Futibatinib was granted priority review and breakthrough designation, the agency said in its announcement.
Futibatinib is indicated for use in adult patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.
The FDA noted that efficacy was evaluated in a multicenter, open-label, single-arm trial (known as TAS-120-101 [NCT02052778]), which involved 103 patients with such tumors. The presence of FGFR2 fusions or other rearrangements was determined using next-generation sequencing.
All the patients in this trial received futibatinib (20 mg orally once daily) until disease progression or unacceptable toxicity.
The overall response rate was 42% (95% confidence interval, 32%-52%), and all of the 43 patients who responded achieved partial responses.
The median duration of response was 9.7 months (95% CI, 7.6-17.1).
The most common adverse reactions that occurred in 20% or more of patients were nail toxicity, musculoskeletal pain, constipation, diarrhea, fatigue, dry mouth, alopecia, stomatitis, abdominal pain, dry skin, arthralgia, dysgeusia, dry eye, nausea, decreased appetite, urinary tract infection, palmar-plantar erythrodysesthesia syndrome, and vomiting.
The manufacturer noted in its announcement that futibatinib covalently binds to FGFR2 and inhibits the signaling pathway. The other approved FGFR inhibitors are reversible ATP-competitive inhibitors.
The company also provided some background information on the cancer.
As a whole, cholangiocarcinoma is an aggressive cancer of the bile ducts. It is diagnosed in approximately 8000 individuals each year in the United States, the company noted.
These cases include both intrahepatic (inside the liver) and extrahepatic (outside the liver) forms of the disease. Approximately 20% of patients diagnosed with cholangiocarcinoma have the intrahepatic form of the disease. Among these 20%, approximately 10%-16% of patients have FGFR2 gene rearrangements, including fusions, which promote tumor proliferation.
Futibatinib is “a key example of the potential of precision medicine in iCCA [intrahepatic cholangiocarcinoma] and represents another advance in the treatment of this rare and challenging disease,” said medical oncologist Lipika Goyal, MD, MPhil, of the Massachusetts General Hospital Cancer Center, Boston, and lead investigator of the pivotal study that supported the approval.
“I am encouraged that treatment options continue to expand and evolve for this disease through the dedicated efforts of many over several years,” she commented in the company’s press release.
A version of this article first appeared on Medscape.com.