EU/3/15/1477

On 24 April 2015, orphan designation (EU/3/15/1477) was granted by the European Commission to Oncolytics Biotech (UK) Limited, United Kingdom, for human reovirus type-3 Dearing strain for the treatment of pancreatic cancer.

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 juice 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 locallyor 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 fewer than 103,000 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).

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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 human reovirus type 3 Dearing strain might be of significant benefit for patients with pancreatic cancer becauseearly studies indicate that it may improve survival when used in combination with currently available medicines. 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?

This medicine is made up of an ‘oncolytic’ virus called reovirus that it is able to target, infect and destroy cancer cells but does not infect normal cells. When inside a cancer cell, the virus is expected to take over the cell’s replication apparatus and use it to make more copies of itself. This is expected to kill the cell, leaving the virus to spread to neighbouring cancer cells.

What is the stage of development of this medicine?

The effects of human reovirus type-3 Dearing strain 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 or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 March 2015 recommending the granting of this designation.

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.