EU/3/18/2043 - orphan designation for treatment of glioma
carboplatin
sodium valproate
OrphanHuman
Overview
On 31 July 2018, orphan designation (EU/3/18/2043) was granted by the European Commission to Dr Ulrich Granzer, Germany, for combination of carboplatin and sodium valproate for the treatment of glioma.
The sponsorship was transferred to Granzer Regulatory Consulting & Services GmbH, Germany in March 2022.
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 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 it is associated with poor long-term survival.
At the time of designation, glioma affected approximately 2.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 135,000 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 517,400,000 (Eurostat 2018).
At the time of designation, several medicines were authorised for the treatment of glioma in the EU. Treatments for glioma included radiotherapy (treatment with radiation), chemotherapy (medicines to treat cancer) and surgery. 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 the medicine might be of significant benefit for patients with glioma because early data showed that patients treated with this medicine lived longer when compared indirectly with results seen with authorised treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is a combination of the cancer medicine carboplatin and the epilepsy medicine sodium valproate, both of which have been used for many years in the EU for other conditions.
Carboplatin belongs to a group of medicines called alkylating agents. Alkylating agents are highly reactive chemicals that attach to genetic material in the cell, and can damage or kill the cell.
In glioma, sodium valproate is expected to have several actions, including keeping the genes that suppress the growth of the cancer cells switched ‘on’. This is expected to slow down the growth of the cancer.
The medicines are expected to be delivered directly into the brain from a special device implanted at the back of the head.
The effects of carboplatin and sodium valproate alone into the brain have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the combination in patients with glioma had been started.
At the time of submission, the combination 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 21 June 2018 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
- carboplatin
- sodium valproate
- Intended use
- Treatment of glioma
- Orphan designation status
- Positive
- EU designation number
- EU/3/18/2043
- Date of designation
- Sponsor
Granzer Regulatory Consulting & Services GmbH
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: