EU/3/17/1916 - orphan designation for treatment of biliary tract cancer
5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate]
OrphanHuman
Overview
On 16 October 2017, orphan designation (EU/3/17/1916) was granted by the European Commission to Voisin Consulting S.A.R.L., France, for 5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate] (also known as Debio 1347) for the treatment of biliary tract cancer.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in December 2020 on request of the Sponsor.
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 1.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 77,000 people1, 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).
1Disclaimer: 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 515,700,000 (Eurostat 2017).
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).
This medicine is a tyrosine kinase inhibitor, which blocks the activity of enzymes known as tyrosine kinases, particularly those that are part of receptors called fibroblast growth factor receptors (FGFRs). FGFRs are found on the surface of cells and are involved in the growth and spread of cancer cells. By blocking the tyrosine kinases in the FGFR receptors, this medicine is expected to prevent or slow down the growth of biliary tract 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 the medicine including patients with biliary tract cancer were ongoing.
At the time of submission, the medicine 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 7 September 2017 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
- 5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate]
- Intended use
- Treatment of biliary tract cancer
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/17/1916
- Date of designation
- Sponsor
Voisin Consulting S.A.R.L.
64, Avenue Pierre Grenier
92100 Boulogne Billancourt
France
Tel. +33 1 41 31 83 00
E-mail: orphan@voisinconsulting.com
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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: