EU/3/12/1093

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Orphan designation

On 24 January 2013, orphan designation (EU/3/12/1093) was granted by the European Commission to Idis Ltd, United Kingdom, for 1,2:5,6-dianhydrogalactitol for the treatment of glioma.

What is glioma?

Glioma is a type of brain tumour that affects the ‘glial’ cells (the cells that surround and support the nerve cells). Patients with glioma can have severe symptoms, but the types of symptoms experienced depend on where the tumour develops in the brain.

Symptoms can include headaches, nausea (feeling sick), loss of appetite, vomiting, and changes in personality, mood, mental capacity and concentration. About a fifth of patients with glioma have seizures (fits) for months or years before the disease is diagnosed.

Glioma is a long-term debilitating and life-threatening disease because of the severe damage to the brain, and is associated with poor long-term survival.

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

At the time of designation, glioma affected approximately 2.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 112,000 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, several medicines were authorised for the treatment of glioma in the EU. Treatments for glioma included surgery, radiotherapy (treatment with radiation), and chemotherapy (medicines to treat cancer) to improve survival. Patients also received treatments for the symptoms of glioma, including corticosteroids to reduce pressure within the skull and medicines to prevent seizures.

The sponsor has provided sufficient information to show that 1,2:5,6-dianhydrogalactitol might be of significant benefit for patients with glioma because early studies show that it might improve the 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 expected to work?

1,2:5,6-Dianhydrogalactitol is a small molecule that can easily cross the blood-brain barrier that separates the blood from the brain tissue. It is expected to work as an ‘alkylating agent’. This means that it attaches a chemical group called an ‘alkyl’ group to DNA of cells while they are reproducing. This damages the DNA and in turn disrupts cell division. As a result, the tumour cells in the brain cannot reproduce, and this slows down the growth of the glioma.

What is the stage of development of this medicine?

The effects of 1,2:5,6-dianhydrogalactitol have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with 1,2:5,6-dianhydrogalactitol in patients with glioma were ongoing.

At the time of submission, 1,2:5,6-dianhydrogalactitol was not authorised anywhere in the EU for glioma.

Orphan designation of 1,2:5,6-dianhydrogalactitol had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 December 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>1,2:5,6-Dianhydrogalactitol</p>
Active substance1,2:5,6-Dianhydrogalactitol
Medicine Name
Disease/conditionTreatment of glioma
Date of decision24/01/2013
OutcomePositive
Orphan decision numberEU/3/12/1093

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

Idis Ltd
Idis House
Churchfield Road
Weybridge KT13 8DB
United Kingdom
Tel. +44 (0)1932 824000
Fax +44 (0)1932 824200
E-mail: gma@idispharma.com

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.