LONDON AstraZeneca is being investigated by U.S. authorities over a major clinical trial that was used to win marketing approval for its new heart drug Brilinta, casting fresh doubts over the medicine.
Brilinta sales have been a disappointment since its launch two years ago, although AstraZeneca has recently stepped up marketing efforts and hopes that further clinical tests will underscore its value in preventing heart attacks.
The British drugmaker said on Thursday it received a civil investigative demand from the U.S. Department of Justice (DOJ) 10 days ago, seeking documents and information regarding the so-called PLATO study.
AstraZeneca plans to cooperate with the DOJ and Chief Executive Pascal Soriot said he was "very confident" in the findings of the clinical trial.
"It was guided by a strong academic group who oversaw the trial and its conduct," Soriot told reporters after announcing third-quarter results. [
AstraZeneca did not disclose the exact nature of the investigation and DOJ spokesman Peter Carr in Washington said he had no immediate comment on the case.
The results of the 18,000-patient PLATO were first reported at a medical meeting in 2009 and went on to form the basis of successful new drug applications in the United States, Europe and other markets. Brilinta was launched in 2011.
CRITICISM OF TRIAL
Various aspects of the study, which relied heavily on patients recruited in eastern Europe, have been criticized over the years by a number of medical experts. Poland and Hungary together accounted for 21 percent of all subjects studied - more than double the United States and Canada combined.
James DiNicolantonio of Ithaca New York and Ales Tomek of Charles University in Prague raised several questions about the conduct of the study in a recent paper in the International Journal of Cardiology.
In particular, their analysis found that patients in the PLATO trial who were monitored by AstraZeneca were reported as having more beneficial effects from taking Brilinta than those monitored by an independent clinical research organization.
Those conclusions, however, have been strongly disputed by Lars Wallentin of Uppsala Clinical Research Center in Sweden, the principal investigator on PLATO.
Brilinta is a key new product for AstraZeneca but its progress to date has been slow, with sales in the third quarter edging up to just $75 million from $65 million in the three months to end-June - a long way from the multibillion-dollar sales potential that analysts once predicted.
Among the challenges it faces is the fact that U.S. doctors tend to use high-dose aspirin for cardiovascular patients, and clinical trials suggest AstraZeneca's new drug does not work as well in this setting.
AstraZeneca hopes that a new clinical study called PEGASUS will put to rest any uncertainties about the effectiveness of Brilinta and also increase uptake of the medicine. Results are expected in 2014.
In addition to the DOJ inquiry into Brilinta, AstraZeneca also disclosed it had received a subpoena from the U.S. Attorney's Office in Boston seeking documents and information related to the safety of its antipsychotic drug Seroquel.
(Additional reporting by Diane Bartz in Washington; Editing by Kate Kelland and Mark Trevelyan)