EU/3/10/781

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Orphan designation

On 20 September 2010, orphan designation (EU/3/10/781) was granted by the European Commission to to-BBB technologies BV, the Netherlands, for glutathione-pegylated liposomal doxorubicin hydrochloride for the treatment of glioma.

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

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 symptom 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 debilitating and life-threatening disease because it causes 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 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 people*, and is below the threshold 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 506,300,000 (Eurostat 2010).

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). 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 glutathione-pegylated liposomal doxorubicin hydrochloride might be of significant benefit for patients with glioma because the medicine is a new formulation of the anticancer medicine doxorubicin. This formulation may allow better delivery of doxorubicin across the blood-brain barrier, which separates the blood from the brain tissue. 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?

Doxorubicin has been available as an anticancer medicine since the 1960s. It is a cytotoxic (cell-killing) medicine that belongs to the group ‘anthracyclines’. It works by interfering with the DNA within cells, preventing them from making more copies of DNA and making proteins. This means that cancer cells cannot divide and eventually die.

In this medicine, doxorubicin is contained in ‘glutathione-pegylated liposomes’, which are tiny fatty spheres that are coated with a natural substance called glutathione. These have been designed to allow doxorubicin to better cross the blood-brain barrier using the ‘glutathione transporters’, proteins that are found at high levels on the blood-brain barrier and usually move glutathione from the blood into the brain. The liposomes are expected to attach to these transporters, to be carried across the blood-brain barrier into the brain tissue, where they will release the doxorubicin that they contain.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of glutathione-pegylated liposomal doxorubicin hydrochloride in experimental models was ongoing.

At the time of submission of the application for orphan designation, no clinical trials with glutathione-pegylated liposomal doxorubicin hydrochloride in patients with glioma had been started.

At the time of submission, glutathione-pegylated liposomal doxorubicin hydrochloride was not authorised anywhere in the EU for glioma 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 2 June 2010 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>Glutathione-pegylated liposomal doxorubicin hydrochloride</p>
Active substanceGlutathione-pegylated liposomal doxorubicin hydrochloride
Medicine Name
Disease/conditionTreatment of glioma
Date of decision20/09/2010
OutcomePositive
Orphan decision numberEU/3/10/781

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

BBB technologies BV
J.H. Oortweg 19
2333 CH, Leiden
The Netherlands
Tel. + 31 71 33 222 55
Fax + 31 84 83 13 409
E-mail: info@tobbb.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.