EU/3/18/2066

Biliary tract cancer is cancer of the bile ducts and gallbladder. These are parts of the digestive system that transport and store bile, a fluid produced by the liver and released into the intestines after a meal to help digest fats. The cancer is characterised by various features such as abnormal liver function tests, pain in the belly, yellowish discoloration of the skin and weight loss.

Biliary tract cancer is a long-term debilitating and life-threatening disease due to liver failure and problems caused when the cancer blocks the bile ducts.

At the time of designation, biliary tract cancer affected approximately 1.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 78,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of biliary tract cancer. Some patients with early disease could undergo surgery to remove the cancer. Other treatments included chemotherapy medicines (medicines to treat cancer), although these were not authorised for biliary tract cancer.

Pemigatinib belongs to a group of medicines called protein kinase inhibitors. It works by blocking enzymes known as protein kinases, particularly those that are part of receptors (targets) called fibroblast growth factor receptors (FGFRs). FGFRs are found on the surface of cancer cells and are involved in the growth and spread of the cancer cells. By blocking the tyrosine kinases in FGFRs, pemigatinib is expected to reduce the growth and spread of the cancer.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with pemigatinib including patients with biliary tract cancer were ongoing.

At the time of submission, pemigatinib was not authorised anywhere in the EU for biliary tract cancer. Orphan designation had been granted in the United States for the treatment of cholangiocarcinoma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 July 2019 recommending the granting of this designation.

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.