Elevar Therapeutics Submits New Drug Application To FDA For Lirafugratinib As Second-Line Treatment For FGFR2-Positive Cholangiocarcinoma Following Strong ReFocus Trial Results

Elevar Therapeutics, Inc., a majority-owned subsidiary of HLB Co., Ltd. and a fully integrated biopharmaceutical company committed to improving treatment experiences and outcomes for patients with limited therapeutic options, announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration for its investigational therapy, lirafugratinib. The NDA seeks approval for lirafugratinib as a second-line treatment for patients with cholangiocarcinoma (CCA) who have FGFR2 fusions or rearrangements. Cholangiocarcinoma, also known as bile duct cancer, is a rare disease affecting approximately 8,000 people in the United States each year, according to the American Cancer Society.

Dong-Gun Kim, Elevar’s Chief Executive Officer, said that the NDA submission reflects the company’s ongoing mission to deliver transformative medicines to cancer patients, including those with rare diseases and advanced-stage conditions where treatment options are limited. He added that the company looks forward to working with the FDA as it reviews the application and is preparing for a potential commercial launch. Kim also expressed gratitude to the patients who participated in clinical trials and to everyone involved in bringing lirafugratinib to this stage.

The NDA is supported by clinical data from the Phase 1/2 ReFocus trial, which evaluated lirafugratinib in patients with advanced or metastatic CCA and other solid tumors with FGFR2 alterations. The trial demonstrated clinically meaningful anti-tumor activity, as measured by objective response rate, duration of response, and progression-free survival, alongside a manageable safety profile. The study results were recently presented at the American Society of Clinical Oncology’s Gastrointestinal Cancers Symposium.

According to the independent review committee, lirafugratinib achieved an objective response rate of 46.5%, with a median duration of response of 11.8 months, of which 76.2% of responses lasted more than six months. Median progression-free survival was 11.3 months, with a 12-month rate of 49.2%, and median overall survival was 22.8 months, with a 12-month rate of 74.6%. The disease control rate was 96.5%. The most common Grade 3 or higher treatment-related adverse events were palmar-plantar erythrodysesthesia (32.8%) and stomatitis (12.1%). These adverse events led to dose reductions in 75.9% of patients, dose interruptions in 82.8%, and treatment discontinuation in 4.3%.

Elevar is also exploring lirafugratinib for patients with other FGFR2-altered solid tumors. The company acquired worldwide rights to develop and commercialize the therapy in December 2024. Earlier that year, discussions with the FDA guided the regulatory strategy, recommending that the company submit an NDA first for FGFR2-driven CCA, followed by a supplemental NDA for other FGFR2-altered solid tumors.