EU/3/17/1911 - orphan designation for treatment of pancreatic cancer
sodium 2-hydroxylinoleate
OrphanHuman
Overview
On 23 August 2017, orphan designation (EU/3/17/1911) was granted by the European Commission to Ability Pharmaceuticals SL, Spain, for sodium 2-hydroxylinoleate (also known as ABTL0812) for the treatment of pancreatic cancer.
Pancreatic cancer is cancer of the pancreas, a small 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. Due to the absence of symptoms in the early stages of pancreatic cancer, most patients are diagnosed when the cancer has spread nearby or to other parts of the body.
Pancreatic cancer is a very severe and life-threatening disease that is associated with shortened life expectancy.
At the time of designation, pancreatic cancer affected less than 2 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 103,000 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 515,700,000 (Eurostat 2017).
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 and chemotherapy (medicines to treat cancer).
The sponsor has provided sufficient information to show that sodium 2-hydroxylinoleate might be of significant benefit for patients with pancreatic cancer because laboratory studies show that it may reduce tumour growth when used in combination with currently authorised cancer medicines (gemcitabine and paclitaxel). This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is expected to work by reducing the activity of several proteins in the 'Akt/mTOR signalling pathway'. This is a mechanism within cells which is important in regulating their growth and survival. In many cancers, including pancreatic cancer, this pathway is overactive, allowing the cancer cells to grow uncontrollably. By reducing the activity of this pathway, the medicine is expected to slow down the progression of the cancer.
The effects of sodium 2-hydroxylinoleate have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with sodium 2-hydroxylinoleate in patients with pancreatic cancer had been started.
At the time of submission, sodium 2-hydroxylinoleate was not authorised anywhere in the EU for pancreatic cancer. Orphan designation of had been granted in the United States for pancreatic cancer.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 July 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
- sodium 2-hydroxylinoleate
- Medicine name
- -
- Intended use
- Treatment of pancreatic cancer
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1911
- Date of designation
- Sponsor
Ability Pharmaceuticals SL
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:
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.
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: