EU/3/18/1994 - orphan designation for treatment of biliary tract cancer
Ivosidenib
Orphan
Human
Overview
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.
Ivosidenib for treatment of biliary tract cancer has been authorised in the EU as Tibsovo since 4 May 2023.
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.
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 is based 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).
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).
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.
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.
- 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
- Active substance
- Ivosidenib
- Medicine name
- Tibsovo
- Intended use
- Treatment of biliary tract cancer
- Orphan designation status
- Positive
- EU designation number
- EU/3/18/1994
- Date of designation
- Sponsor
Les Laboratoires Servier
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Tibsovo at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the Tibsovo : Orphan Maintenance Assessment Report (initial authorisation).
Update history
| Date | Update |
|---|---|
| June 2021 | The sponsorship was transferred to Les Laboratoires Servier, France. |
| December 2020 | The sponsorship was transferred to Agios Netherlands B.V., Netherlands. |
| August 2018 | The sponsorship was transferred to Quality Regulatory Clinical Ireland Limited, Ireland. |
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: