Steering for a U.S. comeback, AstraZeneca's Brilinta runs aground on DOJ investigation

Here's a scenario: You're trying to jump-start sales of a drug. It's one of those products that never has hummed on all cylinders, but you think it could wear racing stripes. And now, you're seeing signs of life.

Then the feds show up, digging into the study data that justified that drug's FDA approval.

That's the conundrum facing AstraZeneca ($AZN) and its Brilinta blood thinner, now under investigation by the Justice Department. It's a probe that could--worst case scenario--end up forcing the drug off the market.

Pascal Soriot--FiercePharma file photo

CEO Pascal Soriot trotted out a list of hope-inducing indicators during a discussion with analysts last week, and some explanations for stats that aren't so positive. For instance, Brilinta's 2013 sales amounted to $283 million, more than three times its 2012 total. In some European markets, the drug has taken the lead in its niche. "[T]his actually demonstrates the potential of this product and it demonstrates what it can do when marketed," Soriot said during the conference call.

And about the market-share figures in the low single digits, prescription-wise? That's as much about the size of the patient pool as anything else. When millions of patients are using blood thinners, even tens of thousands of new prescriptions hardly move the needle on share. Against that background, a 1-percentage-point increase looks pretty good.

Bottom line? Brilinta is actually doing well outside the U.S., Soriot said. In the U.S., AstraZeneca's plans for a stepped-up promotional push finally hit its stride in August. And then, whammo, the Justice Department said it was investigating some disputed data from the PLATO trial, which was key to Brilinta's regulatory clearance.

"We felt we were moving in the right direction," Soriot said. "We saw some nice feedback, some nice movement, until the DOJ investigation impacted us." And that brought the Brilinta push to a screeching halt in the U.S. 

So, as one analyst asked during the call, what needs to happen for Brilinta to actually beat earlier forecasts for 2018 sales, as Soriot has said it can? "[W]e need to see an acceleration in the U.S., very simply," Soriot said.

The company is working to answer the DOJ's questions, but while the investigation proceeds, there's not a lot AstraZeneca can do to amp things up immediately. It can move forward on broadening Brilinta's target market with new research data, which could be a big help; as Soriot repeatedly pointed out, the drug's FDA-approved indication covers only a small segment of the potential market.

Though doctors theoretically could prescribe it off-label to many more patients, payers reimburse only for the approved indication. The PEGASUS trial could back Brilinta, now approved for 12 months of treatment, for use longer-term, for one thing. For another, Brilinta is being studied for broader use in acute stroke patients and in those with peripheral artery disease.

But it all comes down to what happens at the DOJ. "Our plans basically have been in place on this since August of last year ... and I think we have to wait till we can actually clear this phase of the DOJ investigation and see whether we can roll," Soriot said. "If we can't then we have to accept we cannot. But I still remain confident that we have a chance to succeed even in the U.S."

- read the call transcript

Special Reports: Top 10 Drug Launch Disasters - Brilinta

Editor's note: This article was modified to correct the reference to the PEGASUS trial. A previous version identified it as PEGASYS.