Elevar Therapeutics announced that the FDA has accepted for review the company’s New Drug Application (NDA) for lirafugratinib as a treatment for patients with previously treated cholangiocarcinoma (CCA) harboring FGFR2 fusions or rearrangements. The agency has granted the application Priority Review and set a Prescription Drug User Fee Act (PDUFA) target action date of September 27, 2026.
Lirafugratinib, an investigational therapy, demonstrated a confirmed objective response rate (ORR) of 46.5% in the Phase 1/2 ReFocus trial (NCT04526106) among patients with FGFR2 fusion-positive CCA. The drug’s safety profile was described as predictable and manageable with dose adjustments.
Priority Review designation is reserved for therapies that may represent significant improvements in the treatment or safety of serious conditions, according to the FDA.
“Lirafugratinib has established a compelling clinical profile that differentiates it from existing treatment options,” said Dong-Gun Kim, chief executive officer of Elevar. “We are very pleased with the FDA’s priority review designation and focused on advancing the review process efficiently to bring this therapy to patients as quickly as possible.”
Cholangiocarcinoma, or bile duct cancer, is a rare malignancy affecting approximately 8,000 people annually in the United States. Elevar is also evaluating lirafugratinib in additional studies across other FGFR2-altered solid tumors.
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