FDA Grants Chemotherapeutic Uttroside-B Orphan Drug Designation for HCC

January 27, 2021 - The FDA has granted an orphan drug designation to the small molecule chemotherapeutic uttroside-B for use as a treatment in patients with hepatocellular carcinoma.

The FDA has granted an orphan drug designation to the small molecule chemotherapeutic uttroside-B for use as a treatment in patients with hepatocellular carcinoma (HCC), according to an announcement from Q BioMed Inc, the drug developer.1

In preclinical research, uttroside-B was found to be 10 times more potent than sorafenib (Nexavar) in HCC cells.

With the new designation, uttroside-B can potentially benefit from a 7-year market after marketing approval, grant funding for trials that support a marketing approval, protocol assistance, and tax credits.

The biomedical acceleration and development company also stated that the product is currently undergoing preclinical testing to support the submission of an investigational new drug application to the FDA.

“Orphan drug designation gives our Uttroside-B program a substantial boost, and we expect it will significantly accelerate development and reduce costs,” Denis Corin, CEO of Q Biomed Inc, stated in a press release. “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.”

Uttroside-B is a compound that was extracted from the leaves of Solanum nigrum Linn, a plant that serves as a strong resource for many anticancer molecules; as such, it is widely utilized in traditional medicine.2 The product consists of β-D-glucopyranosyl unit at C-26 of the furostanol and β-lycotetraosyl unit at C-3.

The chemotherapeutic has also been shown to elicit cytotoxicity in all liver cancer cell lines, regardless of hepatitis B status; at the same time, it has proven to be nontoxic to normal immortalized hepatocytes.

By downregulating the activation of 2 pathways, MAPK and mTOR, the chemotherapeutic also elicited apoptosis in HepG2 cells. In NOD-SCID mice, uttroside-B severely reduced tumor size in HepG2-xenograft model.

Additionally, the biological safety of the product was examined in Swiss albino mice through the use of both acute and chronic toxicity models. Because all the chemotherapy regimens that are available for patients with HCC are known to come up with substantial systemic adverse effects, the clinical benefits of these products are limited.

One complication linked with chemotherapy is that it results in the reduction of lymphocytes, neutrophils, and monocytes—all important hematologic parameters. Notably, uttroside-B did not showcase a significant difference in these parameters, thus confirming that the product does not create significant toxicity or immunosuppression in animals.


1. Q BioMed’s Uttroside-B receives US FDA orphan drug designation in the treatment of liver cancer. News release. Q BioMed Inc. January 27, 2021. Accessed January 27, 2021. http://prn.to/39oORUp.

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