What changed: Biliary tract cancer (all types)

When this page itself was last checked or edited.

HER2-positive (IHC 3+) unresectable locally advanced or metastatic biliary tract cancer after at least one line of systemic treatment; MHRA 19 February 2026, NICE TA1153 published 7 May 2026 with a commercial arrangement

OS 14.

Accelerated approval, previously treated HER2+ (IHC3+) biliary tract cancer (HERIZON-BTC-01) The confirmatory requirement was still open 1.8 years later, when the FDA's table was read.

Locally advanced, unresectable or metastatic biliary tract cancer with gemcitabine and cisplatin; NICE TA944 recommended 10 January 2024 with a commercial arrangement

HER2+ (IHC 3+) unresectable/metastatic biliary tract cancer, previously treated

OS 6.

Breakthrough Therapy designation, HER2+ biliary tract cancer

3-year overall survival 77.

Endometrial ORR 57.

Confirmed ORR by independent central review 41.

Biliary tract cancer with chemotherapy (TOPAZ-1)

Locally advanced or metastatic biliary tract cancer (gallbladder cancer included) with gemcitabine and cisplatin (TOPAZ-1)

ORR 9.

A milestone in how this cancer is treated.

BICR PFS 7.

Biliary cohort ORR 5.

Biliary cohort ORR 51% (22/43, investigator) and 47% (independent review); grade 3 or worse GGT rise 12%.

A milestone in how this cancer is treated.

Modified FOLFOX plus active symptom control modestly improved overall survival over active symptom control alone.

Response in 23 percent of the first 230 patients; trastuzumab plus pertuzumab gave a 32 percent response rate in HER2-amplified colorectal cancer, now a guideline option; atezolizumab activity rose with tumour mutational burden.

OS 51.

OS 11.

A milestone in how this cancer is treated.

A milestone in how this cancer is treated.