On 9 February 2012, orphan designation (EU/3/12/959) was granted by the European Commission to Endocyte Europe B.V., the Netherlands, for vincaleukoblastin-23-oic acid, O4-deacetyl-, 2-[(2-mercaptoethoxy)carbonyl]hydrazide, disulfide with N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-L-γ-glutamyl-L-α-aspartyl-L-arginyl-L-α-aspartyl-L-α-aspartyl-L-cysteine 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 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 not more than 3.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of not more than 177,000 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 506,300,000 (Eurostat 2011).
- 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 this medicine might be of significant benefit for patients with ovarian cancer because early studies show that it might improve the outcome of women with this condition when used in combination with other treatments and in particular in a subset of the disease called ‘folate receptor positive’ ovarian cancer. 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 made up of two components, desacetylvinblastine monohydrazide (a cell-killing molecule) and folic acid (a tumour-targeting molecule). Most ovarian cancers are ‘folate receptor-positive’. This means that the surface of the cancer cell contains high amounts of a receptor which folate (a vitamin required for cell division) attaches to. This receptor can be targeted for treatment. The medicine is expected to attach to the folate receptor of the cancer cell via its folic acid component and enter the cell. The medicine is then expected to release the desacetylvinblastine monohydrazide component inside the cell to disrupt small structures called ‘microtubules’ which are responsible for cell structure and cell division, thereby stopping the cell from dividing and causing it to die.
- What is the stage of development of this medicine?
The effects of this medicinal product have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the medicinal product in patients with ovarian cancer were ongoing.
At the time of submission, the medicinal product 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 7 October 2011 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.
|Active substance||Vincaleukoblastin-23-oic acid, O4-deacetyl-, 2-[(2-mercaptoethoxy)carbonyl]hydrazide, disulfide with N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-L-γ-glutamyl-L-α-aspartyl-L-arginyl-L-α-aspartyl-L-α-aspartyl-L-cysteine|
|Disease/condition||Treatment of ovarian cancer|
|Date of decision||09/02/2012|
|Orphan decision number||EU/3/12/959|
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:
Endocyte Europe B.V.
Prins Bernhardplein 200
1097 JB Amsterdam
Telephone: +31 20 521 4777
Telefax: +31 20 521 4821
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