Taiho Oncology Europe GmbH and Taiho Pharmaceutical Co., Ltd., announced today that the European Commission has granted conditional marketing authorization for LYTGOBI® (futibatinib) monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma (CCA) with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.

LYTGOBI is a kinase inhibitor indicated for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.

Biliary Tract Cancers...Useful in Certain Circumstances...For CCA with FGFR2 fusions or rearrangements...Futibatinib

...Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd. announced today that the U.S. Food and Drug Administration (FDA) has accepted for priority review the New Drug Application (NDA) for futibatinib in the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring FGFR2 gene rearrangements, including gene fusions. Futibatinib is an investigational, oral, potent, selective and irreversible small-molecule inhibitor of FGFR1, 2, 3 and 4. The FDA provided an anticipated Prescription Drug User Fee Act (PDUFA) action date of September 30, 2022.

Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd. today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for futibatinib (TAS-120), a covalently-binding FGFR inhibitor, for the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene rearrangements, including gene fusions.

...Intrahepatic or extrahepatic cholangiocarcinoma with FGFR2 gene fusions or other FGFR2 abnormalities, i.e., gene mutations (see Appendix A), rearrangements or amplifications...

Taiho Oncology Europe GmbH and Taiho Pharmaceutical Co., Ltd. announced today that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the conditional marketing authorization (CMA) of futibatinib for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma (CCA) with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy....The positive CHMP opinion on futibatinib is based on data from the pivotal Phase 2 FOENIX*-CCA2 trial...

At a median follow-up of 17.1 months, the median progression-free survival was 9.0 months and overall survival was 21.7 months….In previously treated patients with FGFR2 fusion or rearrangement–positive intrahepatic cholangiocarcinoma, the use of futibatinib, a covalent FGFR inhibitor, led to measurable clinical benefit.

The confirmed ORR was 41.7% (43/103) and thereby the same as of the primary analysis, as was the DCR (at 82.5%). The ORR was consistent across pt subgroups. The mDOR was 9.5 mo, and 74% of responses lasted ≥6 mo. mPFS was 8.9 mo, with a 12-mo PFS rate of 35.4%. Mature mOS was 20.0 mo, with a 12-mo OS rate of 73.1%....Findings from the final analysis of FOENIX-CCA2 confirm the results of the primary analysis and reinforce the durable efficacy and continued tolerability of futibatinib in previously treated pts with advanced/metastatic iCCA harboring FGFR2 fusion/rearrangements.

...futibatinib provides longer survival vs chemotherapy among previously treated advanced iCC patients with FGFR2 fusions/rearrangements.

Futibatinib demonstrated an objective response rate (ORR) of 13.7%, with responses in a broad spectrum of tumors (cholangiocarcinoma and gastric, urothelial, central nervous system, head and neck, and breast cancer) bearing both known and previously uncharacterized FGFR1–3 aberrations. The greatest activity was observed in FGFR2 fusion/rearrangement–positive intrahepatic cholangiocarcinoma (ORR, 25.4%).

The study met its primary objective with a confirmed ORR of 41.7% (43/103). Responses were durable, with a median (m) DOR of 9.7 mo and 72% of responses ≥6 mo. DCR was 82.5%. mPFS was 9.0 mo; mOS was 21.7 mo, with a 12-mo OS rate of 72%...ORR was consistent in pts with FGFR2 fusions (43.8%) and other FGFR2 rearrangements (34.8%) and in pts with BICC1 (41.7%)...Futibatinib resulted in frequent, durable objective responses in pts with iCCA harboring FGFR2 fusion/rearrangements

ORR was 37.3%, DCR was 82.1%, and median DOR was 8.3 mo. Objective responses occurred regardless of baseline characteristic (subgroup: ≥65 y, ORR: 57.1%), FGFR2 fusion partner (BICC1, 33.3%), or other genetic mutation (TP53, 16.7%). Median PFS was 7.2 mo. Futibatinib resulted in durable objective responses in pts with iCCA and FGFR2 fusions/rearrangements, including within pt subgroups.

FOENIX-CCA2 (NCT02052778), a single-arm multicenter phase 2 study, enrolled pts with locally advanced/metastatic unresectable iCCA harboring FGFR2 gene fusions or other rearrangements...Pts received futibatinib 20 mg QD until disease progression/unacceptable toxicity….Of these, 82.1% of pts had tumors harboring an FGFR2 fusion...ORR was 34.3% (all partial response, n = 23)…