EU/3/12/1002 - orphan designation for treatment of glioma
Adenovirus-associated vector containing human Fas-c gene (ofranergene obadenovec)
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in September 2022 on request of the Sponsor.
This medicine is now known as ofranergene obadenovec.
On 6 June 2012, orphan designation (EU/3/12/1002) was granted by the European Commission to Gregory Fryer Associates Ltd, United Kingdom, for adenovirus-associated vector containing human Fas-c gene for the treatment of glioma.
Glioma is a type of brain tumour that affects the 'glial' cells (the cells that surround and support the nerve cells). Patients with glioma can have severe symptoms, but the types of symptoms experienced depend on where the tumour develops in the brain. Symptoms can include headaches, nausea (feeling sick), loss of appetite, vomiting, and changes in personality, mood, mental capacity and concentration. About a fifth of patients with glioma have seizures (fits) for months or years before the disease is diagnosed.
Glioma is a long-tem debilitating and life-threatening disease because of the severe damage to the brain that is associated with poor long-term survival.
At the time of designation, glioma affected not more than 2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of not more than 101,000 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).
At the time of designation, several medicines were authorised for the treatment of glioma in the EU. Treatments for glioma included surgery, radiotherapy (treatment with radiation), and chemotherapy (medicines to treat cancer) to improve survival. Patients also received treatments for the symptoms of glioma, including corticosteroids to reduce pressure within the skull and medicines to prevent seizures.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with glioma because it works in a different way to existing treatments and early studies show that it might improve the treatment of patients with this condition, particularly patients with a form of glioma called glioblastoma multiforme. These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Gliomas, like many solid tumours, rely on the growth of new blood vessels (angiogenesis) to obtain the nutrients that help tumour cells to grow and spread.
This medicine is an anti-angiogenic medicine (a medicine that stops the formation of new blood vessels). It is made up of a virus that contains a gene, called Fas-c, responsible for the production of a protein called Fas-c. The virus is expected to deliver copies of the Fas-c gene into cells in the body. As the Fas-c gene can only be activated in cells of the developing blood vessels, only the cells in the developing blood vessels will produce the Fas-c protein. The Fas-c protein is an engineered protein made of two parts: a part of a receptor called 'TNF receptor' to which a substance called TNF-alfa attaches, as well as a part of the Fas protein which activates cell death. When TNF-alfa attaches to the Fas-c protein, the Fas component of the protein is expected to cause the death of the developing blood vessels in gliomas. As a result, the medicine is expected to stop the formation of new blood vessels in glioma cells, which will prevent the growth of the tumour.
The type of virus used in this medicine ('adeno-associated virus') does not cause disease in humans.
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 glioma were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for glioma 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 April 2012 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
- Adenovirus-associated vector containing human Fas-c gene (ofranergene obadenovec)
- Intended use
- Treatment of glioma
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/12/1002
- Date of designation
- Sponsor
KCR S.A.
Ul. Postepu 6
02-676 Warsaw
Mazowieckie
Poland
Tel: +48 695801611
E-mail: karoline.hahn@kcrcro.com
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Update history
| Date | Update |
|---|---|
| September 2022 | Product withdrawn from the Union Register of orphan medicinal products on request of the Sponsor. |
| July 2021 | The sponsorship was transferred to KCR S.A., Poland. |
| April 2019 | The sponsorship was transferred to Envigo CRS S.A., Spain. |
| December 2015 | Gregory Fryer Associates Ltd changed name to Envigo Pharma Consulting Ltd. |
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: