EU/3/17/1904 - orphan designation for treatment of glioma
Picropodophyllin
OrphanHuman
Overview
On 23 August 2017, orphan designation (EU/3/17/1904) was granted by the European Commission to Axelar AB, Sweden, for picropodophyllin for the treatment of glioma.
The sponsorship was transferred to Max Kemikonsult AB, Sweden in November 2020.
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 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 134,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 515,700,000 (Eurostat 2017).
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 the medicine might be of significant benefit for patients with glioma because early results showed responses in patients whose disease had not responded to or had come back after other standard treatments.This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The way that picropodophyllin works is not completely understood, but it is thought to act in two ways: firstly, by blocking the action of a protein called IGF-1R (insulin-like growth factor-1 receptor), which is known to be present in excess in many cancers, including glioma, and which stimulates their growth; secondly, by interfering with a process called microtubule formation, which is needed for cells to be able to divide. The combination of these two actions is expected to reduce the growth of the glioma cells and help prevent the spread of the cancer.
The effects of picropodophyllin 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, picropodophyllin 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 13 July 2017 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
- Picropodophyllin
- Medicine name
- -
- Intended use
- Treatment of glioma
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1904
- Date of designation
- Sponsor
Max Kemikonsult AB
Hanna Nilssons Vag 6 Lgh 1301
176 68 Jarfalla
Stockholms Lan
Sweden
Tel: +46737121632
E-mail: magnus.axelson@outlook.com
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: