WASHINGTON, D.C. — The U.S. Food and Drug Administration announced Tuesday it is reviewing the 2022 approval of Tavneos, a drug for rare blood‑clotting disorders, after an internal audit flagged possible manipulation of pivotal trial data.

The agency’s Office of Compliance said a review of the Phase III trial, which enrolled 1,200 patients across 15 sites, uncovered discrepancies in reported endpoints. “We have identified irregularities that could affect the integrity of the data supporting the drug’s efficacy,” said FDA spokesperson Dr. Maria Alvarez. The review does not immediately suspend the drug’s market status.

Manufacturers of Tavneos, BioPharm Inc., maintain that the product remains safe and effective for patients who have exhausted other options. In a statement, the company cited a post‑marketing safety record that includes 3,500 patient‑years of exposure with no new safety signals. “Our commitment to patients is unwavering, and we will cooperate fully with the FDA’s investigation,” the release read.

Patient advocacy groups warn that a sudden withdrawal could leave thousands without therapy. The Rare Disease Alliance estimates that 12,000 Americans rely on Tavneos for conditions such as atypical hemolytic‑uremic syndrome. “Access to treatment is critical; any disruption must be managed carefully to avoid harm,” said alliance director James Liu.

Key Takeaways

• FDA is re‑examining the 2022 approval of Tavneos after finding data irregularities in a 1,200‑patient trial.
• BioPharm asserts the drug’s safety record remains solid, with over 3,500 patient‑years of exposure post‑approval.
• Approximately 12,000 U.S. patients could be affected if access to the drug is limited.

Regulators have not yet decided whether to revoke Tavneos’s approval, but they have placed the drug under heightened surveillance. The FDA says a final determination will be made after a thorough evaluation, which could take several months. In the meantime, physicians are advised to continue prescribing the medication while monitoring patients closely.