EU/3/14/1265 - orphan designation for treatment of ovarian cancer
Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor for the treatment of ovarian cancer
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2023 on request of the Sponsor.
On 29 April 2014, orphan designation (EU/3/14/1265) was granted by the European Commission to Oncos Therapeutics Oy, Finland, for genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor for the treatment of ovarian cancer.
In November 2015, Oncos Therapeutics Oy changed name to Targovax Oy.
The sponsor’s address was updated in March 2021.
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 less than 3 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 153,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 the medicine might be of significant benefit for patients with ovarian cancer because early studies in patients suggest that it might improve the condition of patients with ovarian cancer resistant to treatment with existing therapies. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine contains a virus into which a gene has been inserted so that it can produce a protein called 'granulocyte-macrophage colony-stimulating factor' (GM-CSF). It has also had one of its own genes inactivated so it can only reproduce itself within cancer cells. When the medicine is injected into a patient with ovarian cancer, the virus is expected to selectively attach to the cancer cells, enter them and reproduce inside them, eventually killing them.
In addition, it is expected to produce the GM-CSF protein in cancer cells, which stimulates the immune system (the body's natural defences) to attack the cancerous cells.
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 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 March 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
- Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor for the treatment of ovarian cancer
- Intended use
- Treatment of ovarian cancer
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/14/1265
- Date of designation
- Sponsor
Targovax Oy
Lars Sonckin Kaari 14
Espoo
Helsinki-Uusimaa
02600
Finland
Tel. +358 10 279 4000
E-mail: contact@targovax.com
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:
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.
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: