Taiho Oncology Europe GmbH announced the UK launch of Lytgobi^® (futibatinib), an irreversibly binding fibroblast growth factor receptor (FGFR) inhibitor.^1,3 This development follows the conditional marketing authorisation (4 September 2024) and subsequent National Institute for Health and Care Excellence (NICE) recommendation (11 September 2024) for futibatinib monotherapy as an option for the treatment of eligible adult patients with locally advanced or metastatic cholangiocarcinoma (CCA) with a FGFR2 fusion or rearrangement that have progressed after at least one prior line of systemic therapy.^1,4

CCA is an aggressive cancer of the bile ducts that carry bile inside the liver and from the liver and gallbladder to the small intestine. In England, approximately 3,110 people were diagnosed with CCA in 2023,^5,6 and the disease is growing in incidence worldwide.⁷ CCA is usually diagnosed at an advanced stage, with a very poor prognosis with less than one in five patients surviving five years, and there is an unmet need for additional treatment options such as futibatinib.^7,8

“Patients with CCA are mostly identified at a late stage of disease and consequently have a poor prognosis. The launch of futibatinib in the UK and positive NICE recommendation for treating locally advanced or metastatic CCA with a FGFR2 fusion or rearrangement that has progressed after at least one prior line of systemic treatment in adults represents a much needed additional treatment option for eligible patients with this aggressive cancer,” said Professor John Bridgewater, Clinical Researcher and Medical Oncologist at University College London Cancer Institute and University College London Hospitals NHS Foundation Trust.

Lytgobi monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or rearrangement that have progressed after at least one prior line of systemic therapy.¹ The medicine is taken orally, once daily, on a continuous basis (treatment should be continued until disease progression or unacceptable toxicity).¹

The conditional marketing authorisation^*, where UK approval is granted for medicines that fulfil an unmet medical need while regulatory data review is ongoing,⁹ is based on findings from the pivotal Phase 2 FOENIX-CCA2^‡ global open-label, single-arm trial evaluating 103 patients with unresectable, locally advanced or metastatic CCA with a FGFR2 fusion or rearrangement.^1,10 The trial showed that futibatinib provided a clinically meaningful benefit, meeting its primary endpoint of objective response (partial or complete response as assessed by independent central review) with a rate of 42% (95% CI, 32–52), and a median duration of response of 9.7 months (95% CI, 7.6–17.0).^10,11 Among the secondary endpoints, the 12-month overall survival rate was 72% (95% CI, 62–80).¹⁰

The most common (≥20%) adverse reactions were hyperphosphatemia (89.7%), nail disorders (44.1%), constipation (37.2%), alopecia (35.2%), diarrhoea (33.8%), dry mouth (31.0%), fatigue (31.0%), nausea (28.3%), dry skin (27.6%), increased AST (26.9%), abdominal pain (24.8%), stomatitis (24.8%), vomiting (23.4%), palmar-plantar erythrodysaesthesia syndrome (22.8%), arthralgia (21.4%), and decreased appetite (20.0%).¹

The results of the FOENIX-CCA2 trial were published in The New England Journal of Medicine in 2023.¹⁰ Within Europe, eligible patients were enrolled in this trial from France, Germany, Italy, the Netherlands, Spain and the United Kingdom.¹⁰

“The launch of futibatinib in the UK is the culmination of extensive and productive collaboration among researchers, healthcare professionals, and patients, all united in their commitment to advance care in cholangiocarcinoma and to improve the lives of eligible patients,” said Dr. Peter Foertig, General Manager of Taiho Oncology Europe. “This marks the first medicine Taiho Oncology has introduced in the UK on our own, signifying our intention to establish a strong presence and reinforcing our ongoing commitment to bringing innovative oral therapies to patients with cancer.”

Futibatinib was developed by Taiho Oncology Europe’s parent company, Taiho Pharmaceutical Co., Ltd., Tokyo. The larger Taiho family of companies continues to investigate its potential in other tumours.¹²

▼ This medicine is subject to additional monitoring. This will allow quick identification of new safety information.

^* A conditional marketing authorisation is granted for medicines that fulfil an unmet medical need to treat serious diseases, and the benefits of having them available earlier outweigh any risks associated with using the medicines while waiting for further evidence. As part of this authorisation there will be ongoing review of data to ensure the benefit-risk balance remains positive.⁹

^‡ The FOENIX-CCA2 trial is a Phase 1 / 2 Study of TAS-120 in Patients with Advanced Solid Tumors Harboring FGF/FGFR Aberrations: FGFR Oral SElective Novel Inhibitor X [across] tumors.