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Orphan designation

On 5 August 2013, orphan designation (EU/3/13/1177) was granted by the European Commission to Ganymed Pharmaceuticals AG, Germany, for chimaeric monoclonal antibody against claudin-18 splice variant 2 for the treatment of pancreatic cancer.

In May 2017 sponsor, Ganymed Pharmaceuticals AG changed name to Ganymed Pharmaceuticals AG GmbH.

What is pancreatic cancer?

Pancreatic cancer is cancer of the pancreas, a small organ that lies behind the stomach. The pancreas has two functions: to produce a juice 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 locally or to other parts of the body.

Pancreatic cancer is a very severe and life-threatening disease that is associated with shortened life expectancy.

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

At the time of designation, pancreatic cancer affected approximately 1.1 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 27), Norway, Iceland and Liechtenstein. This represents a population of 509,000,000 (Eurostat 2013).

What treatments are available?

At the time of designation, several medicines were authorised in the EU for treating pancreatic cancer. The choice of treatment depended on several factors, including how far the disease had advanced. Treatments included surgery, radiotherapy (treatment with radiation) and chemotherapy (medicines to treat cancer).

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with pancreatic cancer because early studies in experimental models suggest that it may improve the survival of patients, when it is used in combination with a chemotherapy medicine called gemcitabine. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Claudin-18 splice variant 2 is a protein found in the cells of the ducts of the pancreas, where it helps the cells to stick to each other. In patients with pancreatic cancer, this protein is produced in large amounts and is thought to be involved in the survival and spread of the cancer cells.

The medicine is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a part of the claudin-18 splice variant 2 protein in the cancer cells. By attaching to this protein, this medicine is expected to stimulate the immune system (the body’s natural defences) to kill the cancer cells, slowing down the spread of the cancer.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

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 EU for the treatment of gastric cancer.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 July 2013 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:
  • 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.

Key facts

Product details for <p>Chimaeric monoclonal antibody against claudin-18 splice variant 2</p>
Active substanceChimaeric monoclonal antibody against claudin-18 splice variant 2
Medicine Name
Disease/conditionTreatment of pancreatic cancer
Date of decision05/08/2013
Orphan decision numberEU/3/13/1177

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details

Ganymed Pharmaceuticals AG GmbH
D-55131 Mainz
Tel. + 49 6131 1440100
Fax + 49 6131 1440114

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.