heterozygous familial hypercholesterolemia.
“I don’t want to deprive them of this drug for another two years,” said Nancy Geller, a panel member and chief of the Biostatistics Research Branch at the National Heart, Lung and Blood Institute.
For most potential patients, Geller and other panel members said they want to see the outcome of the heart trial.
Regeneron shares fell 3.6 percent to $507.23 at 10:38 a.m. in New York, their biggest intraday slide since April 30. Sanofi shares gained 0.4 percent to 88.21 euros in Paris.
The advisers also were concerned about potential harms that needed more study, including a possibility that people on Praluent were at higher risk of developing diabetes and experiencing neurocognitive effects, such as memory loss.
The FDA is expected to decide whether to approve Sanofi and Regeneron’s drug by July 24. Another of the drugs, made by Amgen Inc. and called Repatha, is scheduled for a decision by Aug. 27. Repatha will face an advisory panel vote on Wednesday.
Amgen fell 1.7 percent to $152.20 in New York. Esperion Therapeutics Inc., which is developing a cholesterol-lowering drug that could be combined with PCSK9s, fell as much as 25 percent, its biggest-ever intraday decline.
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. Trials that show whether they lower the risk of death are scheduled to be completed in 2017.
No. 1 Killer
High cholesterol is linked to heart disease, the No. 1 killer of Americans, and drugs that lower bad cholesterol are generally assumed to be good for the heart.
Sanofi and Regeneron are seeking to sell Praluent to patients who can’t get their cholesterol under control with widely used statins, such as Pfizer Inc.’s Lipitor, or who can’t tolerate the drugs.
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.