Advisors to FDA: Approve New Cholesterol Drug, But With Caveats

Xconomy National — 

One down, one to go, and a lot of questions left unanswered. A panel of FDA advisors voted 13-3 today to recommend that the agency give the green light to alirocumab (Praluent), one of two new cholesterol-lowering injectable drugs the panel is reviewing this week.

Beyond the main positive vote, however, some panelists also indicated that the drug should be limited to only the most desperate patients—those with an inherited form of very high cholesterol called familial hypercholesterolemia, or FH. There are roughly 620,000 FH patients in the U.S. There was no formal vote to recommend approval for patient segments—for example, for people who have bad reactions to statins, the current standard of care for high cholesterol; or for people who have high cholesterol that statins don’t lower enough.

Of the 13 who voted to recommend approval, about half said in post-vote comments they would feel comfortable with alirocumab being used for other people with very high cholesterol (not the inherited version), and even fewer said they’d recommend the drug for people who cannot tolerate statins.

The unease stems in large part from an unusual regulatory situation. The FDA allows approval of heart-disease drugs even without direct evidence that they help prevent heart disease. Instead, the FDA uses a stand-in: the drugs only need to demonstrate a reduction in so-called “bad” cholesterol.

Alirocumab, as well as the other drug under review this week, evolocumab (Repatha), have shown that ability. But potential evidence of a stronger link between their use and prevention of heart disease won’t come until 2018, by which time both drugs will have been tested in long-term “outcome” studies in more than 40,000 people, to see if those patients had fewer heart attacks and other cardiovascular events.

The panel now turns to evolocumab, in a day-long review Wednesday. Alirocumab is being developed by France’s Sanofi (NYSE: SNY) and Regeneron Pharmaceuticals (NASDAQ: REGN), of Tarrytown, NY. Evolocumab is from Thousand Oaks, CA-based Amgen (NASDAQ: AMGN).

If the panel ends Wednesday’s session with a similar vote and concerns, the next question is not only whether the FDA approves the drugs later this summer, but also how narrow the label—the types of patients allowed to take the drug—will be.

The FDA is not bound to follow the recommendations of its outside reviewers, but it does so more often than not. The agency is slated to make a decision on alirocumab by July 24 and evolocumab August 27.

In after hours trading, Sanofi shares were up 0.75 percent; Regeneron shares did not trade Tuesday because of the meeting. Amgen shares closed down one percent Tuesday and were down 0.3 percent in after hours trading.