The FDA Should Provisionally Approve Vascepa

October 21, 2013 – 2:36 pm | By Catherine Shaffer | No comments yet

Amarin blog 10-21-13The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) rocked the biotech industry last week by voting 9 to 2 against approval of Amarin Pharmaceuticals Inc.’s fish-oil derived omega-3 supplement Vascepa in the supplemental indication of mixed dyslipidemia. The drug is already approved for lowering triglycerides and its efficacy and safety results from the Phase III ANCHOR trial for the new proposed indication were just about as good as it gets, so it was widely expected the EMDAC would rubber stamp the new indication with perhaps a couple of holdouts.

The voting reversal was due to pointedly worded discussion and voting questions, which put the committee on the spot not only to decide whether the ANCHOR trial had met its goals of showing efficacy and safety in reducing the targeted lipid values, but whether the use of those lipid changes as a surrogate endpoint was valid for approval.

The committee ultimately decided to recommend against immediate approval, asking the company to resubmit its application after the results of its ongoing cardiovascular outcomes trial, REDUCE-IT, are available some time in 2017. Whether that’s even going to happen is now in doubt, as Amarin was counting on increased revenues from the expanded indication to keep the lights on through the end of that trial.

Meanwhile, folks have been wondering if the FDA couldn’t provisionally approve Vascepa for mixed lipidemia, and in some way make its continued approval contingent on a positive outcome from REDUCE-IT.

That question was raised in the adcom discussion. The answer given was that the FDA can give provisional approval in four situations: in the event of accelerated approval, in the case of deferred pediatric studies, using the animal rule, and if there’s a specific safety concern.

Accelerated approval, deferred pediatric studies, and the animal rule do not apply in the case of Vascepa, and the committee agreed that there was no safety concern with Amarin’s application.

So although at least some on the committee would have liked to recommend approval of Vascepa in mixed dyslipidemia, with some option to take it away if REDUCE-IT didn’t pan out, the FDA’s regulations did not provide a mechanism for it. The panel only had the option to recommend approval or not.

Of course, the agency is not bound to the panel’s recommendation.


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