The FDA has granted an Orphan Drug Designation to the novel small molecule chemotherapeutic uttroside-B for the treatment of patients with hepatocellular carcinoma.
The FDA has granted an Orphan Drug Designation to the novel small molecule chemotherapeutic uttroside-B for the treatment of patients with hepatocellular carcinoma (HCC), announced Q BioMed, Inc, in a press release.1
A promising level of potency, which was 10 times that of standard of care sorafenib (Nexavar), was observed with uttroside-B in HCC cells evaluated in preclinical studies.
The ODD granted to uttroside-B may allow it to have market exclusivity for 7 years which would also contribute toward marketing approval of the agent as a treatment of HCC. Uttroside-B is currently being tested in preclinical settings to work toward an FDA Investigational New Drug application to be submitted later this year.
"Orphan Drug Designation gives our uttroside-B program a substantial boost, and we expect it will significantly accelerate development and reduce costs. We are pleased that the FDA recognizes the urgent need for effective treatments for HCC and that the agency sees the potential of uttroside-B to address this difficult to treat cancer," said Denis Corin, CEO, Q BioMed, Inc, in a statement.
Uttroside-B has been shown to induce phosphorylation in the MAPK and mTOR pathways and eliminate constitutive activation of JNK. The agent is a natural compound, fractionated saponin derived from Solanum nigrum Linn leaves, and steroid glycoside in addition to a small molecule.2 Preclinical research has also demonstrated that the agent increases the cytotoxicity in multiple liver cancer cell types.
As uttroside-B moves along in the FDA pipeline, it could serve in the unmet need for frontline therapies for the treatment of HCC.
1. Q BioMed’s uttroside-b receives U.S. FDA orphan drug designation in the treatment of liver cancer. New release. January 28, 2021. Accessed January 28, 2021. https://bit.ly/2NBiSrw
2. Nath LR, Gorantla JN, Thulasidasan AKT, et al. Evaluation of uttroside B, a saponin from Solanum nigrum Linn, as a promising chemotherapeutic agent against hepatocellular carcinoma. Sci Rep. 2016;6:36318. doi:10.1038/srep36318