FDA approves Truseltiq for advanced cholangiocarcinoma
The FDA granted accelerated approval to infigratinib for previously treated locally advanced or metastatic cholangiocarcinoma that harbors FGFR2 fusion or rearrangement.
Infigratinib (Truseltiq; BridgeBio Pharma/QED Therapeutics, Helsinn Group) is an oral FGFR1-3 selective inhibitor in development for people with FGFR-driven conditions, including cholangiocarcinoma, urothelial carcinoma and achondroplasia.
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.
Approximately 20,000 people in the United States are diagnosed each year with cholangiocarcinoma, an often-fatal cancer of the bile ducts.
An estimated 15% to 20% of patients have FGFR2 genetic aberrations. Treatment options for these patients are limited, and only about 9% survive 5 years.
The FDA based approval on results of a phase 2 study that included 108 patients with advanced unresectable cholangiocarcinoma, all of whom had received at least one prior line of systemic therapy.
Researchers reported a confirmed objective response rate of 23% (95% CI, 16-32) and a median response duration of 5 months (95% CI, 3.7-9.3).
Common adverse events 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.
“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 company-issued 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.”
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