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Orphan designation

On 19 June 2013, orphan designation (EU/3/13/1145) 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 soft-tissue sarcoma.

In November 2015, Oncos Therapeutics Oy changed name to Targovax Oy.

What is soft-tissue sarcoma?

Soft-tissue sarcomas are a type of cancer that affect the soft, supportive tissues of the body. They can occur in muscles, blood vessels, fat tissue or in other tissues that support, surround and protect organs. Patients with soft-tissue sarcoma do not usually have symptoms in the early stages of the disease. First symptoms appear when the tumour grows large enough to cause swelling and pain.

Soft-tissue sarcoma is a life-threatening disease particularly when the cancer has spread to other parts of the body.

What is the estimated number of patients affected by the condition?

At the time of designation, soft-tissue sarcoma affected approximately 3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 152,000 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 509,000,000 (Eurostat 2013).

What treatments are available?

At the time of designation, the main treatment for early-stage soft-tissue sarcoma was surgery. For large sarcomas, surgery was usually followed by radiotherapy (treatment with radiation) and chemotherapy (medicines to treat cancer) to kill any cancerous cells that were left behind. Several medicines were authorised in the EU for the treatment of soft-tissue sarcoma.

The sponsor has provided sufficient information to show that genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor might be of significant benefit for patients with soft-tissue sarcoma because early studies suggest that it may make treatment of the tumour more effective when it is combined with existing medicines. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

This medicine contains a virus that has been modified by the insertion of a gene 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 soft-tissue sarcoma, 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, which stimulates the immune system (the body’s natural defences) to attack the cancerous cells.

What is the stage of development of this medicine?

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 soft-tissue sarcoma were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for soft-tissue sarcoma 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 15 May 2013 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:
  • 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

Product details for <p>genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor</p>
Active substancegenetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor
Medicine Name
Disease/conditionTreatment of soft-tissue sarcoma
Date of decision19/06/2013
Orphan decision numberEU/3/13/1145

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details

Targovax Oy
Saukonpaadenranta 2
00180 Helsinki
Tel. +358 10 279 4000

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.