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, today announced 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.

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. Lyrfigtudemonstrated 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.