Why was Truseltiq discontinued? A look into the FDA approval withdrawal

October 6, 2025 •

2 min read

In May 2024, the FDA announced the final withdrawal of approval for Truseltiq (infigratinib), a targeted therapy for bile duct cancer, just three years after its accelerated approval. This decision was voluntary on the part of the drug's manufacturer and provides a compelling case study on the risks inherent in the accelerated approval pathway.

Quick Summary

Approval for the targeted cancer drug Truseltiq was withdrawn by the FDA following a voluntary request from its manufacturer, Helsinn Healthcare SA. The withdrawal was prompted by difficulties recruiting patients for a required confirmatory trial, which ultimately rendered continued commercialization unviable.

Key Points

Accelerated Approval Requirement: Truseltiq's initial FDA approval was accelerated and contingent upon the successful completion of a post-marketing confirmatory Phase 3 trial.
Clinical Trial Recruitment Failure: The primary reason for the withdrawal was the inability to recruit and enroll enough patients for the confirmatory Phase 3 PROOF-301 trial.
Commercial Viability Decision: The manufacturer, Helsinn Healthcare SA, deemed that continued distribution was no longer 'commercially reasonable' given the challenges and costs associated with the failed trial.
Voluntary Withdrawal: The withdrawal was initiated voluntarily by the drug's sponsor, rather than mandated by the FDA due to new safety or efficacy issues.
Molecule Still in Development: The active ingredient, infigratinib, is still being developed by BridgeBio for other conditions, such as achondroplasia, separate from its oncology application.
No New Safety Concerns: The discontinuation was explicitly not a result of unforeseen safety or efficacy concerns emerging from the already-approved indication.

What Was Truseltiq and Its Accelerated Approval?

Truseltiq (infigratinib) was a targeted therapy for previously treated, advanced cholangiocarcinoma with FGFR2 gene alterations, receiving accelerated FDA approval in May 2021. This approval was based on positive results from a Phase 2 trial. The accelerated approval pathway facilitates earlier access to promising drugs for serious conditions with limited treatment options but mandates confirmatory trials to verify clinical benefit.

The Unfinished Confirmatory Clinical Trial

A critical condition of Truseltiq's accelerated approval was the completion of a Phase 3 confirmatory trial, PROOF-301. This trial aimed to compare Truseltiq to standard chemotherapy in the first-line treatment of cholangiocarcinoma patients with FGFR2 alterations. However, the trial faced significant recruitment challenges, primarily due to the rarity of the specific genetic alteration, leading to its premature termination.

Commercial Viability and Voluntary Withdrawal

The inability to complete the PROOF-301 trial meant that the required data to confirm Truseltiq's clinical benefit over standard care could not be gathered. Consequently, the drug's sponsors, Helsinn Healthcare SA and its partners, determined that continued distribution was no longer commercially viable. This led to a voluntary request to the FDA to withdraw Truseltiq's approval, which the FDA granted in May 2024. The decision was influenced by the high costs and diminishing potential for success without sufficient trial enrollment, alongside an evolving competitive landscape in oncology.

Comparison of Truseltiq's Approval and Withdrawal


Aspect	Accelerated Approval (2021)	Reason for Withdrawal (2024)
Basis for Action	Positive data from a single-arm Phase 2 study.	Failure to complete a post-marketing Phase 3 confirmatory trial.
Indication	Previously treated, unresectable locally advanced or metastatic cholangiocarcinoma.	Inability to confirm benefit in the first-line setting, making the second-line market commercially unviable.
Regulatory Requirement	Obligation to conduct post-marketing confirmatory trials.	Non-fulfillment of the requirement to verify clinical benefit.
Trial Status	Completed a Phase 2 trial demonstrating efficacy.	Phase 3 PROOF-301 trial terminated due to recruitment issues.
Driving Factor	Promising early clinical results and unmet medical need.	Difficulties in patient recruitment and commercial non-viability.
Safety/Efficacy Impact	Not a factor in the withdrawal; no new safety concerns.	Not related to new adverse events, but rather a failure to confirm a benefit over standard care.

Future for the Molecule: Infigratinib for Achondroplasia

Despite the withdrawal of Truseltiq for cholangiocarcinoma, the active compound, infigratinib, is still being developed by BridgeBio for achondroplasia. This demonstrates the potential for a drug molecule to have applications in different diseases, independent of a prior failed development pathway.

Conclusion: A Cautionary Tale of Development Challenges

The discontinuation of Truseltiq underscores the challenges of drug development within the accelerated approval pathway. While this route provides timely access to treatments for serious conditions, it is contingent on successful confirmatory trials. Recruitment difficulties halted Truseltiq's trial, leading to a business decision to withdraw the drug. This highlights the rigorous regulatory process that requires verified clinical benefit for full approval. You can find more information about the FDA's decision on their website(https://www.fda.gov/drugs/resources-information-approved-drugs/withdrawn-fda-grants-accelerated-approval-infigratinib-metastatic-cholangiocarcinoma).

Frequently Asked Questions

Truseltiq (infigratinib) was a targeted therapy used to treat adults with previously treated, locally advanced or metastatic cholangiocarcinoma (bile duct cancer) that had an FGFR2 gene alteration.

Yes, Truseltiq showed a clinically meaningful response rate in a Phase 2 trial, which led to its accelerated approval. However, this initial evidence needed to be confirmed in a subsequent Phase 3 trial, which was never successfully completed.

The accelerated approval pathway allows promising drugs for serious conditions with unmet needs to be approved based on surrogate endpoints, like tumor shrinkage. Full approval is contingent on the sponsor conducting post-marketing confirmatory trials to verify the clinical benefit.

No, the discontinuation was not based on any new safety concerns. It was a business decision made by the manufacturer because of the inability to complete the required confirmatory clinical trial.

The manufacturer faced significant difficulties in recruiting and enrolling enough patients with the specific genetic alteration needed for the confirmatory Phase 3 PROOF-301 study.

Following the manufacturer's request, the drug was removed from the market. Patients were transitioned to alternative therapies under the supervision of their healthcare providers. The decision highlighted the limited treatment options for this patient population.

Yes. The molecule infigratinib is still in development for other conditions, including a Phase 3 trial for achondroplasia, a genetic disorder affecting bone growth.

The case of Truseltiq underscores the importance of successful confirmatory trials for drugs granted accelerated approval. It serves as a reminder that even promising early results need validation to secure long-term approval and market presence.