EU/3/04/239 - orphan designation for treatment of pancreatic cancer

Deuterium oxide

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in May 2009 on request of the sponsor.

On 20 October 2004, orphan designation (EU/3/04/239) was granted by the European Commission to BDD Berolina Drug Development GmbH, Germany, for deuterium oxide for the treatment of pancreatic cancer.

What is 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 functions in the body. It makes a juice that helps to digest (break down) food. It also produces hormones, such as insulin, that help to control blood sugar levels. About 95% of pancreatic cancers come from the cells that make the juice to digest. These cancers of the pancreas are called adenocarcinomas. Pancreatic cancer is life-threatening.

What is the estimated number of patients affected by the condition?

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 46,000 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 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What treatments are available?

The choice of the treatment of pancreatic cancer depends on several factors, including the stage of the disease. Treatments 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). There are anti-cancer drugs that have been authorised for treatment of pancreatic cancer.
Deuterium oxide might be of potential significant benefit for the treatment of pancreatic cancer because it may act in a different way than other available medicines. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Deuterium oxide is, in very small amounts, present in normal drinking water. In high amounts deuterium oxide might influence and block several processes in cancer cells necessary for tumour growth. Therefore it might prevent cancer cells from growing when given in higher concentrations.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of deuterium oxide in experimental models was ongoing but no clinical trials in patients were initiated.

The medicinal product was not marketed anywhere worldwide, at the time of submission. Orphan designation of deuterium oxide has not been granted in other countries.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 9 September 2004 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria: 

• the seriousness of the condition;
• the existence or not 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 still investigational products, which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.

EU/3/04/239: Public summary of positive opinion for orphan designation of deuterium oxide for the treatment of pancreatic cancer

Adopted Reference Number: EMEA/COMP/69735/2004 Rev.1

Key facts

• Active substance:
  Deuterium oxide
• Intended use:
  Treatment of pancreatic cancer
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/04/239
• Date of designation:
  20/10/2004
• Sponsor:

  BDD Berolina Drug Development GmbH; Bitterfelder Strasse 19; D-12681 Berlin; Germany; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]