Elevar Therapeutics Wins FDA Approval for Lyrfigtu

Fort Lee, New Jersey, September 23, 2026

Lyrfigtu Targets FGFR2-Altered Cholangiocarcinoma

Lyrfigtu (lirafugratinib) is an oral, selective small-molecule inhibitor of FGFR2, a receptor tyrosine kinase that can become altered in certain cancers. The FDA-approved indication specifically covers patients whose cholangiocarcinoma contains an FGFR2 fusion or other rearrangement, making molecular testing an important component of identifying patients who meet the approved treatment criteria. Cholangiocarcinoma, commonly known as bile duct cancer, is a rare malignancy, with approximately 8,000 new cases diagnosed annually in the United States according to Elevar. The recommended dosage of Lyrfigtu is 70 mg orally once daily, administered until disease progression or unacceptable toxicity. By selectively targeting FGFR2 signaling, the medicine is designed to interfere with a pathway involved in tumor growth in cancers carrying relevant FGFR2 alterations. Elevar holds an exclusive license to lirafugratinib from Relay Therapeutics for commercialization worldwide.

ReFocus Trial Demonstrates Antitumor Activity

The FDA evaluated Lyrfigtu using data from REFOCUS (NCT04526106), a multicenter, open-label, single-arm Phase 1/2 study. The FDA’s efficacy assessment included 116 patients with unresectable or metastatic cholangiocarcinoma who had previously received chemotherapy or chemoimmunotherapy and had not received an FGFR inhibitor. Patients received lirafugratinib at 70 mg once daily until disease progression or unacceptable toxicity. The major efficacy measures were objective response rate (ORR) and duration of response assessed by an independent review committee according to RECIST version 1.1. The trial recorded an ORR of 46%, with a median duration of response of 11.8 months. These results formed the primary clinical evidence supporting the FDA’s approval. The manufacturer also reported a median progression-free survival of 11.3 months in the studied population. The FDA review also incorporated its Real-Time Oncology Review (RTOR) pilot program and an Assessment Aid submitted by the applicant. Lirafugratinib had previously received Priority Review, Breakthrough Therapy, and Orphan Drug designations, reflecting its development pathway for a serious disease with a defined molecular target and limited treatment options.

Safety Monitoring Remains Important

Alongside the efficacy findings, the FDA-approved prescribing information includes important warnings concerning ocular toxicity, hyperphosphatemia and soft-tissue mineralization, and embryo-fetal toxicity. Elevar reported that retinal pigment epithelial detachment occurred in 31% of 385 patients who received Lyrfigtu, including Grade 3 events in 1.8%. The prescribing information calls for comprehensive ophthalmologic assessment before treatment and continued monitoring during therapy, with additional evaluation if visual symptoms develop. Hyperphosphatemia occurred in 21% of patients in the reported safety population, requiring monitoring of serum phosphate during treatment. Other commonly reported adverse reactions included nail toxicity, palmar-plantar erythrodysesthesia syndrome, stomatitis, alopecia, dry eye, fatigue, dry mouth, dysgeusia, and gastrointestinal symptoms.

Elevar expects Lyrfigtu to become available to U.S. patients during the fourth quarter of 2026. The company is also continuing to evaluate lirafugratinib in other FGFR2-altered solid tumors, although any additional indications will require separate regulatory review and approval. The FDA approval therefore establishes Lyrfigtu as a new targeted treatment option for eligible patients with previously treated, FGFR2 fusion- or rearrangement-positive cholangiocarcinoma while further clinical development continues.

Source: Elevar Therapeutics press release