New Investigation Raises Doubts Over Safety and Approval of Ticagrelor
A recent investigation has uncovered troubling evidence that could shake the foundation of how the blockbuster heart drug ticagrelor was approved — and how it’s been used for over a decade.
Published by The BMJ , the report raises fresh concerns about key studies that were used to support ticagrelor’s approval by the U.S. Food and Drug Administration (FDA) , especially in treating patients with acute coronary syndrome (ACS) — a group of conditions caused by sudden reductions in blood flow to the heart.
Sold under the brand names Brilinta in the U.S. and Brilique in Europe, ticagrelor has been widely prescribed for years and has generated billions in revenue for pharmaceutical giant AstraZeneca .
🧾 Past Doubts Resurface: Data Integrity in PLATO Trial Questioned
Back in December, The BMJ revealed serious flaws in the PLATO clinical trial , which was central to ticagrelor’s global regulatory approvals. The findings cast doubt on whether the drug truly offered better outcomes compared to cheaper alternatives like clopidogrel.
Now, as generic versions of ticagrelor are set to hit the market this year, The BMJ has expanded its inquiry into two critical platelet function studies that AstraZeneca submitted to regulators to prove the drug’s effectiveness.
The results are raising even more red flags.
🔍 Missing Data and Authorship Confusion
According to the new report, the primary endpoint results from both trials were inaccurately reported in the journal Circulation , one of the most respected publications in cardiology.
More alarmingly:
- Over 60 out of 282 platelet test readings were missing from the datasets reviewed by the FDA
- One investigator who actively participated in the trial was never listed as an author
- Another listed author told The BMJ he had no involvement in the study at all
- Most researchers, including the principal investigator, either couldn’t be reached or declined to comment
This lack of transparency is fueling growing skepticism among experts.
👨⚕️ Expert Voices Concerns About Patient Safety
Dr. Victor Serebruany , an adjunct faculty member at Johns Hopkins University and a long-time critic of ticagrelor, expressed strong concerns over the implications of these findings.
He explained:
“There have been clear signs of abnormal platelet activity linked to ticagrelor — including dangerous rebounds and extreme inhibition that can lead to bleeding or blood clots. If doctors had known what really happened in these trials, they may never have started prescribing this drug.”
He also criticized the FDA’s handling of the situation:
“It’s been obvious for years that something was off with the data. That the FDA ignored so many issues — some flagged even by their own reviewers — is unacceptable. We deserve a full explanation.”
📞 Silence from Key Players
Neither Circulation nor AstraZeneca responded to The BMJ ‘s requests for comment, adding to the frustration surrounding the lack of accountability.
🧪 What This Means for Patients and Doctors
As ticagrelor’s patent expires and generics become available, questions about the true effectiveness and safety of the drug are becoming harder to ignore.
While the drug has helped many patients, this ongoing investigation highlights the importance of transparent clinical research and the need for regulatory agencies to thoroughly review the data behind major medications .
Doctors and patients alike should be aware of these developments — especially as decisions about treatment options continue to rely heavily on past trial results.
The full investigative report, titled “Ticagrelor Doubts: Inaccuracies Uncovered in Key Studies for AstraZeneca’s Billion-Dollar Drug,” was authored by Peter Doshi and published on June 19, 2025 , by The BMJ .
🔗 Read the full article here
This investigation was conducted by the BMJ Investigations Unit , an independent team dedicated to uncovering issues related to medical research integrity and pharmaceutical transparency.
