FDA panel unanimously backs use of Amarin’s fish-oil heart drug; action could produce gigantic profit haul.

sbtlneet / Pixabay

The News:

Government health experts recently recommended broader use of a prescription-strength fish oil drug to help many more patients at risk for heart attack, stroke and related health problems. Currently the drug, Vascepa, is approved for a relatively narrow group of patients with extremely high levels of triglycerides, a type of fat in the blood linked to heart disease, says the Associated Press.

Irish drugmaker Amarin PLC (Dublin) is seeking approval for a much larger group of patients who have lower fat levels but are still at risk of heart problems, despite taking cholesterol-lowering statin drugs like Lipitor and Zocor. The panel of advisers to the FDA unanimously supported the expansion based on recent study results showing the drug can cut rates of life-threatening heart problems in high-risk patients.

“There is no doubt this drug could benefit a substantial portion of the US population and meet an unmet need,” said Dr. Jack Yanovski, a panelist and hormone specialist from the federal National Institutes of Health.

The FDA is not required to follow the group’s advice and is expected to make its decision by the end of the year.

Financial analysts predict that broader use could translate into billions in sales for Amarin. Vascepa costs around $300 a month. Some panelists urged the FDA to tailor the drug’s label to patients who are most likely to benefit.

“I do not want to see this become what I call cardio candy,” said Philip Posner, a patient representative on the panel.

FDA-written drug labels guide physician prescribing and often determine which uses are paid for by insurers.

Panelists based their decision on a company-funded study showing that Vascepa cut the risk of heart attack, clogged arteries and other cardiovascular problems by about 25%, compared with placebo treatment. Patients in the study were randomly assigned to take Vascepa or mineral oil capsules as a comparison. Patients in the trial had high triglycerides and other risk factors for heart problems, such as diabetes.

The results, published last year, came as a surprise because a string of past studies of fish oil failed to show a positive impact on heart health. The panel noted several potential side effects of the drug, including irregular heartbeats and internal bleeding. But they agreed the drug’s benefits outweighed those risks, which could be monitored and managed by physicians.

Wall Street analysts have high expectations for broader use of the drug, with some projecting annual sales of $3 billion or higher. Vascepa, first approved in 2012, is Amarin’s only product on the US market. Its shares closed the week up 37% at $24.02 in New York.

Steve’s Take:

As a kid I used to go fishing each opening day in the Spring with my father in a little stream in central Virginia called “Big Stoney.” The water was freezing from the winter runoff in the mountains, and we had to wade into the swift current seeking rainbow trout. Mostly we’d catch one or two (after around six hours of casting and reeling), and somedays come back with what my father called a “slim catch,” meaning zero.

But once in a while we’d come back home with a “big haul,” as dad called it. That’s what could be in store for Amarin if the FDA approves Vascepa, and here’s why.

First some history.

Amarin stunned the world last year when a prolonged clinical trial showed that its Vascepa medicine derived from purified fish oil, substantially reduced the risk of cardiovascular events like heart attacks in high-risk patients. After the elation and 300% stock rally waned, the question remained, precisely how big a blockbuster might it be.

Part of that question was answered Thursday after a panel of experts convened by the FDA reviewed Amarin’s data. They voted 16 to 0 that Vascepa was safe and cuts cardiovascular events. The vote doesn’t compel the FDA’s ascent, but the agency often follows panel recommendations, so it would be a surprise now if the drug isn’t made available to more Americans. That’s big news for Amarin–and millions of patients.

Bloomberg’s Max Nisen notes we still don’t know exactly how good the news might be for Amarin, however, and won’t until the FDA makes a final decision by the end of the year. Concerns of the panel included the use of mineral oil in the placebo arm of the trial, which may have mildly impacted trial results, as well as a potentially elevated risk of bleeding and atrial fibrillation. Importantly, though, there was consensus on the drug’s overall effectiveness. Still, the panelists disagreed about how far that impact extends. Access for millions of additional patients and billions of potential sales are still a pipe dream, which means there’s more uncertainty ahead for Amarin investors.

Nisen adds that Vascepa is already available for a small group of people with very high triglycerides, a blood fat that the drug goes after. Now, Amarin is understandably looking to leverage its impressive success into the largest, new commercial opportunity. But though Vascepa’s benefit looks clear in patients that have already had a cardiac event–ergo, the unanimous vote–some panelists weren’t as convinced on its impact on lower-risk patients, the largest potential market for the drug. In their view, Nisen notes, because the benefits aren’t as strong in that group, questions about the drug’s impact and safety should predominate and rightly constrain the ultimate scope of approval.

The number of potential nuances in the drug’s expanded approval and the size of the population in question mean that the particulars of the agency’s decision will still be a colossal swing factor–great, good, or not so much–for Amarin. The FDA’s final take will drive Vascepa’s upside, growth trajectory, and even whether another trial is warranted.

Bottom Line:

Vascepa proponents who spoke during the public portion of the meeting, as well as at least one panelist, noted that a restrictive approval by the agency could lead insurers to reduce coverage of a medicine that could benefit scores of people. On the one hand, the FDA will consider the concerns raised on Thursday, says Nisen. On the flip side, it will also look at the risks of being overly cautious, in light of the mammoth economic and human price tag created by cardiovascular disease.

Harkening back to my childhood fishing reminiscence, seldom did we have one of those “big-haul” experiences up there at Big Stoney. And if Amarin’s fish-oil medicine turns into a big commercial haul for the Irish company, isn’t that ironic? Nowadays, how often do we see a medicine that can beneficially treat the No. 1 health hazard for millions of people in the US with something as bountiful as fish oil?

Print Friendly, PDF & Email