SUMMARY

• There are no published data describing the efficacy and safety of BALVERSA in patients with hepatocellular carcinoma.
• RAGNAR (NCT04083976) is an ongoing, phase 2, open-label, single-arm, multicenter study evaluating the efficacy and safety of BALVERSA in adult and pediatric patients (children aged ≥6 years) with unresectable, locally advanced, or metastatic solid tumor malignancies, fibroblast growth factor receptor (FGFR) mutations or gene fusions, and documented disease progression. Patients must have received ≥1 prior line of systemic therapy in the advanced, unresectable, or metastatic setting; or is a child or adolescent with a newly diagnosed solid tumor and no acceptable standard therapies. The study enrolled 316 patients from 172 global study sites and is closed for the recruitment of both adult and pediatric patients. Safety and efficacy results in hepatocellular carcinoma from the RAGNAR study have not been published.^1,2 For complete study details, refer to: https://clinicaltrials.gov/ct2/show/NCT04083976.²
  • Lugowska et al (2024)³ reported results from an exploratory cohort of patients with FGFR mutations that were not predefined as potentially susceptible alterations (n=53). Of the 53 patients, 1 had hepatocellular cancer. Efficacy and safety were not separately evaluated for patients with hepatocellular carcinoma.
• A phase 1/2a study (NCT02421185) evaluating BALVERSA for the treatment of advanced hepatocellular carcinoma has completed patient recruitment; however, no efficacy or safety results are available. For complete study details, refer to https://clinicaltrials.gov/ct2/show/NCT02421185.⁴

Literature Search

A literature search of MEDLINE^®, Embase^®, BIOSIS Previews^®, and Derwent Drug Files (and/or other resources, including internal/external databases) was conducted on 28 May 2024.