It's taken years for Amarin's fish oil derivative Vascepa to prove itself as a game-changing treatment for cardiovascular disease. After a "landmark" outcomes trial last year and a hotly anticipated FDA advisory meeting Thursday, Vascepa may now have its ticket to the big leagues.
An FDA advisory committee unanimously backed a Vascepa approval in tandem with statin drugs to reduce the risk of cardiovascular events in patients with cardiovascular disease.
That patient population, far larger than Vascepa's current target in patients with high triglycerides, could drive it into the blockbuster fold, provided the FDA goes along with its expert panel's advice.
The advisory committee recommended approval after reviewing data from Vascepa's phase 3 Reduce-It trial released last year. That much-ballyhooed trial showed that adding Vascepa to statin therapy reduced the risk of cardiovascular events such as heart attack and stroke by 25% compared with statins alone.
The advisers gave their full-throated approval for the new indication, but the FDA still has some kinks to iron out before its final label approval.
For one, the agency will decide whether to include lowering the risk of cardiovascular death on Vascepa's label after advisers saw few data supporting that claim. Second, the committee left it up to the FDA to decide whether to approve Vascepa as a primary preventative for CV disease––a move that would give Vascepa a remarkably broad indication.
The committee's recommendation came after last-minute questions from FDA staff, laid out in documents posted ahead of Thursday's meeting. The mineral oil placebo used in the trial may have skewed biomarker results in the study's control arm, the reviewers suggested.
The trial tested Vascepa as an add-on to statin therapy, compared with the mineral oil placebo paired with statins. John Sharretts, acting deputy director of the FDA's division of metabolism and endocrinology products, said in a briefing the FDA examined whether that mineral oil interfered with patients' ability to absorb statin therapy and artificially raised patients' cholesterol levels but called the study "inconclusive."
"Due to lack of certain key measurements, we could neither rule out the possibility that mineral oil––at least to some extent––interfered with statin absorption, nor estimate the magnitude of LDL-C or other biomarker increase that could be attributed to such an interaction," Sharretts wrote in the review documents.
On the positive side, Sharretts also outlined a few label possibilities that ranged from narrow to broad, including treating patients with established CV disease or expanding treatment to high-triglyceride patients at risk of CV disease.
The committee's positive recommendation followed Reduce-It data released in October 2018 showing Vascepa added on to statin therapy reduced the risk of cardiovascular events by 25% compared with statin therapy plus placebo. That win was touted as a potential blockbuster turn for Amarin by analysts.
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Amarin originally expected a label decision by the FDA in September, but that was before the agency opted to convene an advisory committee to discuss the data. Amarin has already been forced to delay its plan to double its sales force to 800 in anticipation of Vascepa's new label, which may be ready to market as soon as January.