EU/3/12/994

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Orphan designation

On 25 May 2012, orphan designation (EU/3/12/994) was granted by the European Commission to Laboratoires OncoTherapy Science France, S.A.R.L., France, for yttrium (90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10 for the treatment of soft tissue sarcoma.

What is soft tissue sarcoma?

Soft tissue sarcoma is a type of cancer that affects the soft, supportive tissues of the body. It 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 this condition?

At the time of designation, soft tissue sarcoma 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 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).

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 the medicine might be of significant benefit for patients with soft tissue sarcoma because early studies in experimental models show that it works in a different way to existing treatments. The medicine is expected to specifically target and kill certain types of soft tissue sarcoma cells. These assumptions 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 consists of three parts: an antibody (a type of protein), which can recognise and attach to a receptor called ‘frizzled homologue 10’ that is present at high levels on the surface of some soft tissue sarcoma cells; a radioactive compound called yttrium (90Y), which emits a type of radiation known as beta radiation; and a chemical (DTPA). The antibody acts as a carrier for the radioactive compound to the tumour cells, while DTPA is expected to prevent yttrium detaching from the antibody before it has reached the tumour, avoiding radiation to other organs. When the antibody attaches to the soft tissue sarcoma cells expressing frizzled homologue 10, the radioactive compound yttrium (90Y) emits radiation which is expected to kill the tumour 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 8 March 2012 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>Yttrium (<sup>90</sup>Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10</p>
Active substanceYttrium (90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10
Medicine Name
Disease/conditionTreatment of soft tissue sarcoma
Date of decision25/05/2012
OutcomePositive
Orphan decision numberEU/3/12/994

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:

Laboratoires OncoTherapy Science France, S.A.R.L.
World Trade Center Lyon
Tour Oxygène
10-12 boulevard Vivier Merle
69393 Lyon Cedex 03
France
Telephone: +33 472 40 57 52
E-mail: info@oncotherapy.co.jp

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.