Summary: development and regulatory status as of September 2026

Lirafugratinib (RLY-4008), discovered by Relay Therapeutics and licensed worldwide to Elevar Therapeutics in December 2024, was approved by the FDA on 23 September 2026 as Lyrfigtu capsules for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 fusion or other rearrangement. The new drug application (NDA 220425) was received on 27 January 2026, was granted priority review, and used the Real-Time Oncology Review pilot program and the Assessment Aid. Lirafugratinib had received orphan drug designation for cholangiocarcinoma in January 2022 and breakthrough therapy designation in 2023. The application was not referred to an advisory committee. The approval letter states the application is approved for use as recommended in the agreed-upon labeling, and neither the letter nor the prescribing information contains the accelerated approval statement that appears in the labeling of pemigatinib, futibatinib, and infigratinib; the ReFocus cohort had been described in 2024 as designed to support accelerated approval. Approval rests on 116 FGFR inhibitor-naive participants in the single-arm phase 1/2 REFOCUS trial (NCT04526106), with an objective response rate of 46% (95% CI 36 to 55) and a median duration of response of 11.8 months. The FDA required a randomized trial comparing 70 mg once daily with a lower dosage and a P-gp and BCRP drug interaction trial, and accepted a commitment to validate a companion diagnostic. Elevar expects product availability in the fourth quarter of 2026. A marketing authorization application was submitted to the European Medicines Agency in September 2026, and a phase 2 tumor-agnostic study (ReFocus202, NCT07359820) began dosing in June 2026.

Regulatory submission: NDA submitted January 2026

“Please refer to your new drug application (NDA) received January 27, 2026, and your amendments, submitted under section 505(b) of the Federal Food, Drug, and Cosmetic Act” (opens the source at this quote in a new tab)

“today announced it submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational drug lirafugratinib as a second-line treatment option for cholangiocarcinoma (CCA) patients with FGFR2 fusion or rearrangement.” (opens the source at this quote in a new tab)

Launch: Anticipated in the fourth quarter of 2026

“Lyrfigtu is expected to be available to patients in the U.S. by Q4 2026.” (opens the source at this quote in a new tab)
“Our team is thrilled to bring this therapy to market and focused on getting Lyrfigtu to doctors and patients as quickly as possible” (opens the source at this quote in a new tab)

FDA approval on 23 September 2026

“On September 23, 2026, the Food and Drug Administration approved lirafugratinib (Lyrfigtu, Elevar Therapeutics, Inc.), a kinase inhibitor, for the treatment of adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma harboring a fibroblast growth factor receptor 2 (FGFR2) gene fusion or other rearrangement.” (opens the source at this quote in a new tab)
“Efficacy was evaluated in REFOCUS (NCT04526106), a multicenter, open-label, single-arm trial in 116 patients with unresectable or metastatic cholangiocarcinoma who were naïve to FGFR inhibitor treatment and had received prior chemotherapy or chemoimmunotherapy.” (opens the source at this quote in a new tab)
“ORR was 46% (95% CI: 36, 55) and median DOR was 11.8 months (95% CI: 7.5, 13.0).” (opens the source at this quote in a new tab)

“We have completed our review of this application, as amended. It is approved, effective on the date of this letter, for use as recommended in the enclosed agreed-upon labeling.” (opens the source at this quote in a new tab)

Approval pathway: pivotal cohort designed for accelerated approval, approved without an accelerated approval statement in labeling

“The study includes a pivotal cohort in patients with FGFR2-fusion CCA that was designed to support accelerated approval and is fully enrolled.” (opens the source at this quote in a new tab)
“Earlier in 2024, Relay Therapeutics met with the U.S. Food and Drug Administration (FDA) to discuss data from the ReFocus trial and potential regulatory pathways. The FDA recommended that the company first file an NDA for FGFR2-driven CCA, followed by a supplemental NDA for FGFR2-altered other solid tumors with data from an expanded cohort of patients.” (opens the source at this quote in a new tab)

“LYRFIGTU is indicated for the treatment of adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma harboring a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement [see Dosage and Administration (2.1)].” (opens the prescribing information at this quote)

Expedited programs: priority review, breakthrough therapy, orphan drug designation, and Real-Time Oncology Review

“This review used the Real-Time Oncology Review (RTOR) pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.” (opens the source at this quote in a new tab)
“This application was granted priority review. Lirafugratinib received breakthrough therapy and orphan drug designations.” (opens the source at this quote in a new tab)

“Lirafugratinib received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in 2022 and Breakthrough Therapy Designation in 2023. Elevar submitted a New Drug Application for lirafugratinib to the FDA in January 2026, and the application was granted Priority Review.” (opens the source at this quote in a new tab)

“In January 2022, the FDA granted orphan drug designation to lirafugratinib for the treatment of cholangiocarcinoma.” (opens the source at this quote in a new tab)

“The FDA determined that the NDA is sufficiently complete to permit a substantive review and granted Priority Review” (opens the source at this quote in a new tab)

Advisory committee

“Your application for Lyrfigtu was not referred to an FDA advisory committee because outside expertise was not necessary; there were no controversial issues that would benefit from advisory committee discussion.” (opens the source at this quote in a new tab)

Clinical development: ReFocus dose escalation and recommended phase 2 dose

“The first part of the trial, or the dose escalation, is complete, and the second part of the trial, or the dose expansion, is ongoing at the 70mg once daily recommended Phase 2 dose.” (opens the source at this quote in a new tab)

Development beyond cholangiocarcinoma: ReFocus202 tumor-agnostic phase 2 study

“The trial, known as ReFocus202 (Protocol ID ELE-4008-202; NCT07359820), is an open-label, single-arm study evaluating the efficacy and safety among a broad scope of tumors containing an FGFR2 fusion or rearrangement. The primary endpoint is objective response rate.” (opens the source at this quote in a new tab)
“In the Phase 1/2 ReFocus study, data from 42 non-CCA solid tumor patients with FGFR2 fusion/rearrangement (13 tumor types) showed meaningful antitumor activity of lirafugratinib.” (opens the source at this quote in a new tab)

Regulatory submission outside the United States: European Medicines Agency

“today announced the submission of a marketing authorization application to the European Medicines Agency (EMA) for lirafugratinib as a second-line treatment for patients with advanced/metastatic cholangiocarcinoma (CCA) harboring FGFR2 fusions or rearrangements.” (opens the source at this quote in a new tab)

Patent term

“As of the date of this Annual Report on Form 10-K, we own granted patents in Eurasia, Europe, Taiwan, Japan, Hong Kong, and the United States covering the composition of matter of lirafugratinib.” (opens the source at this quote in a new tab)

Lirafugratinib (Lyrfigtu)

Phase of product development