In Europe, Orphan Drug designation is granted to therapies intended to treat, prevent, or diagnose a rare, life-threatening, or debilitating disease. For the purpose of this status, rare conditions are those affecting no more than 5 out of every 10,000 individuals. Drug developers who receive this status on one of their products also receive incentives. These include fee reductions, protocol assistance, and 10 years of market exclusivity upon approval. According to Pharmaceutical Business Review, the European Medicines Agency (EMA) recently granted Orphan Drug designation to CPI-613 (devimistat). This therapy was designed to treat patients with advanced biliary tract cancer that cannot be surgically removed.
What is Biliary Tract Cancer?
Also known as cholangiocarcinoma, biliary tract cancer is a rare cancer which affects the bile ducts, or the slender tubes which carry bile (a digestive fluid) from the liver to the small intestine. There are multiple subtypes depending on the cancer’s location, such as intrahepatic, hilar, and distal. Typically, cholangiocarcinoma affects those older than 50, though it can occur in people of all ages. Additional risk factors include biliary cysts, diabetes, a history of bile duct inflammation, and obesity. Biliary tract cancer is often difficult-to-treat, especially as it is diagnosed in later stages. When symptoms appear, they can include:
- Jaundice (yellowing of the skin, eyes, and mucus membranes)
- Nausea and vomiting
- Appetite loss
- Unintended weight loss
- Dark urine and pale stools
- Itchiness
- Fever and chills
- Pain on the right side of the abdomen
About CPI-613 (Devimistat)
Devimistat, developed by Cornerstone Pharmaceuticals (“Cornerstone”), is described by the National Cancer Institute as:
a synthetic alpha-lipoic lipoic acid analogue with potential chemopreventive and antineoplastic activities. Although the exact mechanism of action is unknown, devimistat has been shown to inhibit metabolic and regulatory processes required for cell growth in solid tumors.
In short, Cornerstone believes that devimistat is able to target and inhibit enzymes which help tumor cells grow. Outside of cholangiocarcinoma, devimistat has also received Orphan Drug designation in Europe for pancreatic cancer, acute myeloid leukemia, and Burkitt’s lymphoma. Cornerstone remains committed to pursuing additional testing and research around devimistat, and hopes to get this treatment into patients’ hands in the future.
