HLB Applies for FDA Pre-NDA Meeting for Biliary Tract Cancer Drug, Initiates New Drug Approval Process

On July 30, HLB announced that its U.S. subsidiary, Elevar Therapeutics, has applied for a pre-NDA (New Drug Application) meeting with the U.S. Food and Drug Administration (FDA) in preparation for the new drug application of the biliary tract cancer treatment 'Rilapugratinib (RLY-4008)'.

The pre-NDA meeting is a key procedure to increase the likelihood of new drug approval. It is held before submitting the NDA and serves to coordinate with the FDA in advance on clinical results, the structure of submission documents, and approval strategies.

Rilapugratinib demonstrated excellent therapeutic efficacy, high target selectivity, and outstanding tolerability in early clinical trials for patients with FGFR2 fusion-positive biliary tract cancer. Recognized for its potential to bring significant therapeutic innovation to the field of biliary tract cancer, where treatment options are limited, it was designated as a 'Breakthrough Therapy' by the U.S. FDA in 2023. Upon NDA submission, it is highly likely to be designated for 'Priority Review', which shortens the review period from the standard 10 months to 6 months.

In the global phase 2 clinical trial conducted on patients with FGFR2 fusion-positive biliary tract cancer, the drug showed superior efficacy compared to existing treatments in key endpoints such as objective response rate (ORR) and duration of response (DOR), as assessed by the Independent Review Committee (IRC). Cases of complete response (CR) were also reported.

In terms of safety, the incidence rates of major side effects such as diarrhea and hyperphosphatemia were significantly lower than those of existing approved therapies, further demonstrating its differentiation and competitiveness as a best-in-class drug.

The company expects the meeting to be held around the end of September.

Currently, Elevar is preparing to submit materials focusing on the key issues to be discussed with the FDA, while thoroughly reviewing core data, including clinical data, to maximize the chances of NDA approval. The company is also making thorough preparations across all NDA submission requirements, such as GMP qualification review for both the active pharmaceutical ingredient and the finished product of Rilapugratinib.

HLB stated that for the combination therapy of Rivoceranib and Camrelizumab, which is being developed as a liver cancer treatment, its partner company Jiangsu Hengrui Pharmaceuticals is diligently addressing CMC-related supplementary matters in consultation with the FDA. For the biliary tract cancer treatment Rilapugratinib as well, the company aims to sequentially secure new drug approvals for both indications through thorough preparation.

Han Yonghae, Chief Technology Officer (CTO) of HLB Group, said, "The key data based on IRC standards obtained from the global phase 2 clinical trial will be presented at 'ASCO GI 2026' next year," and added, "The NDA submission for the second-line treatment of biliary tract cancer is also proceeding smoothly as scheduled." He further stated, "The global expansion trial targeting FGFR2 mutations regardless of cancer type, with the aim of obtaining 'tumor-agnostic therapy' approval, is also progressing smoothly."