On May 28, 2021, the FDA accelerated the approval of Truseltiq (infigratinib; from QED Therapeutics), a new oral kinase inhibitor, for the treatment of adults with previously treated, unresectable (cannot be removed by surgery) locally advanced or metastatic cholangiocarcinoma (a rare biliary tract cancer) and FGFR2 fusion or other rearrangement, as detected by an FDA-approved test.
This is the second targeted therapy approved by the FDA for patients with cholangiocarcinoma and the FGFR2 biomarker.
The approval of Truseltiq was based on results of a clinical trial of 108 patients with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma and an FGFR2 fusion or rearrangement. Patients received Truseltiq once daily for 21 days, followed by 7 days off, in 28-day cycles, until disease progression or unacceptable side effects.
The overall response rate to treatment with Truseltiq was 23%, including 1 complete response (no sign of cancer) and 24 partial responses. The average duration of response was 5 months, but among the patients who responded to therapy, 8 patients had a response that lasted 6 months or longer.
The most common side effects with Truseltiq were hyperphosphatemia, increased creatinine, nail side effects, stomatitis, dry eye, fatigue, hair loss, palmar-plantar erythrodysesthesia syndrome, arthralgia, dysgeusia, constipation, abdominal pain, dry mouth, eyelash changes, diarrhea, dry skin, decreased appetite, blurred vision, and vomiting.