HLB's Cholangiocarcinoma Drug "Lificty" Receives FDA Approval... U.S. Launch in Q4

Direct NDA Submission for Novel Cancer Drug Wins FDA Approval
Approved as a Second-Line Therapy for FGFR2-Altered Cholangiocarcinoma
U.S. Launch Set for Q4; European Regulatory Pursuit Underway

HLB's targeted therapy for cholangiocarcinoma, "Lificty (ingredient: lirafugratinib)," has crossed the threshold of the U.S. Food and Drug Administration (FDA).

HLB's U.S. subsidiary, Elevar Therapeutics, announced on September 24 (Korean time) that it received FDA approval for Lificty on the 23rd (local time). The approval is for adult patients with unresectable locally advanced or metastatic cholangiocarcinoma who have previously undergone treatment, in which fibroblast growth factor receptor 2 (FGFR2) gene fusion or rearrangement has been detected.

According to HLB, this is the first case in which a domestic pharmaceutical or biotech company directly submitted a new drug application (NDA) for an anticancer drug holding global rights to the FDA and received its approval.

This approval demonstrates HLB's capability to secure rights to a novel drug candidate and see it through late-stage development, regulatory approval, and commercialization in the United States. In December 2024, Elevar acquired global development and commercialization rights for lirafugratinib from Relay Therapeutics in the United States.

Lificty is an oral targeted therapy that selectively inhibits FGFR2, a key player in cancer initiation and progression. It is designed to irreversibly bind to FGFR2, ensuring sustained inhibition, while minimizing unnecessary inhibition of other FGFR subtypes (FGFR1, 3, and 4).

The FDA approval was based on the global phase 1·2 "ReFocus" trial, which enrolled 116 patients with cholangiocarcinoma who had not previously received FGFR inhibitor treatment following prior chemotherapy or chemoimmunotherapy. According to the FDA, the objective response rate (ORR) was 46%, and the median duration of response (mDOR) was 11.8 months.

Elevar plans to launch Lificty in the U.S. market within the fourth quarter of this year. On September 14, the company also submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for approval as a treatment for cholangiocarcinoma, marking the start of the European approval process.

Dongkeun Kim, CEO of Elevar, stated, "The FDA approval of Lificty is significant in providing an important second-line treatment option for patients with cholangiocarcinoma and their families. We will do our best to ensure that Lificty is supplied as quickly as possible to healthcare professionals and patients, and to improve the treatment environment for patients whose treatment options have been limited."

This content was produced with the assistance of AI translation services.