EU/3/18/2085: 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. 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 debilitating and life-threatening disease that is associated with shortened life expectancy.

At the time of designation, pancreatic cancer affected approximately 2.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 124,000 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).

At the time of designation, several medicines were authorised in the EU for treating pancreatic cancer. 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 laboratory studies showed that it slows down the growth of tumours. When given in combination with other cancer medicines it led to better outcomes than when these other medicines were used alone.

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

The medicine is made of cells that have been modified to produce TRAIL, a protein in the body that triggers cancer cell death. When the medicine is injected into the patient’s cancer, the cells in the medicine will produce the TRAIL protein, which will attach to receptors (targets) on the surface of cancer cells and trigger their death.

At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.

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 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 11 October 2018 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.