EU/3/14/1385: Orphan designation 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, the majority of patients are diagnosed when the cancer has spread locally 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 in 10,000 people in the European Union (EU). This was equivalent to a total of around 102,000 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 511,100,000 (Eurostat 2014).

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 early results show improved responses in patients when the medicine was added to currently authorised treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

In the early stages of cancer, the body's immune system (its natural defences) can often combat the growth and spread of tumour cells, but over time the immune system may become less effective in controlling the cancer, allowing it to grow. This medicine contains a species of bacteria called Mycobacterium obuense (NCTC 13365) that have been killed by heating so they can no longer grow or cause infection. When the medicine is injected, the body's immune system is activated to become more effective, because the bacteria are considered a possible new threat. This activation of the immune system is expected to help the body also combat the cancer more effectively.

The effects of heat-killed Mycobacterium obuense (whole cell) 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, heat-killed Mycobacterium obuense (whole cell) 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 13 November 2014 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.