EU/3/15/1516

On 28 July 2015, orphan designation (EU/3/15/1516) was granted by the European Commission to Lokon Pharma AB, Sweden, for modified adenovirus serotype 5/35 containing a CMV promoter-driven transgene cassette with the human transgenes for a membrane-bound CD40 ligand and full length 4-1BBL for the treatment of pancreatic cancer.

What is 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 to digest 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 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 the medicine might be of significant benefit for patients with pancreatic cancer because experimental studies suggest that its use in combination with available treatments may lead to improved effects on cancer. 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 is made up of an ‘oncolytic’ virus, a virus that has been modified so that it can target, infect and destroy cancer cells, but not 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. In addition, the modifications to the virus prepare the cell for self-destruction and cause infected cells to produce substances that stimulate the immune system (the body’s natural defences), which will also help to destroy the cancer cells.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with pancreatic cancer had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for pancreatic cancer. Orphan designation of the medicine has been granted in the United States for the treatment of pancreatic cancer.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 June 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.