HLB's 'Rivoceranib' Designated as Orphan Drug by US FDA for Liver Cancer Treatment

[Asia Economy Reporter Hyunseok Yoo] HL Biopharma announced on the 8th that its globally licensed targeted anticancer drug, rivoceranib, was designated as an orphan drug for liver cancer by the U.S. FDA on the 4th.

The FDA's orphan drug designation is a system that supports the development and approval of treatments for rare and intractable diseases. When designated as an orphan drug, new drug developers can benefit from various advantages such as shortened clinical trial approval and authorization periods, exemption from user fees for prescription drug approval applications, tax reductions, and seven years of market exclusivity after approval.

HL Biopharma is currently conducting a global Phase 3 clinical trial for first-line liver cancer using a combination therapy of rivoceranib and the immuno-oncology drug camrelizumab. Following camrelizumab's orphan drug designation for liver cancer in April this year, rivoceranib's orphan drug designation is expected to significantly shorten the approval period in the future.

The Phase 3 clinical trial for first-line liver cancer with rivoceranib is being conducted in Korea, the U.S., Europe, China, and Taiwan, comparing the drug against a control using overall survival (OS) and progression-free survival (PFS) as primary endpoints. A total of 510 patients have been enrolled. In Korea, the trial is being conducted at 10 sites including Seoul Asan Medical Center, Samsung Seoul Hospital, and Seoul National University Hospital.

Rivoceranib is an oral anticancer agent that inhibits vascular endothelial growth factor receptor (VEGFR)-2, suppressing angiogenesis, normalizing blood vessels, and activating immune cells. Due to this mechanism, it has good compatibility with other types of anticancer drugs such as cytotoxic agents and immuno-oncology drugs, with fewer side effects. Notably, in China, rivoceranib and camrelizumab were approved as second-line treatments for liver cancer in March and December 2020, respectively, and are currently on the market. Clinical results of rivoceranib have been published in the prestigious journal The Lancet, raising high expectations for the efficacy of the combination therapy of these two drugs.

Rivoceranib was designated as an orphan drug for gastric cancer by the FDA in June 2017 and for soft tissue sarcoma in February 2021.

In addition to the Phase 3 first-line liver cancer trial, HL Biopharma is preparing a new drug application (NDA) for rivoceranib as a treatment for advanced gastric cancer and is simultaneously conducting Phase 2 trials for first-line soft tissue sarcoma, Phase 2 trials for first-line gastric cancer, and Phase 1b/2 trials for third-line colorectal cancer. Patient recruitment for soft tissue sarcoma has been completed in the U.S. and Korea.