EU/3/16/1804 - orphan designation for treatment of pancreatic cancer
pegylated recombinant human interleukin-10
OrphanHuman
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Overview
On 12 December 2016, orphan designation (EU/3/16/1804) was granted by the European Commission to Larode Ltd, United Kingdom, for pegylated recombinant human interleukin-10 (also known as AM0010) for the treatment of pancreatic cancer.
The sponsorship was transferred to Eli Lilly Nederland B.V., the Netherlands, in October 2018.
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, the majority of 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 approximately 2.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 123,000 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 513,700,000 (Eurostat 2016).
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 this medicine might be of significant benefit for patients with pancreatic cancer. Early data indicate that it can be used in combination treatments for advanced cancer that has worsened despite treatment with gemcitabine (a cancer medicine). In addition, the improvement seen so far with this medicine compares well with that seen with other treatments. These assumptions will need to be confirmed at the time of marketing authorisation in order to maintain the orphan status.
This medicine is a type of immunotherapy, which means that it acts on the body's immune system (the body's natural defences). It activates white blood cells known as CD8+ T cells. These cells can infiltrate pancreatic cancer tumours, where they are expected to kill cancer cells and improve survival.
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 pancreatic cancer. Orphan designation had been granted in the United States for the condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 4 November 2016 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
- pegylated recombinant human interleukin-10
- Intended use
- Treatment of pancreatic cancer
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1804
- Date of designation
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: