WASHINGTON — A panel of outside advisers to the Food and Drug Administration voted 7-6 on Thursday that the benefits of a twice-yearly cholesterol-lowering shot outweigh its risks, a split that leaves the agency with a close call ahead of its decision date in February.
The drug, veltisiran, is made by Corvane Therapeutics, a mid-size company based in New Jersey. It works by switching off a gene in the liver that helps control how much LDL cholesterol stays in the blood. In Corvane’s main trial, which enrolled about 9,400 people with heart disease or a high risk of it, the shot cut LDL levels by about half compared with a placebo.
The disagreement on the panel was not about whether the drug lowers cholesterol. It was about who was studied, and for how long.
The case against
Only about 11 percent of trial participants were 75 or older, according to the FDA’s briefing documents. That group made up a far larger share of the people who would likely be prescribed the shot, several panelists said, and it was also the group with the highest rate of a side effect that worried agency reviewers: a rise in liver enzymes, seen in 3.1 percent of people on the drug compared with 1.2 percent on placebo.
«We are being asked to approve this for the patients we know the least about,» said Dr. Celia Brandauer, a cardiologist at a university hospital in Minnesota, who voted no. «I’d like to see two more years of data in people over 75 before this is on every pharmacy shelf.»
The trial also measured cholesterol, not heart attacks or deaths. Corvane is running a separate outcomes study, but its results are not expected for about three years.
The case for
Members who voted yes pointed to the same number every drug in this field is judged on: LDL cholesterol, which decades of research tie to heart attacks and strokes. They also pointed to the dosing. A shot given twice a year by a nurse could help patients who stop taking daily pills, which studies suggest is about half of people within a year.
«Adherence is the hidden epidemic in cardiology,» said Dr. Raymond Osei, a preventive cardiologist from Georgia, who voted yes. «This drug takes that problem off the table.»
What we know, and what we don’t
We know: – The shot lowered LDL cholesterol by about half over 18 months. – Liver enzyme rises were more common on the drug, and most went back to normal.
We don’t know yet: – Whether it prevents heart attacks or deaths. That study reads out in about three years. – How it works in people over 75, who were 11 percent of the trial. – What it will cost. Corvane has not said.
The FDA is not bound by its advisers’ votes, though it usually follows them. Agency staff could approve the drug with a requirement for more study in older patients, or ask for a narrower label.
Corvane’s shares fell 9 percent in after-hours trading. In a statement, the company said it was «encouraged» by the vote and would «keep working with the agency.» Its chief executive, Lorna Vasquez-Hale, declined an interview request.