Amarin's (AMRN +0.68%) decision from the Food and Drug Administration came a few days later than expected, but the result was as expected: The FDA turned down Amarin's appeal of the agency's decision to rescind its Special Protocol Assessment.
The division that reviewed the appeal was the same one that made the initial decision to rescind the SPA, so it's not surprising it would come to the same conclusion.
Upward and onward
Amarin plans to move the appeal up the chain of command. With each level taking 30 to 60 days to make a decision, it could take 10 months to reach the commissioner.
The SPA was supposed to allow Amarin to expand the indication for its triglyceride-lowering drug, Vascepa, to include patients with moderately high triglyceride levels as long as the biotech showed that Vascepa lowered triglycerides (it did) and that the company substantially enrolled an outcomes study (it has).
But the FDA rescinded the SPA after clinical trials run on AbbVie's (ABBV 2.86%) Tricor and Merck's (MRK +0.03%) Cordaptive lowered triglyceride levels but failed to show an effect on outcomes. Never mind that AbbVie's and Merck's drugs work under a different mechanism of action and tested a different set of patients than Amarin is trying to get approved for, the agency concluded that lowering triglycerides isn't a meaningful surrogate for reducing heart attacks and strokes.
Give us an expanded indication or else
Amarin's understanding is that the FDA won't take action on the application to expand the market until the appeal moves at least two levels higher, which should occur in late spring or early summer.
Interestingly, Amarin has given the FDA an ultimatum, telling the agency that it won't continue the REDUCE-IT outcomes study unless the FDA lives up to its side of the bargain and expands Vascepa's indication. "FDA is aware that if we don't get the ANCHOR indication that we are not going to be able to continue REDUCE-IT in its current form," John Thero, Amarin's president and CEO, told investors on the conference call.
I'm sympathetic to Amarin's dilemma. SPAs allow companies to run clinical trials with the confidence that they'll gain approval if things go as planned. But I'm not sure the FDA is going to be swayed into changing its mind because Amarin threatens to not follow through with its end of the deal after the FDA reaffirms its decision to back out. The agency wants to encourage innovation, but not at the expense of accurate calls on efficacy.
Moving on
Beyond trying to get the expanded indication, Amarin has a couple of backup plans. The FDA might let Amarin include the data from the trial of patients with moderately high triglyceride levels in the clinical-trials section of the package insert without technically being approved for that indication. Amarin might also try to argue it can tell doctors about the trial based on its First Amendment right of free speech.
Neither seems as good as a full approval, allowing Amarin to advertise the drug for the expanded indication, which is probably necessary to get Vascepa to blockbuster status.
Even without the expanded indication, Amarin thinks it has enough cash to become cash flow-positive selling Vascepa just to patients with extremely high triglyceride levels, although that likely includes modifying the outcomes study to reduce expenses.
Stay tuned; it's going to be an interesting year for Amarin.
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