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Orphan designation

On 21 March 2018, orphan designation (EU/3/18/1994) was granted by the European Commission to QRC Consultants Ltd, United Kingdom, for ivosidenib for the treatment of biliary tract cancer.

What is biliary tract cancer?

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 which is 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.

What is the estimated number of patients affected by the condition?

At the time of designation, biliary tract cancer affected approximately 1.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 67,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

* Disclaimer: 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 (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 517,400,000 (Eurostat 2018).

What treatments are available?

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 to treat cancer).

How is this medicine expected to work?

Some patients with biliary tract cancer have a fault in a gene called IDH1, which causes production of an abnormal IDH1 protein. The abnormal protein makes a substance, 2-hydroxyglutarate, that causes cells to become cancerous. Ivosidenib is expected to block the activity of the abnormal IDH1 protein and so reduces production of 2-hydroxyglutarate, thereby preventing formation of cancer cells.

What is the stage of development of this medicine?

The effects of ivosidenib have been evaluated in experimental models.

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

At the time of submission, ivosidenib was not authorised anywhere in the EU for biliary tract cancer or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 15 February 2018 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria
  • 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.

Key facts

Product details for <p>Ivosidenib</p>
Active substanceIvosidenib
Medicine Name
Disease/conditionTreatment of biliary tract cancer
Date of decision21/03/2018
Orphan decision numberEU/3/18/1994

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details

QRC Consultants Ltd
Silvaco Technology Centre
Compass Point
St Ives
Cambridgeshire PE27 5JL
United Kingdom
Tel. +44 (0)1480 309 349~

Patients’ organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases which includes a directory of patients’ organisations registered in Europe.
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.