Cholangiocarcinoma Drug Receives Orphan Drug Designation from FDA

Etoposide toniribate is intended for the treatment of relapsed/refractory biliary tract cancer.

Approximately 8000 patients in the United States and nearly 10,600 in Europe are diagnosed each year with rare biliary tract tumors, which qualifies this cancer as a rare disease, according to the U.S. Food and Drug Administration (FDA) (defined as less than 200,000 people in the United States). The only cure for biliary tract cancer today involves radical surgery, but, for most patients, surgery is not an option. When first-line chemotherapy fails for these patients, the standard of care is generally palliative. The five-year survival rate is low at just 15%.

A candidate being developed by Mundipharma EDO GmbH and Imbrium Therapeutics for the treatment of relapsed/refractory biliary tract cancer, or cholangiocarcinoma, was recently designated an orphan drug by FDA, giving patients some hope for another treatment option.

In phase II clinical trials, etoposide toniribate has shown positive results, which led the FDA to grant it an orphan drug designation (ODD). Etoposide toniribate already received an ODD from the European Medicines Agency in 2014. In both countries, ODD brings assistance with protocol development, lower fees and a 10-year marketing exclusivity in the specific disease once approval has been received.


The two companies are currently planning a global phase III study with sites in the EU, United States, Australia and other countries.

Cynthia A. Challener, Ph.D.

Dr. Challener is an established industry editor and technical writing expert in the areas of chemistry and pharmaceuticals. She writes for various corporations and associations, as well as marketing agencies and research organizations, including That’s Nice and Nice Insight.