EU/3/15/1604

On 11 January 2016, orphan designation (EU/3/15/1604) was granted by the European Commission to Medpace Germany GmbH, Germany, for two allogeneic irradiated pancreatic tumour cell lines (also known as GVAX) for the treatment of pancreatic cancer.

The sponsorship was transferred to Aduro Biotech Holdings, Europe B.V., The Netherlands, in April 2016.

What is 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, 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.

What is the estimated number of patients affected by the condition?

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).

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*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 512,900,000 (Eurostat 2015).

What treatments are available?

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 because early results suggest that it may improve survival of patients whose disease had not responded to previous therapy, when given as part of a combination treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

The medicine works by stimulating the patient’s immune system, the body’s natural defences, so that it targets and destroys the cancer cells. It is made of two types of pancreatic cancer cells that have been modified to produce granulocyte-macrophage colony stimulating factor (GM-CSF) and have been treated with radiation to prevent them from growing. GM-CSF stimulates the immune system to recognise as ‘foreign’ certain proteins, such as mesothelin, which are found at high levels on the surface of pancreatic cancer cells. This is expected to stimulate the immune system to destroy pancreatic cancer cells.

The medicine is given as part of a combination treatment including another medicine (called ‘live attenuated Listeria monocytogenes delta actA/delta inlB strain expressing human mesothelin’ or CRS-207) which helps the immune system to recognise mesothelin as foreign and so stimulate the body to attack the cancer.

What is the stage of development of this medicine?

The effects of this 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 of the medicine had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 December 2015 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

Opinions on orphan medicinal product designations are based on the following three criteria:

• 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.

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.