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 patients who have limited or inadequate therapeutic options, 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.

CCA, also known as bile duct cancer, is rare, with about 8,000 people in the U.S. diagnosed each year, according to the American Cancer Society.

“This NDA reaffirms Elevar’s mission of bringing life-changing medicines to cancer patients worldwide, including for rare indications and for advanced stages where treatment options are limited,” said Dong-Gun Kim, the company’s chief executive officer. “We are excited to work with the FDA as it reviews the submission while simultaneously preparing for a potential commercial launch. We could not be more appreciative of the patients who participated in lirafugratinib-focused clinical trials and everyone who brought us to this crucial point in its development.”

The NDA is supported by positive clinical data from the phase 1/2 ReFocus trial (NCT04526106), in which lirafugratinib demonstrated clinically meaningful anti-tumor activity, measured by objective response rate (ORR), duration of response (DOR), and progression-free survival (PFS), as well as manageable and tolerable safety in patients with advanced/metastatic CCA and other solid tumors with fibroblast growth factor receptor 2 (FGFR2) alterations.

The abstract “Efficacy and safety of lirafugratinib in FGFRi-naïve cholangiocarcinoma (CCA) patients harboring FGFR2 fusions/rearrangements (FGFR2 f/r)” was presented earlier this month at the American Society of Clinical Oncology’s Gastrointestinal Cancers Symposium.

Independent review committee-assessed ORR was 46.5%, and median DoR was 11.8 months (mos) (95% CI, 7.5-13.0), where 76.2% of responses lasted >6 mos. Median PFS was 11.3 mos (95% CI, 9.2, 14.8), with 12-month rate of 49.2%. Median OS was 22.8 mos (95% CI, 18.1-27.2), with 12-month rate of 74.6%. The disease control rate was 96.5%. Most common Grade ≥3 treatment-related adverse events (TRAEs) were palmar-plantar erythrodysesthesia (32.8%) and stomatitis (12.1%). TRAEs led to dose reduction, dose interruption, and treatment discontinuation were observed in 75.9%, 82.8%, and 4.3%, respectively.

Elevar is also developing lirafugratinib for patients with other FGFR2-altered other solid tumors. The company acquired worldwide rights to develop and commercialize lirafugratinib in December 2024. Earlier that year, Relay Therapeutics met with the FDA to discuss data from the ReFocus trial and potential regulatory pathways. The FDA recommended the company first file an NDA for FGFR2-driven CCA, followed by a supplemental NDA for FGFR2-altered other solid tumors.