EU/3/19/2146 - orphan designation for treatment of biliary tract cancer
1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one (futibatinib)
OrphanHuman
Overview
This medicine is now known as futibatinib.
On 1 April 2019 orphan designation (EU/3/19/2146) was granted by the European Commission to Taiho Pharma Europe Limited, United Kingdom, for 1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one (also known as TAS-120) for the treatment of biliary tract cancer.
Please note that this product (marketed as Lytgobi) was withdrawn from the Union Register of orphan medicinal products in May 2023 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.
Biliary tract cancer affects 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 to help digest fats. The cancer is characterised by 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).
*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 518,400,000 (Eurostat 2019).
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.
This medicine is a tyrosine kinase inhibitor, which blocks the activity of enzymes known as tyrosine kinases, particularly those that are part of receptors (targets) 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 in 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. Orphan designation of the medicine had been granted in the United States for the treatment of cholangiocarcinoma (bile duct cancer).
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 21 February 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.
Key facts
- Active substance
- 1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one (futibatinib)
- Intended use
- Treatment of biliary tract cancer
- Orphan designation status
- Positive
- EU designation number
- EU/3/19/2146
- Date of designation
- Sponsor
Taiho Pharma Netherlands B.V.
Barbara Strozzilaan 201
1083 HN Amsterdam
Noord-Holland
Netherlands
Email: sruini@taiho.eu
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of the product at the time of marketing authorisation. The sponsor formally requested the withdrawal of the orphan designation prior to the Committee’s final opinion. More information is available in the Lytgobi : Orphan designation withdrawal assessment report (initial-authorisation).
Update history
| Date | Update |
|---|---|
| April 2022 | The sponsor’s address was updated. |
| June 2021 | The sponsorship was transferred to Taiho Pharma Netherlands B.V. Netherlands. |
| September 2020 | The sponsorship was transferred to Taiho Oncology Europe B.V., Netherlands. |
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: