HLB's Cholangiocarcinoma Drug 'Lyfigtu' Approved by FDA, Set for U.S. Market Launch

HLB has received approval from the U.S. Food and Drug Administration (FDA) for its targeted cancer drug, Lyfigtu (ingredient name: liraglutide), for the treatment of cholangiocarcinoma (CCA). This marks the first instance of a domestic pharmaceutical and biotech company directly submitting a New Drug Application (NDA) to the FDA and obtaining approval for a global cancer drug.
HLB's U.S. subsidiary, Elevate Therapeutics, announced on September 23 (local time) that the FDA has approved Lyfigtu as a second-line treatment for patients with cholangiocarcinoma harboring FGFR2 fusions or rearrangements. This approval establishes a foundation for HLB to pursue not only the development and approval of cancer drugs but also their commercialization in the U.S. market.
The key data supporting the approval comes from the global multicenter, single-arm Phase 1/2 trial, ReFocus (NCT04526106). An independent review committee (BICR) analysis of 116 cholangiocarcinoma patients with confirmed FGFR2 fusions or rearrangements showed an objective response rate (ORR) of 46% (95% confidence interval: 36-55%).
The median duration of response (mDOR) for responding patients was 11.8 months, while the median progression-free survival (mPFS) was 11.3 months (95% CI: 9.2-14.8). The 12-month progression-free survival rate was 49.2%, and the median overall survival (mOS) was reported at 22.8 months. The safety profile was deemed predictable and manageable through dose adjustments.
Lyfigtu is an irreversible targeted cancer drug with high selectivity for FGFR2. It is designed to bind to FGFR2, providing sustained inhibition while minimizing unnecessary suppression of FGFR1, 3, and 4. This characteristic helps reduce the incidence of side effects commonly seen with non-selective FGFR inhibitors, such as hyperphosphatemia and diarrhea.
The drug holds significant clinical importance as it offers a new targeted therapy option for patients with FGFR2 fusion or rearrangement cholangiocarcinoma, who have limited treatment choices. Dr. Allison Shram, a gynecologic oncology specialist at Memorial Sloan Kettering Cancer Center (MSKCC), stated, "Lyfigtu demonstrates sustained treatment responses and manageable safety, presenting new treatment possibilities for patients with FGFR2 fusion-positive cholangiocarcinoma."
Elevate Therapeutics plans to launch Lyfigtu in the U.S. market in the fourth quarter of this year. Cholangiocarcinoma is a rare cancer that occurs in the bile ducts, which transport bile produced by the liver. According to the American Cancer Society, approximately 8,000 new cases are diagnosed in the U.S. each year.
Approval procedures are also underway in Europe. HLB submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in September, prior to the FDA approval. The European approval is also based on the ReFocus clinical results and, like in the U.S., targets FGFR2 fusion or rearrangement cholangiocarcinoma as an indication for second-line treatment.
HLB previously received three Complete Response Letters (CRLs) related to manufacturing facility issues during the FDA approval process for its liver cancer drug, riboceranib. The approval of Lyfigtu is seen as a demonstration of HLB's capabilities in global cancer drug development, overcoming the challenges faced in the past.
Kim Dong-geun, CEO of Elevate Therapeutics, remarked, "The FDA approval of Lyfigtu signifies an important second-line treatment option for cholangiocarcinoma patients and their families. We will do our utmost to ensure that Lyfigtu is supplied to healthcare providers and patients as quickly as possible, improving the treatment landscape for patients with limited options."