EU/3/11/880 - orphan designation for treatment of pancreatic cancer
genetically modified human adenovirus encoding human PH20 hyaluronidase
OrphanHuman
Overview
On On 21 June 2011, orphan designation (EU/3/11/880) was granted by the European Commission to VCN Biosciences S.L., Spain, for genetically modified human adenovirus encoding human PH20 hyaluronidase for the treatment of pancreatic cancer.
For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.
Pancreatic cancer is cancer of the pancreas, a small organ that lies behind the stomach. The pancreas has two functions: to produce a juice that helps with the digestion of food, and to produce hormones such as insulin. Due to the absence of symptoms in the early stages of pancreatic cancer, the majority of patients are diagnosed when the cancer has spread locally or to other parts of the body.
Pancreatic cancer is a very severe and life-threatening disease that is associated with shortened life expectancy.
At the time of designation, pancreatic cancer affected approximately 1.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 66,000 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. At the time of designation, this represented a population of 507,700,000 (Eurostat 2011).
At the time of designation, several medicines were authorised in the EU for treating pancreatic cancer. The choice of treatment depended on several factors, including how advanced the disease is. Treatments included surgery, radiotherapy (treatment with radiation) 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 pancreatic cancer because it works in a different way to existing treatments and early studies in experimental models show that it might be used in combination with other treatments to improve the outcome of patients with this condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is made up of an 'oncolytic' virus, a virus that has been genetically modified so that it is able to target, replicate itself in and destroy tumour cells while sparing normal cells. When inside a tumour cell, the virus is expected to take over the cell's replication apparatus and use it to make more copies of itself. This is expected to kill the cell, leaving the virus to spread to neighbouring tumour cells.
At the time of submission of the application for orphan designation, the evaluation of the effects of genetically modified human adenovirus encoding human PH20 hyaluronidase in experimental models was ongoing.
At the time of submission, no clinical trials with the medicine in patients with pancreatic cancer had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for pancreatic 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 April 2011 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 human adenovirus encoding human PH20 hyaluronidase
- Intended use
- Treatment of pancreatic cancer
- Orphan designation status
- Positive
- EU designation number
- EU/3/11/880
- Date of designation
- Sponsor
Theriva Biologics S.L.
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 2024 | The sponsor's name changed to Theriva Biologics S.L. |
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: