dm+d

Unassigned

New Medicines

Advanced solid tumours with FGFR1-4 genetic aberration, including cholangiocarcinoma

Information

New molecular entity
Taiho Oncology
Taiho Oncology

Development and Regulatory status

Phase II Clinical Trials
Pre-registration (Filed)
Pre-registration (Filed)
Yes
Jun 22Filed in EU; presumably for this indication [5].
Mar 22FDA accepts for priority review a NDA for futibatinib in the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring FGFR2 gene rearrangements, including gene fusions. PDUFA action date is 30/09/22 [4].
Feb 21FDA grants breakthrough therapy status for futibatinib for the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene rearrangements, including gene fusions. This designation was granted on the basis of safety and efficacy of phase II FOENIX-CCA2 study [1,3].
May 18Has orphan drug status in US [3].

Category

Irreversible covalently-binding FGFR inhibitor of fibroblast growth factor receptors 1, 2, 3 and 4 (reduces tumour cell proliferation)
Cholangiocarcinoma (CCA), also known as bile duct cancer, is not common. About 8,000 people in the U.S. are diagnosed with CCA each year.4 This includes both intrahepatic (inside the liver) and extrahepatic (outside the liver) cancers. CCA can occur at younger ages, but it is seen mainly in older people. Five year survival is 9% [1].
Advanced solid tumours with FGFR1-4 genetic aberration, including cholangiocarcinoma
Oral

Trial or other data

Apr 21PIII FOENIX-CCA3 trial is recruiting. In the experimental arm, patients will receive futibatinib at an oral dose of 20 mg, administered daily (QD) on every day of a 21-day cycle. In the control arm, on days 1 and 8 of a 21-daycycle, patients will receive cisplatin 25 mg/m2 in 1000 mL 0.9% saline by intravenous (I.V.) infusion over 1 hour, followed by 500 mL 0.9% saline over 30 minutes; and gemcitabine 1000 mg/m2 in 250-500 mL 0.9% saline by I.V. infusion over 30 minutes, beginning after completion of the cisplatin and saline infusions [2].
Jan 20PIII FOENIX-CCA3 trial to evaluate futibatinib versus gemcitabine-cisplatin chemotherapy as first-line treatment of patients with advanced cholangiocarcinoma harboring FGFR 2 gene rearrangements FOENIX-CCA3 starts (NCT04093362). The primary endpoint is progression-free survival (PFS). Secondary endpoints include objective response rate (ORR) and disease control rate and safety. The randomised, open label trial intends to enrol 216 patients in the US. Collection of primary outcome data is due to complete Apr 25 [3].
Jul 14PI/II FOENIX-101 trial to evaluate TAS 120 in patients with advanced solid tumours harbouring FGF/FGFR aberrations starts (FOENIX-CCA2; NCT02052778). The open-label trial will enrol 386 patients in the US, UK, France, Australia, Hong Kong, Taiwan, Netherlands, Japan, South Korea, Spain, Germany and Portugal [2].