EU/3/20/2329

Cholangiocarcinoma is a type of cancer that begins in the bile ducts. These are small tubes through which bile, which is produced by the liver and stored in the gall bladder, enters the intestine where it helps to digest fats. Symptoms usually only occur once the cancer is in its advanced stages, when it may have spread to other parts of the body.

Cholangiocarcinoma is a life-threatening condition with a high mortality rate.

At the time of designation, cholangiocarcinoma affected approximately 1.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 88,000 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).

*For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

At the time of the application for orphan designation, no satisfactory methods were authorised in the EU for the treatment of cholangiocarcinoma. Surgery was used to remove the cancer, and chemotherapy (medicines to treat cancer) and radiotherapy (treatment with radiation) were used to try to prevent the cancer from spreading to other parts of the body.

Some patients with cholangiocarcinoma have mutations (changes) in the genes responsible for the production of proteins called fibroblast growth factor receptors (FGFRs). These mutations are thought to play a role in the development of cancer, including cholangiocarcinoma.

This medicine blocks the action of FGFR 1, 2, and 3. By blocking these FGFRs, the medicine is expected to prevent cancer cells from multiplying and the development of blood vessels that help tumours grow. This is expected to slow down the worsening of the disease.

The effects of infigratinib have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with infigratinib in patients with cholangiocarcinoma were ongoing.

At the time of submission, infigratinib was not authorised anywhere in the EU for the treatment of cholangiocarcinoma. Orphan designation of infigratinib had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 16 July 2020, 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.