EU/3/17/1926 - orphan designation for treatment of ovarian cancer
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Please note that this product was withdrawn from the Union Register of orphan medicinal products in September 2022 on request of the Sponsor.
On 16 October 2017, orphan designation (EU/3/17/1926) was granted by the European Commission to Envigo Pharma Consulting Limited, United Kingdom, for ofranergene obadenovec (also known as VB-111) 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 aged over 50 years. Due to the absence of clear symptoms in the early stages of the disease, the majority of women are diagnosed when the cancer has spread to other parts of the body.
Ovarian cancer is a 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 154,000 people1, 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).
1Disclaimer: 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 515,700,000 (Eurostat 2017).
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 results from early studies suggest that, when used in patients in whom platinum-based treatments have failed, the medicine may increase the length of time patients live compared with other authorised treatments.
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Ovarian cancer, like many solid tumours, relies on the growth of new blood vessels (angiogenesis) to obtain the nutrients that cancer cells need to grow and spread.
This medicine is made up of a virus that contains the gene for an engineered protein made up from two parts called Fas and TNFR1. The medicine has been designed so that the engineered protein is produced only in cells that line up the new blood vessels that supply the cancer tissue. When a substance, TNF, which is produced by the body, attaches to the TNFR1 part of the protein, it activates the Fas part which is involved in the regulation of cell death (apoptosis). This is expected to cause the death of the blood vessel cells, slowing down the growth of the cancer.
The type of virus used in this medicine ('adenovirus') is modified so that it 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 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. Orphan designation of the medicine had been granted in the EU for the treatment of glioma (a type of brain cancer).
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 September 2017 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
- ofranergene obadenovec
- Intended use
- Treatment of ovarian cancer
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/17/1926
- 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. |
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: