EU/3/06/384: Orphan designation for the treatment of pancreatic cancer

Pancreatic cancer is a cancer of the pancreas, a small organ that lies behind the stomach. The pancreas has two functions: producing a juice that helps with the digestion of food, and producing hormones such as insulin. About 95% of pancreatic cancers affect the cells that make the pancreatic juice. These are called adenocarcinomas.
Pancreatic cancer is a very severe and life-threatening disease.

At the time of designation, pancreatic cancer affected approximately1.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 56,000 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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 468,900,000 (Eurostat 2006).

At the time of submission of the application for orphan drug designation, several medicines were authorised for the treatment of pancreatic cancer in the European Union. The choice of treatment for pancreatic cancer depends on several factors, including the stage of the disease. Treatments may include surgery, radiotherapy (treatment with radiation), and chemotherapy (medicines used to treat cancer). Satisfactory argumentation has been submitted by the sponsor to justify the assumption that human telomerase reverse transcriptase peptide (611-626) might be of potential significant benefit for the treatment of pancreatic cancer, mainly because it has a new mechanism of action and may be used in combination with other treatments. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

Human telomerase reverse transcriptase peptide (611-626) is a part of the enzyme (a protein that triggers chemical reactions) telomerase reverse transcriptase, which is often present in pancreatic cancer tumour cells. Human telomerase reverse transcriptase is needed for tumour cells to be able to divide many times (proliferate) and subsequently for the tumour to grow. The medicinal product is designed to activate the body's natural defence system, the immune system against the cells containing the human telomerase reverse transcriptase. According to the sponsor the product will trigger the immune system against the pancreatic cancer tumour cells, thus destroying the tumour cells with the body's own defence system.

The effects of human telomerase reverse transcriptase peptide (611-626) were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with pancreatic cancer were ongoing.

Human telomerase reverse transcriptase peptide (611-626) was not authorised anywhere worldwide for the treatment of pancreatic cancer nor designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 15 June 2006 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 Community) 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.