Mark your calendars Amarin (AMRN 2.65%) investors. The biotech's fish oil, Vascepa, will get its day in front of a Food and Drug Administration advisory committee on Oct. 16.
There's no way this is good news for Amarin. It's safe to say that if the FDA didn't schedule an advisory committee meeting, investors could be confident that the FDA would approve the drug since the trial supporting the expanded indication was performed under a Special Protocol Assessment, or SPA. It's unlikely the FDA would reject the drug without consulting the panel unless there was some issue like manufacturing that Amarin hasn't disclosed.
But it's hard to see scheduling the meeting as horrible news, either. Vascepa is approved for treating patients with extremely high triglyceride levels at or above 500 mg/dL. But no fish oil has been approved for the larger expanded population of moderately high triglyceride levels from 200 mg/dL to 499 mg/dL that Amarin is asking to treat.
It's certainly in the FDA's right to ask outside experts for their opinion; I would argue it's not all that surprising even if the FDA is leaning strongly toward approving the drug. There are plenty of cases -- Seattle Genetics' (SGEN +0.00%) Adcetris, for example -- where the FDA called a meeting only to gush all over the drug. The drug got a unanimous positive recommendation from the panel and was approved early by the FDA.
There shouldn't be an issue over whether Vascepa lowers triglycerides in the expanded population. In Amarin's Anchor trial, Vascepa lowered triglycerides by a placebo-adjusted 21.5% at the higher dose. It also lowered bad LDL cholesterol by 6.2% versus placebo. And that was in patients who were already taking a cholesterol-lowering statin.
The bigger concern is whether lowering triglyceride levels has any clinical outcome, whether taking Vascepa reduces heart related events, such as heart attacks and strokes. Amarin has started a trial, Reduce-It, to test that hypothesis, but the study isn't expected to read out until November 2016.
The FDA already signed off on the Anchor clinical trial being sufficient for approval as long as Amarin had an outcomes trial substantially enrolled. But SPAs always comes with a caveat that the FDA is able to change its mind if new information becomes available.
In Vascepa's case, the problem is that there have been quite a few drugs recently that have looked good on laboratory tests, but failed to show a clinical effect. Merck's (MRK 1.49%) Cordaptive, for instance, increased good HDL cholesterol, and lowered bad LDL cholesterol and lipid levels, but failed to improve clinical outcomes.
I have a feeling that many of the doctors on the panel will be OK with approving Vascepa with the current data. Its competitor, GlaxoSmithKline's (GSK 0.40%) Lovaza, is already being used off label by some doctors, and Lovaza actually raises bad LDL cholesterol. Vascepa is relatively safe, too, so even if it isn't reducing heart problems, it's not going to cause too many problems before Reduce-It gives a definitive answer.
But the tone for the meeting -- and the ultimate decision -- will likely be set by the FDA reviewers ahead of the meeting when the FDA posts its briefing documents for the committee, likely on Oct. 11 because the usual time -- two business days prior -- falls on Columbus Day, a federal holiday.
The doctors on the panel might be able to change the FDA reviewers' minds, but since the FDA has the final say, investors should pay close attention to the briefing documents.
