Cholangiocarcinoma Treatment Update: FDA Withdraws Accelerated Approval of Infigratinib

Cholangiocarcinoma, a type of cancer affecting the bile ducts, saw a significant development with the recent announcement from the US Food and Drug Administration (FDA) regarding the status of infigratinib (Truseltiq). This decision marks the withdrawal of the drug’s accelerated approval status for patients with previously treated cholangiocarcinoma. Infigratinib, a medication initially approved by the FDA for the treatment of adults with unresectable locally advanced or metastatic cholangiocarcinoma, marked a significant breakthrough in the management of this challenging cancer.

Its approval hinged upon specific criteria, particularly the presence of a fibroblast growth factor receptor 2 (FGFR2) fusion or another form of rearrangement, detectable through an FDA-approved test. This milestone approval not only provided a ray of hope for patients grappling with the complexities of cholangiocarcinoma but also represented the first and only approval for this medication in this therapeutic context.

Advancing Treatment Paradigms for Cholangiocarcinoma: The Case of Infigratinib

The FDA’s approval of infigratinib was a crucial step forward in addressing the unmet medical needs of individuals diagnosed with cholangiocarcinoma. However, this approval was conditional, necessitating QED Therapeutics, Inc., the drug’s manufacturer, to substantiate its clinical benefit through confirmatory trials. These trials were pivotal in corroborating the initial findings regarding the drug’s efficacy and safety profile, ensuring that patients received treatments that were not only promising but also rigorously evaluated and validated by scientific standards.

The requirement for confirmatory trials underscores the FDA’s commitment to upholding rigorous standards in the evaluation and approval of new therapies. While the initial approval of infigratinib offered a glimmer of hope for patients and their families, the mandate for confirmatory trials reflects the FDA’s dedication to ensuring that promising treatments undergo comprehensive assessment and validation. This multifaceted approach to drug evaluation is essential in safeguarding patient safety and promoting confidence in the therapeutic interventions offered within the realm of oncology and beyond.

Navigating Challenges in Cholangiocarcinoma Treatment: The Withdrawal of Infigratinib’s Accelerated Approval

Unfortunately, the circumstances surrounding the confirmatory trials led to the voluntary withdrawal of infigratinib’s accelerated approval. The decision stemmed from challenges encountered during the recruitment and enrollment of participants for the necessary confirmatory clinical trial. This trial aimed to investigate the drug’s efficacy in a new indication—treating cholangiocarcinoma in the first-line setting. However, due to difficulties in recruiting an adequate number of participants, the manufacturer concluded that it was not commercially viable to continue distributing the drug for its previously approved indication as a second-line treatment for cholangiocarcinoma.

This development underscores the complex nature of clinical research and the challenges inherent in conducting confirmatory trials. Despite the initial promise of infigratinib and its potential to address an unmet medical need in the cholangiocarcinoma patient population, the realities of clinical trial execution have led to the withdrawal of its accelerated approval status. Moving forward, stakeholders in the oncology community will continue to explore alternative treatment options and investigational therapies to improve outcomes for patients with cholangiocarcinoma.

Resource: Food and Drug Administration, May 17, 2024