Scientists Uncover New Concerns About Billion-Dollar Heart Drug

New findings suggest serious data issues behind a major heart drug’s approval, reigniting concern over its decade-long use. Credit: Stock

An investigation has uncovered evidence of significant misreporting, raising new concerns about the approval and long-term use of ticagrelor over the past decade.

In a follow-up investigation into the multibillion-dollar drug ticagrelor, The BMJ has identified new concerns, this time focusing on key platelet studies that supported the drug’s approval by the FDA.

For over ten years, ticagrelor (sold as Brilinta in the US and Brilique in Europe) has been recommended for patients with acute coronary syndrome, a group of conditions involving a sudden reduction in blood flow to the heart.

In December, The BMJ reported serious issues with data integrity in the landmark PLATO clinical trial, which played a central role in ticagrelor’s global approval. The findings raised doubts about the drug’s claimed benefits over less expensive alternatives.

Concerns resurface as generics enter the market

Now, with generic versions of the drug expected to launch this year, The BMJ has broadened its investigation to examine two key platelet studies that AstraZeneca cited as evidence of ticagrelor’s effectiveness in treating acute coronary syndrome.

It finds that the “primary endpoint” results (the trial’s key measurement) for both clinical trials were inaccurately reported in the leading cardiology journal Circulation, and reveals that more than 60 of 282 readings from platelet machines used in the trials were not present in US Food and Drug Administration (FDA) datasets.

What’s more, one active trial investigator never became a study author, while one author told The BMJ he was not involved in the trial, and most investigators, including the principal investigator, were unreachable or declined to be interviewed.

Expert criticism and lack of transparency

Victor Serebruany, an adjunct faculty member at Johns Hopkins University and ticagrelor’s most renowned critic, told The BMJ that “there are episodes of skyrocketing rebound and profound platelet inhibition after ticagrelor making patients prone to thrombosis or bleeding. If doctors had known what happened in these trials, they would never have started using ticagrelor.”

Circulation and AstraZeneca did not respond to a request for comment.

Serebruany added: “It’s been obvious for years that there is something wrong with the data. That the FDA’s leadership could look past all these problems—on top of the many problems their own reviewers identified and are now being discovered by The BMJ—is unconscionable. We all need to know how and why that happened.”

Reference: “Ticagrelor doubts: inaccuracies uncovered in key studies for AstraZeneca’s billion dollar drug” by Peter Doshi, 19 June 2025, BMJ.
DOI: 10.1136/bmj.r1201

Funding: BMJ Investigations Unit

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