Microbiome-Derived Therapy Gets Orphan Drug Status for Hepatocellular Carcinoma

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The Food and Drug Administration (FDA) has granted Orphan Drug designation to SFA001 (SFA Therapeutics) for the treatment of hepatocellular carcinoma (HCC).

SFA001 is an investigational microbiome-derived drug that is developed from natural substances using the Company’s proprietary discovery process to match metabolites and adjuvants to treat chronic inflammatory diseases. 

The designation is based on preclinical studies in transgenic HBX animal models in which SFA001 significantly reduced the number of HCC tumors as well as the size of the tumors. Additionally, the mechanism of action of SFA001 was found to be non-chemotoxic in 2 different animal models of HCC.

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“By granting an Orphan Drug designation to SFA001, FDA has given a special status to treatment of the most prevalent form of liver cancer,” stated Ira Spector, PhD, SFA Therapeutics’ CEO. “These advantages undoubtedly will speed the drug’s availability for a disease that kills hundreds of thousands of patients annually world-wide.”

For more information visit sfatherapeutics.com.