FDA panel backs Sanofi/Regeneron cholesterol drug with limits

A new type of cholesterol-lowering drug made by Sanofi SA (SASY.PA) and Regeneron Pharmaceuticals (REGN.O) should be approved, an advisory committee to the U.S. Food and Drug Administration concluded on Tuesday.

The panel voted 13-3 to approve the drug, Praluent, the first in a new class of LDL-lowering drugs that inhibit a protein known as PCSK9.

Most panelists said they would like to see use of the product limited, at least initially, to patients with a genetic predisposition to high cholesterol and those at high risk of cardiovascular disease who are on maximum doses of statin therapy.

They want to see results of a large ongoing trial assessing whether the drug’s LDL-lowering effect translates into reduced cardiovascular risk before recommending it be used in a wider patient population.

The FDA is not obliged to follow the advice of its advisory panels but typically does so.

A second drug in the class, Amgen Inc’s (AMGN.O) Repatha, will be considered by the panel on Wednesday.

If approved, the drugs are expected to generate annual sales of more than $2.5 billion each by 2020, according to Thomson Reuters data.

The panel was the first in years to discuss the use of LDL as a surrogate measure of a drug’s ability to reduce heart attacks and other cardiovascular problems.

Panelists agreed LDL-lowering remains an appropriate surrogate for cardiovascular benefit for statins but were not as convinced that it is appropriate for other classes of cholesterol-lowering drugs.

Sanofi expects to complete enrollment in an 18,000-patient cardiovascular outcomes trial by the end of the year. It expects to report results by the end of 2017.

They expressed some concern about suggestions in the data that the drug could increase the risk of diabetes but said it is unclear how meaningful the risk was.

There were also hints that the drug may impact cognition and the liver. It may have to be used in millions of people before such risks can be teased out, panelists said.

They also expressed concern that the drug, when combined with a statin, could lower LDL too much, and that physicians would try to compensate by taking patients off statins or reducing their dose. That concern was also expressed by the FDA in its previously released briefing documents.

Praluent and Amgen’s drug are designed to be given twice a week by injection.

(Reporting by Toni Clarke in Washington; Editing by Matthew Lewis and Cynthia Osterman)