On May 28, 2021, the FDA granted accelerated approval to infigratinib (Truseltiq; QED Therapeutics, Inc), an oral FGFR1-3 selective inhibitor, for the treatment of adults with previously treated locally advanced or metastatic cholangiocarcinoma (CCA) with an FGFR2 fusion or other rearrangement. The FDA previously granted infigratinib orphan drug and fast-track designations.
The FDA also approved FoundationOne CDx (Foundation Medicine Inc) as a companion diagnostic to help select patients with FGFR2 fusion or other rearrangement who may benefit from infigratinib treatment.
This approval was based on a phase 2 study of 108 patients with previously treated, unresectable locally advanced or metastatic CCA with an FGFR2 fusion or arrangement as determined by local or central testing. The study’s primary outcomes were overall response rate and duration of response. The overall response rate was 23% and the median duration of response was 5 months.
The most common (≥20%) adverse reactions included increased creatinine, increased phosphate, decreased phosphate, nail toxicity, stomatitis, increased alkaline phosphatase, decreased hemoglobin, increased alanine aminotransferase, dry eye, fatigue, increased lipase, decreased lymphocytes, increased calcium, decreased sodium, alopecia, increased triglycerides, increased aspartate aminotransferase, decreased platelets, increased urate, palmar-plantar erythrodysesthesia syndrome, arthralgia, and dysgeusia.