FDA Approves Therapy for Bile Duct Cancer

The FDA has given accelerated approval to Taiho’s Lytgobi (futibatinib) tablets to treat adult patients with previously treated, unresectable, locally advanced or metastatic advanced bile duct cancer. It is approved to treat patients with the intrahepatic (inside the liver) form of the disease who have fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.

Cholangiocarcinoma is an aggressive, rare cancer of the bile ducts and is diagnosed in about 8,000 people each year in the United States, according to the American Cancer Society. About 20% of patients have the intrahepatic form of the disease. Within this 20%, about 10% to 16% of patients have FGFR2 gene rearrangements, including fusions, which promote tumor proliferation. Five-year relative survival data for the intrahepatic form of the disease is 9%, according to data from Surveillance, Epidemiology, and End Results (SEER) database, maintained by the National Cancer Institute (NCI).

“Lytgobi is a key example of the potential of precision medicine in iCCA and represents another advance in the treatment of this rare and challenging disease,” Lipika Goyal, M.D., of the Massachusetts General Hospital Cancer Center and lead investigator of the pivotal study, said in a press release. “I am encouraged that treatment options continue to expand and evolve for this disease through the dedicated efforts of many over several years.”

The approval of Lytgobi is based on the results of the primary analysis of the FOENIX-CCA2 trial, a global phase 2 open-label trial evaluating 103 patients with unresectable, locally advanced or metastatic iCCA harboring FGFR2 gene rearrangements. In this trial, patients received Lytgobi orally once daily at a dose of 20 mg until disease progression or unacceptable toxicity.

Updated data from this trial were released in June 2022 at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago.

The trial met its primary endpoint with an objective response rate of 41.7% as measured by independent central review. The median duration of response (DOR) was 9.7 months, with 74% of responses lasting at least six months. The most common treatment-related adverse events were hyperphosphatemia, alopecia, dry mouth, diarrhea, dry skin and fatigue. Most events were of mild or moderate intensity and manageable. There were two patients with grade 4 adverse events and four patients discontinued treatment. No treatment-related deaths occurred.