EU/3/06/419

The pancreas is a small organ that is located in the abdomen, behind the stomach. The pancreas has two main functions in the body; it produces a mix of enzymes (proteins that trigger chemical reactions) that help digest (break down) food and it produces hormones, such as insulin, which help to control blood sugar levels. Cancer of the pancreas is often detected at a late stage of the disease, because it rarely causes any symptoms in its earlier stages. Pancreatic cancer is life-threatening.

At the time of designation pancreatic cancer affected approximately 1.2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 55,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and 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 27), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

The choice of the treatment of pancreatic cancer depends on several factors, including the stage of the disease. Treatment may include surgery, radiation therapy (using high-dose x-rays or other high-energy rays to kill cancer cells), and chemotherapy (using drugs to kill cancer cells). Several medicinal products have been authorised for the treatment of pancreatic cancer in the Community.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that paclitaxel (liposomal) might be of potential significant benefit for the treatment of pancreatic cancer, because it may improve the long-term outcome of the patients. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

When cells divide and grow, there are structures (tubules) inside the cells that need to assemble and disassemble in a very orderly way. Paclitaxel interferes with the assembly of these tubules and subsequently with the growth of cells. Liposomal paclitaxel is delivered to the cancer cells in the tumour in little lipid particles (liposomes) that bind specifically to the cells that line blood vessels. According to the sponsor, paclitaxel (liposomal) will, by inhibiting the growth of newly formed blood vessels, contribute to the destruction of the tumour.

The effects of paclitaxel (liposomal) 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.

Paclitaxel (liposomal) was not authorised anywhere worldwide for the treatments of pancreatic cancer nor designated as orphan medicinal product elsewhere for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 4 October 2006 a positive opinion recommending the grant of the above-mentioned 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.