On September 23, 2026 Elevar Therapeutics, Inc., a majority-owned subsidiary of HLB Co., Ltd. and a fully integrated biopharmaceutical company dedicated to elevating treatment experiences and outcomes for cancer patients, reported the U.S. Food and Drug Administration (FDA) granted approval for LYRFIGTU (lirafugratinib) as a treatment option for patients with cholangiocarcinoma (CCA) with FGFR2 fusion or other rearrangement.
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CCA, also known as bile duct cancer, is rare, with about 8,000 people in the U.S. newly diagnosed each year, according to the American Cancer Society.
"The FDA approval of Lyrfigtu introduces a vital second-line treatment option for patients living with CCA and their families," said Dong-Gun Kim, chief executive officer of Elevar. "Our team is thrilled to bring this therapy to market and focused on getting Lyrfigtu to doctors and patients as quickly as possible, while continuing to deliver on our mission to improve treatment for patients whose therapeutic options were previously limited."
Lyrfigtu is expected to be available to patients in the U.S. by Q4 2026.
In the Phase 1/2 ReFocus trial (NCT04526106), Lyrfigtu demonstrated a confirmed objective response rate (ORR) of 46% and a median duration of response of 11.8 months in patients with the proposed indication. Median progression-free survival was 11.3 months (95% CI, 9.2, 14.8), with a 12-month rate of 49.2%. Its safety profile in the clinical data was shown to be predictable and manageable through dose adjustments.
Lyrfigtu in March 2026 was given a priority review designation by the FDA, which is reserved for drugs that if approved would lead to "significant improvements in the safety or effectiveness of the treatment" of a serious condition.
Expert Perspectives on Lyrfigtu Approval
Lipika Goyal, M.D., lead author on the ReFocus study and director of gastrointestinal oncology at the Stanford Cancer Center:
"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. And unlike earlier pan-FGFR inhibitors, it selectively targets FGFR2 while minimizing off-isoform toxicities. This FDA approval brings an important next-generation precision medicine option directly to patients with advanced bile duct cancer."
Robin Kate Kelley, M.D., professor of medicine in the division of Hematology & Oncology, Helen Diller Family Comprehensive Cancer Center, University of California, San Francisco:
"Lyrfigtu achieved remarkably deep and durable treatment responses in patients on the ReFocus trial. Beyond the confirmed ORR of 45.7% which speaks for itself, I’ve witnessed, first-hand, many of my own patients who were able to regain meaningful quality of life and precious time with their loved ones, owing to the robust tumor shrinkage they achieved on this treatment."
Alison Schram, M.D., gynecologic medical oncologist at Memorial Sloan Kettering Cancer Center:
"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. 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."
Elevar continues to evaluate Lyrfigtu for other indications in ongoing clinical development programs, including studies in other FGFR2-altered solid tumors. Any future indications will be subject to regulatory review and approval.
Important Safety Information for LYRFIGTU (lirafugratinib)
Warnings and Precautions:
Ocular Toxicity
LYRFIGTU can cause retinal pigment epithelial detachment (RPED), which may cause symptoms such as blurred vision.
Among 385 patients who received LYRFIGTU, RPED occurred in 31% of patients, including Grade 3 events in 1.8%. The median time to first onset was 57 days. RPED led to dose interruption in 15% of patients and dose reduction in 10%.
Perform a comprehensive ophthalmological examination, including OCT of the macula, prior to initiation of therapy, every 2 months for the first 13 months, and every 4 months thereafter. For onset of visual symptoms, obtain ophthalmologic evaluation urgently, with follow-up every 3 weeks until resolution or discontinuation of LYRFIGTU. Withhold, reduce the dose, or discontinue LYRFIGTU based on severity.
Among 385 patients who received LYRFIGTU, blurred vision occurred in 18% of patients with Grade 3 events in 1.3% of patients.
Dry eye occurred in 38% of patients. Treat patients with ocular demulcents as needed.
Among 385 patients who received LYRFIGTU, corneal toxicity/keratitis occurred in 11% of patients. Treat patients with ocular demulcents as needed.
Hyperphosphatemia and Soft Tissue Mineralization
LYRFIGTU can cause hyperphosphatemia leading to soft tissue mineralization, calcinosis, nonuremic calciphylaxis, and vascular calcification.
Hyperphosphatemia occurred in 21% of patients. The median time to onset was 15 days. Hyperphosphatemia led to dose interruption in one (0.3%) patient and no permanent discontinuation. Monitor serum phosphate throughout treatment and manage as clinically appropriate.
Embryo-Fetal Toxicity
Based on its mechanism of action and findings from animal studies, LYRFIGTU can cause fetal harm or loss of pregnancy when administered to a pregnant woman.
Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for 6 months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment and for 3 months after the last dose.
Contraindications:
None.
Adverse reactions:
Serious adverse reactions occurred in 32% of patients. Serious adverse reactions reported in ≥2% of patients were infection (6%), pneumonia (3.4%), fatigue (2.6%), and hemorrhage (2.6%). A fatal adverse reaction of hemorrhage occurred in one patient.
The most common adverse reactions (≥20%) were nail toxicity, palmar-plantar erythrodysesthesia syndrome, stomatitis, alopecia, dry eye, dry mouth, fatigue, dysgeusia, retinal pigment epithelial detachment, constipation, dry skin, infection, rash, abdominal pain, hemorrhage, blurred vision, diarrhea, musculoskeletal pain, nausea, and decreased appetite.
The most common laboratory abnormalities (≥20%) were increased phosphate, increased alanine aminotransferase, increased creatinine, decreased hemoglobin, decreased sodium, decreased lymphocytes, increased blood bilirubin, increased aspartate aminotransferase, decreased platelets, increased glucose, decreased leukocytes, increased alkaline phosphatase, decreased albumin, decreased phosphate, decreased neutrophils, decreased bicarbonate.
Reporting Suspected Adverse Events: To report SUSPECTED ADVERSE REACTIONS, contact Elevar Therapeutics at 1-866-4ELEVAR or contact the FDA at 1-800-FDA-1088, or visit www.fda.gov/medwatch.
Please see full Prescribing Information, including Patient Information, for Lyrfigtu.
For more information about Elevar, visit ElevarTX.com.
About LYRFIGTU (Lirafugratinib)
Lyrfigtu (lirafugratinib, aka RLY-4008) is a potent, selective and oral small molecule inhibitor of FGFR2, a receptor tyrosine kinase that is frequently altered in certain cancers. FGFR2 is one of four members of the FGFR family, a set of closely related proteins with highly similar protein sequences and properties. Lyrfigtu is currently being evaluated in a clinical trial to enroll additional patients with previously treated, advanced or metastatic solid tumors other than CCA harboring FGFR2 fusion or rearrangement, who have not been treated with prior FGFR inhibitors. Elevar has an exclusive license to lirafugratinib from Relay Therapeutics, Inc. for commercialization worldwide.
(Press release, Elevar Therapeutics, SEP 23, 2026, View Source [SID1234671040])