Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in September 2012 on request of the sponsor.
On 9 February 2012, orphan designation (EU/3/12/958) was granted by the European Commission to Endocyte Europe B.V., the Netherlands, for N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-L-alpha-aspartyl-L-cysteine and folic acid for the diagnosis of positive folate receptor status in 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.
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.
Ovarian cancer is a life-threatening disease that is associated with poor long-term survival.
At the time of designation, the number of patients eligible for diagnosis of positive folate-receptor status in ovarian cancer was estimated to be not more than 1.3 people in 10,000 per year in the European Union (EU)*. This is equivalent to a total of 65,819 people per year, which was considered to be below the ceiling for orphan designation. 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).
At the time of designation, there were no satisfactory methods of diagnosing positive folate-receptor status of ovarian cancer in the EU. Existing methods were limited to detecting the presence of ovarian cancer cells. They included vaginal examination to check for any visible abnormalities of the womb or ovaries. In addition, blood tests and ultrasound were used to help identify masses in the abdomen. If a mass was discovered, the patient underwent surgery to determine the nature of the mass.
The medicine is made of two components:
Once injected into a vein, the medicine is expected to enter ovarian cancer cells by attaching to the folate receptor. The radioactive technetium is expected to emit radiation which can be detected by an imaging method. The medicine therefore identifies those patients who have folate-receptor-positive ovarian cancers and are most likely to respond to treatment targeting the folate receptor.
The effects of this medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with this medicine in patients with ovarian cancer were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for the diagnosis of 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.
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.
Endocyte Europe B.V.
Prins Bernhardplein 200
1097 JB Amsterdam
The Netherlands
Telephone: +31 20 521 4777
Telefax: +31 20 521 4821
E-mail: info@endocyte.com
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: