• Email
  • Help

Orphan designation

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in June 2015 on request of the sponsor.

On 3 December 2008, orphan designation (EU/3/08/584) was granted by the European Commission to Ziopharm Oncology Limited, United Kingdom, for palifosfamide for the treatment of soft tissue sarcoma.

What is soft tissue sarcoma?

Soft tissue sarcoma is a type of cancer that affects the supportive tissues of the body. It can occur in muscles, blood vessels, fat tissue or in other tissues that support, surround and protect the organs. The cause of soft tissue sarcoma is largely unknown. Soft tissue sarcoma is a serious condition that can cause disability and is life threatening.

What is the estimated number of patients affected by soft tissue sarcoma?

At the time of designation, soft tissue sarcoma affected less than 2.5 in 10,000 people in the European Union (EU)*. This is below the threshold for orphan designation which is 5 in 10,000, and is equivalent to a total of fewer than 125,000 people. This is based on the information provided by the sponsor and 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 based on data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).

What treatments are available?

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

The sponsor has provided sufficient information to show that palifosfamide might be of significant benefit for patients with soft tissue sarcoma because it might improve treatment 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.

How is this medicine product expected to work?

Palifosfamide is the ‘active metabolite’ of ifosfamide, an anticancer medicine used to treat soft tissue sarcoma. It is called ‘a metabolite’ because it is formed when ifosfamide is broken down in the body and ‘active’ because it is the substance that does the work to treat the disease.

Palifosfamide is expected to work in the same way as ifosfamide by acting as an ‘alkylating agent’. Alkylating agents kill cancer cells by attaching to the cells’ DNA while they are dividing. This stops cell division and slows down the growth of the tumour.

Palifosfamide might also have fewer side effects than ifosfamide because when ifosfamide is broken down it forms other toxic substances in addition to palifosfamide. When palifosfamide is used these other toxic substances will not be produced.

What is the stage of development of this medicine?

The effects of palifosfamide have been evaluated in experimental models.

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

At the time of submission, palifosfamide was not authorised anywhere in the EU for soft tissue sarcoma. Orphan drug designation of the medicine was granted in the United States of America in May 2008 for the treatment of soft tissue sarcoma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 October 2008 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 Community) 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 marketing authorisation.

Key facts

Product details for <p>Palifosfamide</p>
Active substancePalifosfamide
Medicine Name
Disease/conditionTreatment of soft tissue sarcoma
Date of decision02/12/2008
Orphan decision numberEU/3/08/584

Review of designation

Sponsor’s contact details

Ziopharm Oncology Limited
3rd Floor
24 Chiswell Street
London EC1Y 4YX
United Kingdom
Tel. +44 (0)20 7382 1820
Fax +44 (0)20 7382 1821

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.