NewG Lab's Rare Drugs for Liver and Bile Duct Cancer Approved by US FDA: "Marketable After Phase 2 Clinical Trials"

[Asia Economy Reporter Hyungsoo Park] Newgelab Pharma has acquired the U.S. Food and Drug Administration (FDA) orphan drug designation rights for liver cancer and cholangiocarcinoma, which is expected to accelerate clinical trials.

Newgelab announced on the 29th that its U.S. subsidiary, Newgelab Pharma, obtained the FDA orphan drug designation rights for liver cancer and cholangiocarcinoma from GoDiscovery.

GoDiscovery, founded by Dr. Ko Younghee in 2013, received orphan drug designation for 3-Bromopyruvic acid (3BP)-based substances targeting liver cancer and cholangiocarcinoma, and is recognized for significantly improving the success rate and efficiency of orphan drug clinical trials.

While liver cancer is prevalent in Asia, the number of patients in the U.S. is less than 200,000. According to pharmaceutical and biotech analysis data, the clinical success rate of orphan drugs is approximately 2.6 times higher than the overall average. The FDA orphan drug designation system supports the smooth development and approval of treatments for rare, intractable, or life-threatening diseases, providing various benefits to the developing pharmaceutical companies.

Once designated as an FDA orphan drug, companies are guaranteed seven years of market exclusivity, along with tax reductions and exemption from application fees. This allows for shortened development time, significantly reducing clinical costs and the number of clinical trial patients. Clinical trials can be conducted with far fewer patients than general protocols, and Phase 3 trials can be conducted at about 25% of the usual cost.

A company representative stated, "By acquiring orphan drug designation rights, we can minimize development costs and establish multi-faceted development strategies, including additional clinical trials for other cancer types." They also added, "In February, we separately applied for orphan drug designation for melanoma, a type of skin cancer."

They continued, "For cancers designated as orphan drugs, we are working towards accelerated approval, a conditional approval system," emphasizing, "If conditional approval is granted, sales can begin after completing Phase 2 clinical trials, significantly shortening the new drug approval timeline."

In September last year, Newgelab Pharma signed a preclinical trial contract, including an ODD program, with the global contract research organization (CRO) Covance, and is currently conducting toxicity tests for the anticancer agent KAT.

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