First Orphan Drug Designation (ODD) in the field of microbiome-based treatment for metabolic diseases
SEOUL, South Korea, Dec. 8, 2022 /PRNewswire/ -- LB-P8, a novel drug being developed by LISCure Biosciences, Inc. (LISCure) as a treatment for Primary Sclerosing Cholangitis (PSC), has been designated as an orphan drug by the US FDA.
On December 1, 2022, the US FDA notified LISCure of the orphan drug designation of LB-P8, LISCure's microbiome novel drug, as treatment of PSC. LB-P8 is, to the best of company's knowledge, the first drug to be granted orphan drug designation in the field of microbiome-based treatment for metabolic diseases.
The US FDA's ODD program supports the development of treatments for rare diseases with high unmet medical needs and provides various incentives. Once designated as an orphan drug, the company qualifies for incentives such as tax credits for qualified clinical trials, exemption from user fees, and potential seven years of market exclusivity after approval. In addition, the likelihood of successfully developing the orphan drug is increased with the Agency's support when designing the drug development strategy.
PSC is a chronic cholestatic liver disease that is caused by bile duct inflammation and fibrosis of unknown cause. The inflammation can stop the flow of bile acids and lead to liver fibrosis or liver cancer. There are no treatments to date and liver transplantation is the only treatment option. LISCure, through research collaboration with Mayo Clinic, has demonstrated therapeutic efficacy in PSC in animal models.
Global Pharmaceutical companies have been accelerating the development of treatments for orphan diseases by acquiring companies with orphan disease pipelines. Pfizer, in August 2022, acquired Global Blood Therapeutics, and AstraZeneca acquired Alexion Pharmaceuticals. In addition, Sanofi, Takeda, Bristol Myers Squibb (BMS), Novartis, Merck, etc. are fortifying their orphan disease pipelines through acquiring companies specializing in developing orphan drugs.
"LB-P8, which is being developed in collaboration with the Mayo Clinic, was presented in an oral presentation and chosen in 'The Best of The Liver Meeting® 2022' in the recent AASLD (American Association for Study of Liver Diseases), and demonstrated efficacy in not only PSC but also Nonalcoholic Steatohepatitis (NASH). The company has recently successfully completed its global Phase 1 study, and with the US FDA's orphan drug designation, the US Phase 2 study will proceed as planned to meet the market's expectations" said Chin Hwa Sub, LISCure's Chief Executive Officer.