EU/3/02/129: Orphan designation for the treatment of pancreatic cancer

Cancer that begins in the pancreas is called pancreatic cancer. The pancreas is a small organ that lies behind the stomach and in front of the spine. The pancreas has two main jobs in the body. It makes juices that help digest (break down) food. It makes hormones, such as insulin, that help to control blood sugar levels. About 95% of pancreatic cancers begin in the cells that make digestive juices. These cancers of the pancreas are called adenocarcinomas. Pancreatic cancer is life-threatening.

At the time of designation, pancreatic cancer affected approximately 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 40,000 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: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 377,000,000 (Eurostat 2001) and may differ from the true number of patients affected by the condition.

The choice of the treatment of pancreatic cancer depends on several factors, including the stage of the disease. Treatments may include surgery, radiation therapy, and drug therapy. Medicinal products have been authorised for use in pancreatic cancer in the European Union. Due to its new mode of action, G17(9) gastrin-diphtheria toxoid conjugate may be of potential significant benefit for patients affected by the condition. The benefit of G17(9) gastrin-diphtheria toxoid conjugate will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

Gastrin is a hormone that is normally produced by the body. Gastrin stimulates the stomach to produce gastric juice. Gastrin also makes certain digestive system cancers grow faster. G17(9) gastrin-diphtheria toxoid conjugate is designed to stimulate the body to make antibodies against gastrin. Antibodies are proteins which specifically recognise and block certain substances. Antibodies against gastrin are expected to specifically link to this hormone, thus blocking its activity. This would consequently slow down the growth of pancreatic cancer.

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

At the time of submission, the medicinal product was not marketed anywhere worldwide for pancreatic cancer. Orphan designation of G17(9) gastrin-diphtheria toxoid conjugate was granted in the United States and in Australia in pancreatic cancer.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 13 December 2002 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.