Futibatinib Receives Accelerated FDA Approval for Intrahepatic Cholangiocarcinoma
Futibatinib (Lytgobi, Taiho Oncology, Inc.) received accelerated FDA approval on 30 September 2022 for adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma positive for a fibroblast growth factor receptor 2 (FGFR2) gene mutation.
Bile duct cancers are called cholangiocarcinoma in medical terminology. At diagnosis, most patients with cholangiocarcinoma have advanced-stage disease, meaning that the disease can no longer be treated surgically. A combination of chemotherapy drugs has been the standard initial treatment for these patients. FGFR2 genetic alterations (such as fusions) have been found in the tumours of approximately 9% to 14% of patients with cholangiocarcinoma. Futibatinib became the 3rd targeted drug to receive FDA approval targeting FGFR2 in cholangiocarcinoma. Previously, infigratinib in May 2021 and pemigatinib in April 2020 had received FDA approval for this indication.
The efficacy of futibatinib was evaluated in TAS-120-101, a multicentre, open-label, single-arm trial involving 103 patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harbouring an FGFR2 gene fusion or another rearrangement. The presence of FGFR2 fusions or other rearrangements was established using next-generation sequencing (NGS) genetic testing. Patients received 20 mg futibatinib once daily until disease progression or unacceptable adverse effects.
The main efficacy outcome measures were overall response rate and duration of response, determined by an independent review committee.
- The overall response rate was 42% (95% confidence interval [CI]: 32, 52).
- All 43 patients who responded to treatment achieved partial responses.
- The median duration of response was 9.7 months (95% CI: 7.6, 17.1).
The most common adverse effects, occurring in 20% or more of patients, were nail toxicity, musculoskeletal pain, constipation, diarrhoea, fatigue, dry mouth, hair loss, stomatitis (mouth sores), abdominal pain, dry skin, arthralgia (joint pain), taste disturbance, dry eyes, nausea, decreased appetite, urinary tract infection, palmar-plantar erythrodysesthesia syndrome and vomiting.
The recommended futibatinib dose is 20 mg orally once daily until disease progression or unacceptable adverse effects.
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