Experimental Treatment for Cholangiocarcinoma Earns Orphan Drug Designation

According to a story from BioPortfolio, Imbrium Therapeutics and Mundipharma EDO GmbH recently announced that the US Food and Drug Administration (FDA) has awarded Orphan Drug designation to their experimental drug etoposide toniribate. This therapy is in development as a treatment for relapsed/refractory cholangiocarcinoma, a rare cancer that affects the bile ducts.

About Cholangiocarcinoma

Cholangiocarcinoma is a form of cancer that affects the bile ducts. More specifically, the epithelial cells of the bile duct are the ones affected by this disease. The cause is not directly known; many patients who develop the disease also have no known risk factors, but there are some factors that are known to increase risk, such as primary sclerosing cholangitis, chronic liver diseases such as hepatitis, certain genetic disorders such as Caroli’s syndrome, infection with Helicobacter bacteria, and certain liver flukes (a type of parasitic animal that infiltrates the bile ducts and feeds on bile). Symptoms include fever, weight loss, jaundice, itching, and abdominal pain. Treatment may include surgery, chemotherapy, radiation, or liver transplant. Transplant is usually only useful if the disease is detected early; surgery is the best chance for curing cholangiocarcinoma. This disease is rapidly lethal if surgical removal is not possible. To learn more about cholangiocarcinoma, click here.

About Orphan Drug Designation

Orphan Drug designation is reserved for therapies that are intended to treat rare diseases. This is defined as any disease that affects less than 200,000 people in the US. In order to qualify for the designation, a drug must show possible advantages in effectiveness or safety over currently available therapies. Alternatively, it must fulfill a medical need that is not being met by current treatments. Orphan Drug designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a period of market exclusivity to last seven years if the drug is approved.

About Etoposide Toniribate

The designation comes after the presentation of encouraging data from a Phase II clinical trial in which etoposide toniribate showed significant potential in improving outcomes for cholangiocarcinoma patients. One year survival was 44 percent when using the therapy and just 11 percent with the best available supportive care. The experimental drug also earned orphan designation in the EU in June 2018.

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