Infigratinib Granted Accelerated Approval for FGFR2+ Cholangiocarcinoma

Objective response data from a phase 2 trial supported the FDA’s decision to grant infigratinib accelerated approval to treat patients with certain a certain type of cholangiocarcinoma.

The FDA has granted accelerated approval to the kinase inhibitor infigratinib (Truseltiq) as therapy for patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements, according to the company responsible for the agent, BridgeBio Parma, Inc.

Data supporting the decision were from the multicenter, open-label, single-arm phase 2 CBGJ398X2204 (NCT02150967) trial in 108 patients who had received at least 1 prior therapy to receive daily infigratinib at 125 mg for 21 days of 28-day cycles.

“This is an important milestone for patients diagnosed with FGFR2-fusion-driven cholangiocarcinoma who have recurred after first-line therapy and are in need of targeted options for further treatment,” Susan Moran, MD, MSCE, Chief Medical Officer for QED, said in a press release. “Based on the efficacy seen to date, our team believes infigratinib possesses promise for a range of FGFR-driven conditions, including other cancers. We will continue to evaluate its safety and efficacy in these areas of unmet need.”

Data from the trial were presented at the 2021 Gastrointestinal Cancers Symposium and showed the agent was capable of eliciting a objective response rate per blinded independent review of 23.1% (95% CI, 15.6%-32.2%), which was comprised of complete response and 24 partial responses; 66 participants achieved stable disease with the agent, while 11 experienced disease progression. The median duration of response (DOR) was 5.0 months (95% CI, 0.9-19.1).

Most adverse effects (AEs) reported with infigratinib were grade 1 or 2 in severity and were mostly reversible and easily managed. The most frequently reported AEs were mechanism based including calcium phosphate homeostasis (85.2%), tissue calcification (2.8%), pathological fracture (0.9%), vascular calcification/mineralization (0.9%), and eye disorders (70.4%). Central serous retinopathy/retinal pigment epithelial detachment–like events occurred in 16.7% of patients.

“While targeted treatments have extended survival for many types of cancer, people diagnosed with cholangiocarcinoma have previously been presented with extremely limited treatment options coupled with low statistical survival data,” Milind Javle, MD, professor of Gastrointestinal Medical Oncology at The University of Texas MD Anderson Cancer Center, said in a press release. “In this study, Truseltiq showed promise as a targeted treatment option for patients with FGFR2-fusion-driven cholangiocarcinoma with a well-tolerated safety profile in line with previous observations in this patient population.”

Continued approval of this medication may be based on verification in a phase 3 trial. Currently, safety and efficacy of infigratinib is being explored in a phase 3 trial (NCT03773302) versus with standard-of-care gemcitabine or cisplatin in the frontline treatment of patients with unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or translocations.


BridgeBio Pharma’s Affiliate QED Therapeutics and Partner Helsinn Group Announce FDA Approval of Truseltiq (infigratinib) for Patients with Cholangiocarcinoma. News release. BridgeBio Pharma, Inc. May 28, 2021. Accessed May 28, 2021.

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