In explaining the rationale for the FDA delay, Amarin released a brief statement saying the agency has not disclosed when a decision may be made on the Vascepa indication. But Amarin attributed the delay to its recent appeal of an FDA decision to rescind a so-called Special Protocol Agreement that was reached with the agency for using a clinical trial for that wider indication. The delay is yet another twist in what has been one of the more closely watched biopharma stock stories on Wall Street this year. Investors were captivated by the possibility that Amarin might have been able to market a potential blockbuster amid a heated debate over the virtue of using lower triglycerides as a predictive metric for lowering cardiovascular risks.
As was reported previously, Vascepa is already approved for treating people with very high triglyceride levels, or more than 500 mg/dL, which is approximately 4 million people in the US. Amarin, however, had sought FDA approval to market Vascepa to people with high cholesterol and high triglycerides, which is between 200 and 500 mg/dL, a market that is estimated to be as many as 36 million people.
The Vascepa approval had been based on a trial called Anchor that was the basis for the special protocol agreement, and Amarin hoped to steal market share from Lovaza, a $1 billion seller for GlaxoSmithKline (GSK). If the clinical effect of combining Vascepa and a statin were to prove beneficial to cardiovascular outcomes, then a drugmaker that inks a deal with – or acquires – Amarin would have a blockbuster. In November 2013, though, Amarin appeared to hit a brick wall. An FDA advisory committee voted 9-to-2 against recommending broader use until an outcomes study is completed in 2016, prompting the FDA to rescind the Special Protocol Agreement. Amarin has since cut its staff by 50 percent and now faces a growing number of shareholder lawsuits that accuse the drugmaker of making overly optimistic predictions. Whether sufficient funds can be obtained to continue the trial are also uncertain.
Amarin quickly filed an appeal in hopes the FDA would change its mind about rescinding the agreement and, if nothing else, the gambit prompted the agency to review its strategy. In its statement, Amarin (AMRN) maintains that the FDA “now views Amarin’s appeal of the Anchor agreement rescission and the Anchor (application) as separate administrative decisions worthy of separate consideration.” And so, the FDA apparently plans to complete its review of Amarin’s request to re-instate the Anchor SPA agreement and convey its decision to Amarin no later than January 15, 2014. By separating the decisions, the agency could be attempting to minimize the threat of legal action by the drugmaker, according to sources who have been following the Amarin saga.
Consequently, investors are now betting that, perhaps, some form of FDA approval to widen the Vascepa indication is possible, after all. The stock rose significantly after the Friday announcement. Some speculation the agency may require a “black box” warning that a reduction in cardiovascular events was not a goal or proven in the Anchor study and that physicians must be cautious about prescribing the pill to patients with triglycerides below 500mg/dl.
REFERENCE: PharmaLive; PharmaLot; 23 DEC 2013 – Ed Silverman