FDA Grants Priority Review for Elevar's Lirafugratinib in Cholangiocarcinoma
The FDA has granted Priority Review for lirafugratinib, an FGFR2 inhibitor for second-line cholangiocarcinoma treatment, with a PDUFA date of September 27, 2026. Clinical trial data showed a 46.5% objective response rate and 11.8-month median duration of response. The therapy demonstrated a manageable safety profile with lower rates of common FGFR inhibitor side effects.
The U.S. Food and Drug Administration has accepted for review the New Drug Application for lirafugratinib, an investigational therapy for patients with cholangiocarcinoma with FGFR2 fusions or rearrangements who have received prior therapy. The FDA granted Priority Review and set a Prescription Drug User Fee Act target action date of September 27, 2026, for the approval decision.
The Priority Review designation was supported by positive clinical data from the Phase 1/2 ReFocus trial, which demonstrated a confirmed objective response rate of 46.5% in patients with the proposed indication. In the Phase 2 ReFocus clinical trial conducted in patients with cholangiocarcinoma, lirafugratinib achieved a median duration of response of 11.8 months, representing the highest outcomes reported to date for a second-line treatment compared with previously approved therapies.
Its safety profile in the clinical data has been shown to be predictable and manageable through dose adjustments. Notably, the incidence rates of hyperphosphatemia and diarrhea, recognized as common side effects of pan-FGFR inhibitors, were 20.7% and 21.6%, respectively, which are markedly lower than those reported for previously approved FGFR inhibitors.
Lirafugratinib is a potent, selective, and oral small molecule inhibitor of FGFR2, a receptor tyrosine kinase that is frequently altered in certain cancers. The company continues to evaluate lirafugratinib in ongoing clinical development programs, including studies in other FGFR2-altered solid tumors. A global clinical trial is underway to advance the candidate as a 'tumor-agnostic' treatment, with the objective of achieving key clinical endpoints by 2027.
Cholangiocarcinoma, also known as bile duct cancer, is rare, with about 8,000 people in the U.S. diagnosed each year. Priority Review designations are given to applications for medicines that, if approved, would lead to "significant improvements in the safety or effectiveness of the treatment" of a serious condition, according to the FDA.