LYTGOBI (futibatinib): FDA granted accelerated approval for adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma.
On September 30th, 2022, FDA granted accelerated approval to LYTGOBI® (futibatinib) by Taiho Oncology, Inc., for adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma.
Efficacy was evaluated in TAS-120-101 (NCT02052778), a multicenter, global, phase 2 open-label, single-arm trial that enrolled 103 patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma harboring a FGFR2 gene fusion or other rearrangement. The presence of FGFR2 fusions or other rearrangements was determined using next generation sequencing testing. Patients received 20 mg of futibatinib orally once daily until disease progression or unacceptable toxicity.
The major efficacy outcome measures were overall response rate (ORR) and duration of response (DoR) as determined by an independent review committee according to RECIST v1.1. ORR was 42% (95% Confidence Interval [CI]: 32, 52); all 43 responders achieved partial responses. The median DoR was 9.7 months (95% CI: 7.6, 17.1).
Cholangiocarcinoma is an aggressive cancer of the bile ducts and is diagnosed in approximately 8,000 individuals each year in the U.S. This includes both intrahepatic (inside the liver) and extrahepatic (outside the liver) forms of the disease. Approximately 20% of patients diagnosed with CCA have the intrahepatic form of the disease. Within this 20%, approximately 10-16% of patients have FGFR2 gene rearrangements, including fusions, which promote tumor proliferation.
LYTGOBI covalently binds to FGFR2 and inhibits the signaling pathway. The other approved FGFR inhibitors are reversible ATP-competitive inhibitors. Additionally, the drug previously received breakthrough, orphan drug and priority review designations from the FDA.
source: Press Release