FDA health advisers back first-in-class cholesterol drug
Federal health advisers on Tuesday recommended approval for a highly anticipated cholesterol drug from Sanofi and Regeneron Pharmaceuticals, but with the caveat that more data is needed about its long-term ability to reduce heart attacks.
The expert panel recommended by a 13-3 vote that the Food and Drug Administration approve the injectable drug, called Praluent.
But in an unexpected development, a number of panelists said the drug should only be used in patients with abnormally high cholesterol levels caused by an inherited disorder. Those panelists said they wanted to see more data about whether the drug ultimately reduces heart problems, before it is used more broadly.
If the FDA follows the group’s recommendation, the drug could be approved for a much smaller population than its developers have proposed.
Praluent is part of a new class of injectable drugs that lower cholesterol more than older medications called statins, which have been the standard treatment for more than 20 years. A similar drug from Amgen will go before the same panel on Wednesday.
Both drugs block a substance called PCSK9, which interferes with the liver’s ability to remove cholesterol from the blood. Analysts expect the drugs to grow into blockbuster sellers, generating billions of dollars in new sales. But Tuesday’s meeting underscores the uncertainty about who should receive such medications.
More than 73 million U. S. adults, or nearly one-third, have high LDL cholesterol, according to the Centers for Disease Control and Prevention. Those patients have twice the risk of heart disease, the leading cause of death in the U.S.
Paris-based Sanofi and Regeneron, which is based in Tarrytown, New York, have suggested marketing their drug to a large group of patients, including those who are at high-risk for heart attacks and those who cannot tolerate statin drugs due to side effects.
But many panelists said they would limit the drug’s use to a much narrower group of patients who have abnormally high cholesterol levels caused by a family disorder.
Key to the panel’s decision was whether lowering bad, or LDL, cholesterol translates into reduced heart attacks. For over 20 years, the FDA has used reduced LDL levels as a benchmark to approve cholesterol drugs, including statins like Lipi- tor and Zocor.
But recent studies of drugs like niacin have shown that reducing cholesterol levels does not always lead to reductions in heart attack, stroke and related problems.
The FDA’s internal scientists pressed panelists on whether they thought lower LDL cholesterol was still an accurate predictor of a drug’s benefit.
Despite the measured endorsement, company representatives said they were “gratified” by the meeting’s outcome.
Regeneron’s stock fell about 3 percent in extended trading following the announcement. Sanofi’s U.S.-traded shares rose about 1 percent.