FDA clears targeted cholangiocarcinoma therapy - Scorecard - MDSpire
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FDA clears targeted cholangiocarcinoma therapy

In the single-arm REFOCUS trial, 46% of patients responded to treatment, with a median response duration of 11.8 months.

  • By

  • Kathryn Wighton

  • September 24, 2026

  • 2 min

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Clinical Scorecard: FDA clears targeted cholangiocarcinoma therapy

At a Glance

CategoryDetail
ConditionCholangiocarcinoma
Key MechanismsKinase inhibitor targeting fibroblast growth factor receptor 2 gene fusion or rearrangement
Target PopulationAdults with unresectable, locally advanced or metastatic cholangiocarcinoma previously treated with chemotherapy or chemoimmunotherapy
Care SettingOncology

Key Highlights

  • Lirafugratinib approved for previously treated unresectable cholangiocarcinoma
  • Efficacy assessed in the REFOCUS trial with 116 patients
  • 46% objective response rate observed
  • Median response duration of 11.8 months
  • Safety warnings include ocular toxicity and hyperphosphatemia

Guideline-Based Recommendations

Diagnosis

  • Diagnosis of unresectable or metastatic cholangiocarcinoma confirmed

Management

  • Administer lirafugratinib at 70 mg once daily until disease progression or unacceptable toxicity

Monitoring & Follow-up

  • Monitor for ocular toxicity, hyperphosphatemia, and embryo-fetal toxicity

Risks

  • Potential for ocular toxicity and soft tissue mineralization

Patient & Prescribing Data

Adults with previously treated unresectable cholangiocarcinoma

Lirafugratinib is a targeted therapy with a significant response rate in this patient population

Clinical Best Practices

  • Consider genetic testing for fibroblast growth factor receptor gene fusions or rearrangements
  • Utilize independent review for assessing treatment response

Related Resources & Content

Original Source(s)

FDA clears targeted cholangiocarcinoma therapy

  • by Kathryn Wighton

  • September 24, 2026

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