EU/3/14/1305 - orphan designation for treatment of glioma
Humanised recombinant monoclonal antibody against epidermal growth factor receptor conjugated to maleimidocaproyl monomethylauristatin F(depatuxizumab mafodotin
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in October 2019 on request of the Sponsor.
This medicine is now known as depatuxizumab mafodotin.
On 29 July 2014, orphan designation (EU/3/14/1305) was granted by the European Commission to AbbVie Ltd, the United Kingdom, for humanised recombinant monoclonal antibody against epidermal growth factor receptor conjugated to maleimidocaproyl monomethylauristatin F for the treatment of glioma.
The sponsorship was transferred to AbbVie Deutschland GmbH & Co. KG, Germany, in May 2018.
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 one 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.
At the time of designation, glioma affected less than 2 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 102,000 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 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).
At the time of designation, several medicines were authorised for the treatment of glioma in the EU. Treatments 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 this medicine might be of significant benefit for patients with glioma because early studies suggest it could reduce tumour size in patients resistant to other treatments, when the medicine is used in combination with standard treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is made up of a monoclonal antibody (a type of protein) that has been designed to attach mainly to receptors on the surface of glioma cells called epidermal growth factor receptors (EGFRs). In glioma cells, these receptors are involved in the growth and spread of the tumour. The monoclonal antibody in this medicine is attached to monomethyl auristatin F, a cytotoxic (cell-killing) molecule, and is expected to deliver the monomethyl auristatin F to the glioma cells. Once inside the tumour cells, it is expected to kill them and thereby slow the progression of the disease.
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 glioma were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for glioma. Orphan designation has 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 12 June 2014 recommending the granting of this designation.
- 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
- Active substance
- Humanised recombinant monoclonal antibody against epidermal growth factor receptor conjugated to maleimidocaproyl monomethylauristatin F(depatuxizumab mafodotin
- Intended use
- Treatment of glioma
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/14/1305
- Date of designation
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: