On 24 January 2013, orphan designation (EU/3/12/1092) was granted by the European Commission to Ganymed Pharmaceuticals AG, Germany, for chimeric monoclonal antibody against claudin 6 for the treatment of ovarian cancer.
- What is ovarian cancer?
Ovarian cancer is cancer of the ovaries (two organs in the female reproductive system that produce eggs). Most ovarian cancers occur in women over the age of 50 years. Due to the absence of symptoms in the early stages of the disease, the majority of patients are diagnosed when the cancer has spread to other parts of the body.
Ovarian cancer is a long-term debilitating and life-threatening disease that is associated with poor long-term survival.
- What is the estimated number of patients affected by the condition?
At the time of designation, ovarian cancer affected approximately 3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 153,000 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 the treatment of ovarian cancer. The choice of treatment depended mainly on how advanced the disease was. Treatments included surgery and chemotherapy (medicines to treat cancer).
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with ovarian cancer because studies in experimental models suggest it might improve the treatment of patients with this condition. 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?
This medicine 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 on the surface of cancer cells. This medicine has been designed to target an antigen called claudin 6, which is a protein found on the surface of many ovarian cancer cells (particularly in advanced disease), but not on the surface of healthy cells. By attaching to claudin 6 on the cancer cells, the monoclonal antibody is expected to cause the cells to die, slowing down the growth of the cancer.
- What is the stage of development of this medicine?
The effects of chimeric monoclonal antibody against claudin 6 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 ovarian cancer had been started.
At the time of submission, chimeric monoclonal antibody against claudin 6 was not authorised anywhere in the EU for ovarian cancer or designated as an orphan medicinal product elsewhere 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.
|Name||Language||First published||Last updated|
|EU/3/12/1092: Public summary of opinion on orphan designation: Chimeric monoclonal antibody against claudin 6 for the treatment of ovarian cancer||(English only)||01/03/2013|
|Active substance||Chimeric monoclonal antibody against claudin 6|
|Disease/condition||Treatment of ovarian cancer|
|Date of decision||24/01/2013|
|Orphan decision number||EU/3/12/1092|
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
Tel. +49 61 31 1440 100
Fax +49 6131 1440 114
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.