FDA Approves New Option for Previously Treated Bile Duct Cancer
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The FDA approved the oral fibroblast growth factor receptor 2 (FGFR2) inhibitor lirafugratinib (Lyrfigtu) for adults with previously treated cholangiocarcinoma (CCA) with FGFR2 fusion or other rearrangements, the agency announced Wednesday.
The approval stipulates use for unresectable, locally advanced, or metastatic CCA (also called bile duct cancer). About 8,000 new cases are diagnosed in the United States each year.
“Lyrfigtu’s unique, irreversible, covalent-binding mechanism enables potent and sustained inhibition of FGFR2, including many resistance mutations that can emerge with earlier FGFR inhibitors,” said Lipika Goyal, MD, of Stanford Cancer Center in California, in a news release from Elevar Therapeutics. “Unlike earlier pan-FGFR inhibitors, it selectively targets FGFR2 while minimizing off-isoform toxicities.”
Principal support for the approval came from the phase I/II ReFocus trial. Data analysis included 116 patients with previously treated FGFR2-positive CCA and no prior exposure to an FGFR inhibitor. Treatment led to an objective response rate of 46%, median progression-free survival (PFS) of 11.3 months (49.2% 12-month PFS), median overall survival (OS) of 22.8 months (12-month OS of 74.6%), and disease control rate of 96.5%.
“These results represent a meaningful step forward for patients with FGFR2-fusion-positive CCA, a disease where treatment options have historically been limited and outcomes poor,” said Alison Schram, MD, of Memorial Sloan Kettering Cancer Center in New York City. “Lyrfigtu demonstrated durable responses and a manageable safety profile, providing a much-needed treatment option for patients. The results also reinforce the importance of molecular testing at diagnosis so that patients can be matched to therapies most likely to benefit them.”
The most common grade ≥3 treatment-related adverse events were palmar-plantar erythrodysesthesia (32.8%) and stomatitis (12.1%). According to the FDA, prescribing information includes warnings and precautions for ocular toxicity, hyperphosphatemia, soft tissue mineralization, and embryo-fetal toxicity.
According to Elevar, lirafugratinib will be available in the U.S. before the end of the year.
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