EU/3/20/2259 - orphan designation for treatment of pancreatic cancer
2-hydroxy-N,N,N-trimethylethan-1-aminium (Z)-4-(5-((3-benzyl-4-oxo-2-thioxothiazolidin-5-ylidene)methyl)furan-2-yl)benzoate
OrphanHuman
Overview
On 24 March 2020, orphan designation EU/3/20/2259 was granted by the European Commission to MWB Consulting S.A.R.L., France, for 2-hydroxy-N,N,N-trimethylethan-1-aminium (Z)-4-(5-((3-benzyl-4-oxo-2-thioxothiazolidin-5-ylidene)methyl)furan-2-yl)benzoate (also known as GB1275) for the treatment of pancreatic cancer.
Pancreatic cancer is cancer of the pancreas, an organ that lies behind the stomach. The pancreas has two functions: to produce a fluid that helps with the digestion of food, and to produce hormones such as insulin. Because symptoms in the early stages of pancreatic cancer are vague (such as itching, fever and feeling sick), the majority of patients are diagnosed when the cancer has spread nearby or to other parts of the body.
Pancreatic cancer is a severe and life-threatening disease that can shorten life.
At the time of designation, pancreatic cancer affected approximately 2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 104,000 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).
*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 designation, several medicines were authorised in the EU for treating pancreatic cancer. The choice of treatment depended on several factors, including how far the disease had advanced. Treatments included surgery, radiotherapy (treatment with radiation) and chemotherapy (medicines to treat cancer).
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with pancreatic cancer. Laboratory studies have suggested that adding the medicine to two other medicines used to treat pancreatic cancer (gemcitabine and paclitaxel) could prolong lives compared with just gemcitabine and paclitaxel.
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine attaches to a protein called CD11b which is found on the surface of certain cells of the immune system (the body’s natural defences) called myeloid cells. In pancreatic cancer, myeloid cells do not work properly and prevent the cancer from being attacked by the immune system, and can promote cancer growth. By attaching to CD11b, the medicine stops the myeloid cells from moving to the site where the cancer is located and weakens their ability to promote cancer growth, allowing the immune system to attack the cancer. These effects are expected to slow down growth of the 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 pancreatic cancer were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of pancreatic cancer. Orphan designation had been granted in the USA for this condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 20 February 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.
Key facts
- Active substance
- 2-hydroxy-N,N,N-trimethylethan-1-aminium (Z)-4-(5-((3-benzyl-4-oxo-2-thioxothiazolidin-5-ylidene)methyl)furan-2-yl)benzoate
- Intended use
- Treatment of pancreatic cancer
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/20/2259
- Date of designation
- Sponsor
MWB Consulting S.A.R.L.
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Update history
| Date | Update |
|---|---|
| February 2023 | Please note that this product was withdrawn from the Union Register of orphan medicinal products on request of the Sponsor. |
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: