EU/3/12/1090

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Orphan designation

On 24 January 2013, orphan designation (EU/3/12/1090) was granted by the European Commission to Astellas Pharma Europe B.V., the Netherlands, for recombinant human monoclonal antibody of the IgG1 kappa class against prostate stem-cell antigen for the treatment of pancreatic cancer.

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 1.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 71,000 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 suggest that it may improve the survival of patients with adenocarcinoma (the most common form of pancreatic cancer), when it is used in combination with another 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?

The medicine ‘recombinant human monoclonal antibody of the IgG1 kappa class’ is a monoclonal antibody, a type of protein that has been designed to recognise and attach to a specific structure (called an antigen) that is found in the body. It is expected to attach to an antigen called ‘prostate stem=cell antigen’, which is found on the surfaces of normal cells as well as in high amounts on the surface of prostate, bladder and pancreatic cancer cells. The exact function of prostate stem-cell antigen is not fully understood, although it is thought to play a role in regulating cell growth through regulating the transmission of chemical signals between cells. When the medicine attaches to prostate stem-cell antigen, it is expected to alter the antigen’s normal activity, including blocking the transmission of chemical signals, thereby reducing the growth and spread of the pancreatic cancer cells.

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, clinical trials with the medicine in patients with pancreatic cancer were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for pancreatic cancer. Orphan designation of this medicine had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 December 2012 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>Recombinant human monoclonal antibody of the IgG1 kappa class against prostate stem-cell antigen</p>
Active substanceRecombinant human monoclonal antibody of the IgG1 kappa class against prostate stem-cell antigen
Medicine Name
Disease/conditionTreatment of pancreatic cancer
Date of decision24/01/2013
OutcomePositive
Orphan decision numberEU/3/12/1090

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:

Astellas Pharma Europe B.V
Sylviusweg 62
2333 BE Leiden
The Netherlands
Tel. +31 715 455 174
Fax +31 715 455 840
E-mail: tanja.megens-delange@astellas.com

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.