EU/3/14/1303 - orphan designation for treatment of ovarian cancer
cediranib
OrphanHuman
Overview
On 29 July 2014, orphan designation (EU/3/14/1303) was granted by the European Commission to AstraZeneca AB, Sweden, for cediranib for the treatment of ovarian cancer.
Ovarian cancer is cancer of the ovaries, the 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.
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 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).
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 cediranib might be of significant benefit for patients with ovarian cancer because studies in patients show that it improves the survival of patients who were previously treated with standard platinum-based chemotherapy. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Cediranib is expected to work by blocking the activity of 'vascular endothelial growth factor' (VEGF) receptors. These receptors are found in high amounts on the surface of vascular cells and are involved in the development of new blood vessels that supply nutrients to cancer cells. By blocking VEGF receptors, this medicine is expected to reduce blood supply to the cancer cells, thus slowing down the growth and spread of the cancer.
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 cediranib in patients with ovarian cancer were ongoing.
At the time of submission, the medicine 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 12 June 2014 recommending the granting of this designation.
- 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
- Active substance
- cediranib
- Intended use
- Treatment of ovarian cancer
- Orphan designation status
- Positive
- EU designation number
- EU/3/14/1303
- Date of designation
- Sponsor
AstraZeneca AB
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: