The U.S. Food and Drug Administration has approved Lyrfigtu (lirafugratinib) as a treatment option for patients with cholangiocarcinoma, or bile duct cancer, whose tumors carry an FGFR2 fusion or other rearrangement, Elevar Therapeutics has announced.

 

The approval gives patients with this rare and difficult-to-treat cancer a new second-line treatment option. Cholangiocarcinoma affects about 8,000 people in the United States 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.”

 

Elevar, a majority-owned subsidiary of HLB Co., Ltd., said Lyrfigtu is expected to become available to U.S. patients in the fourth quarter of 2026.

 

The approval was supported by data from the Phase 1/2 ReFocus trial (NCT04526106). In patients with the approved indication, Lyrfigtu produced a confirmed objective response rate of 46%, while the median duration of response was 11.8 months.

 

Median progression-free survival was 11.3 months, with a 12-month progression-free survival rate of 49.2%, according to Elevar.

 

The company said the treatment's safety profile was predictable and manageable through dose adjustments.

 

The FDA granted Lyrfigtu priority review in March 2026. The designation is reserved for applications for drugs that, if approved, could provide “significant improvements in the safety or effectiveness of the treatment” of a serious condition.

 

Lipika Goyal, lead author of the ReFocus study and director of gastrointestinal oncology at the Stanford Cancer Center, highlighted Lyrfigtu's mechanism of action and its potential to address resistance to earlier FGFR inhibitors.

 

“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, professor of medicine in the division of Hematology & Oncology at the Helen Diller Family Comprehensive Cancer Center at the University of California, San Francisco, pointed to the depth and durability of responses seen in the trial.

 

“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, gynecologic medical oncologist at Memorial Sloan Kettering Cancer Center, said the findings also underscore the importance of molecular testing.

 

“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.”

 

The FDA-approved treatment carries important safety warnings, including ocular toxicity, hyperphosphatemia and potential embryo-fetal toxicity.

 

Among 385 patients who received Lyrfigtu, retinal pigment epithelial detachment occurred in 31%, including Grade 3 events in 1.8%. Blurred vision occurred in 18%, while dry eye occurred in 38% and corneal toxicity or keratitis in 11%.

 

Hyperphosphatemia occurred in 21% of patients and can lead to soft-tissue mineralization and other complications, according to the prescribing information.

 

Serious adverse reactions occurred in 32% of patients. Reported serious reactions occurring in at least 2% of patients included infection, pneumonia, fatigue and hemorrhage. One patient died from hemorrhage.