EU/3/20/2259: Orphan designation for the treatment of 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 with the digestion of food, and to produce hormones such as insulin. Because symptoms in the early stages of pancreatic cancer are vague (such as itching, fever and feeling sick), the majority of patients are diagnosed when the cancer has spread nearby or to other parts of the body.

Pancreatic cancer is a severe and life-threatening disease that can shorten life.

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 104,000 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).

*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

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. Laboratory studies have suggested that adding the medicine to two other medicines used to treat pancreatic cancer (gemcitabine and paclitaxel) could prolong lives compared with just gemcitabine and paclitaxel.

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

The medicine attaches to a protein called CD11b which is found on the surface of certain cells of the immune system (the body’s natural defences) called myeloid cells. In pancreatic cancer, myeloid cells do not work properly and prevent the cancer from being attacked by the immune system, and can promote cancer growth. By attaching to CD11b, the medicine stops the myeloid cells from moving to the site where the cancer is located and weakens their ability to promote cancer growth, allowing the immune system to attack the cancer. These effects are expected to slow down growth of the cancer.

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 the treatment of pancreatic cancer. Orphan designation had been granted in the USA for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 20 February 2020, 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.