The Lynx Group

Truseltiq Receives FDA Approval for the Treatment of Patients with Metastatic Cholangiocarcinoma Harboring FGFR2 Alterations

August 2021, Vol 12, No 4

On May 28, 2021, the FDA accelerated the approval of infigratinib (Truseltiq; QED Therapeutics), an oral kinase inhibitor, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma (CCA) and FGFR2 fusion or other rearrangement, as detected by an FDA-approved test. The FDA granted infigratinib priority and fast-track review, as well as orphan drug designation for this indication.

On the same day, the FDA approved the FoundationOne CDx test as a companion diagnostic to infigratinib for selecting patients with CCA and FGFR2 fusion or other rearrangement for infigratinib treatment.

“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,” said Milind M. Javle, MD, Professor, Department of Gastrointestinal Medical Oncology, The University of Texas M.D. Anderson Cancer Center, Houston, TX. “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.”

The approval of infigratinib was based on results from CBGJ398X2204, a multicenter, open-label, single-arm clinical trial of 108 patients with previously treated, unresectable locally advanced or metastatic CCA and an FGFR2 fusion or rearrangement. Patients received infigratinib 125 mg once daily for 21 days, followed by 7 days off, in 28-day cycles, until disease progression or unacceptable side effects.

The primary end points were overall response rate (ORR) and duration of response (DOR). The ORR was 23% (95% CI, 16-32), including 1 complete response and 24 partial responses. The median DOR was 5 months (95% CI, 3.7-9.3). Among the 23 patients who responded to therapy, the DOR was ≥6 in 8 patients.

The most common (≥20%) adverse reactions were hyperphosphatemia, increased creatinine, nail toxicity, stomatitis, dry eye, fatigue, alopecia, palmar-plantar erythrodysesthesia syndrome, arthralgia, dysgeusia, constipation, abdominal pain, dry mouth, eyelash changes, diarrhea, dry skin, decreased appetite, blurred vision, and vomiting. Grade 3 or 4 adverse events included hyperphosphatemia and retinal pigment epithelial detachment.

This indication is approved under accelerated approval. Continued approval for this indication may be contingent on clinical benefit in confirmatory trial(s).

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