“There’s a definite need for additional therapeutic approaches,” said Kenneth D. Burman, chief of the endocrine section at MedStar Washington Hospital Center, who chaired the panel. Despite some side effects, he said, “this seems a very useful new agent for addition to the armamentarium for the treatment of these patients.”
The drug, a purified version of the Omega-3 fatty acid found in fish, is aimed at some of the more than 40 million people in the U.S. who take statins to control their LDL, or “bad” cholesterol, and have adopted lifestyle changes, yet remain at risk of cardiovascular problems because of elevated triglyceride levels.
Triglycerides are another type of fat in the blood. When their levels are too high, generally more than 200 milligrams per deciliter of blood, the result can be deaths, heart attacks, strokes, unstable angina or the need for cardiac surgery.
A landmark 2018 study, led by a researcher at Brigham and Women’s Hospital and sponsored by Amarin, showed that patients who took four grams of Vascepa daily fared 25 percent better in staving off those events than those given a placebo. The researchers spent more than six years following more than 8,000 middle-aged and older patients in 11 countries who had coronary artery disease or diabetes and at least one other risk factor, such as high blood pressure.
The drug already has FDA approval for patients with diseases that cause extremely high triglyceride levels. Allowing its use for more routine care, company officials and other speakers argued Thursday, would give doctors another tool to combat heart disease, the leading killer in the United States.
It also could be hugely profitable for the company, which has begun expanding its sales force and announced better-than-expected revenue for 2019, possibly from off-label use by physicians after the study results were announced.
In July, the company issued a statement saying that while it “remains optimistic that Vascepa will generate billions of dollars in revenue in the years to come, the history of other therapies for chronic conditions suggests that growth builds over multiple years, and thus, the company is not prepared to provide quantified guidance regarding revenue levels beyond 2019.”
Though the approval was unanimous, panel members struggled with exactly who should take the drug. Many said they believed the research clearly showed a benefit for people with diabetes aged 55 and older who have at least one other risk factor for cardiovascular disease. But they were less convinced the study proved that Vascepa would help those with already-established cardiovascular disease.
In the end, they offered a wide variety of suggestions on age and triglyceride limits, among other things, that the agency will have to sort out before instructing doctors and patients on its use.
The panel also considered two safety issues. The study showed those taking the medication had minor internal bleeding slightly more often than people in the control group. There was no difference in serious blood loss events.
Minor bleeding may be a concern for patients taking blood thinners, but that could be addressed in labeling and instructions included with the medication, experts said.
“There is a small increase in minor bleeding but no increase in the kinds of bleeding we worry about the most,” said Deepak L. Bhatt, who led the study and spoke on behalf of the company Thursday.
The drug also causes short-term irregular heartbeats in a small number of patients, a concern for some members of the review panel, known as the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee.
In considering the study, the panel debated whether the mineral oil given to patients in the placebo group may have affected absorption of statins, exaggerating the difference in results between that group and the subjects given Vascepa. After lengthy discussion of that issue, many experts said they could not be certain whether that had occurred, but they were convinced of the drug’s benefits nonetheless.