EU/3/11/853

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Orphan designation

On 15 April 2011, orphan designation (EU/3/11/853) was granted by the European Commission to Sanofi-Aventis, France, for ombrabulin for the treatment of soft tissue sarcoma.

In October 2012, Sanofi Aventis changed name to Sanofi-Aventis Groupe.

For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.

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 serious and 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 2.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 122,000 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).

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 ombrabulin might be of significant benefit for patients with soft tissue sarcoma because it works in a different way to existing treatments, and early studies show that it might be used in combination with chemotherapy to improve the outcome of patients with this condition. 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?

Ombrabulin is derived from combretastatin, a natural substance extracted from a bark of a South African tree. It is expected to work in soft tissue sarcoma mainly by disrupting the vessels that bring blood to the tumour, blocking the normal flow of the blood. Cancer cells grow rapidly and thus require a large amount of blood. By blocking the blood flow, ombrabulin is expected to stop the growth of cancer cells, which eventually die.

Ombrabulin is also expected to disrupt the division of cancer cells by attaching to a protein in cells called ‘tubulin’, which is important in the formation of the internal ‘skeleton’ that cells need to assemble when they divide.

What is the stage of development of this medicine?

The effects of ombrabulin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with ombrabulin in patients with soft tissue sarcoma were ongoing.

At the time of submission, ombrabulin 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 12 January 2011 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.

Name Language First published Last updated
EU/3/11/853: Public summary of positive opinion for orphan designation: Ombrabulin for the treatment of soft tissue sarcoma (English only) 26/04/2011 09/04/2013

Key facts

Product details for <p>Ombrabulin</p>
Active substanceOmbrabulin
Medicine Name
Disease/conditionTreatment of soft tissue sarcoma
Date of decision15/04/2011
OutcomePositive
Orphan decision numberEU/3/11/853

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

Sanofi-Aventis Groupe
54 rue de la Boétie
75008 Paris
France
Tel. +33 1 53 77 40 00
Fax +33 1 53 77 41 33
www.sanofi-aventis.com/contact/contact.asp

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.