EU/3/16/1764 - orphan designation for diagnosis of glioma
tetrofosmin
OrphanHuman
Overview
On 14 October 2016, orphan designation (EU/3/16/1764) was granted by the European Commission to ProActina, Greece, for tetrofosmin for the diagnosis of 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 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, the number of patients eligible for diagnosis of glioma was estimated to be approximately 1.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 82,000 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 513,700,000 (Eurostat 2016).
At the time of designation, several imaging methods were used for the diagnosis of glioma, such as magnetic resonance imaging (MRI), computed tomography (CT) and positron emission tomography (PET) using so-called contrast agents or radioactive tracers to obtain better images of organs and tissues. Agents approved for the diagnosis of glioma included fludeoxyglucose (18F), 6-[18F]fluoro-L-3,4-dihydroxyphenylalanine (FDOPA) and 5-aminolevulinic acid (Gliolan).
The sponsor has provided sufficient information to show that tetrofosmin might be of significant benefit for patients with glioma, with published data showing it could improve recognition of advanced tumour. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Tetrofosmin is combined with a radioactive substance (technetium 99m) before being used for imaging. The combination is given by injection and normally does not enter the brain as it cannot pass the blood-brain barrier that separates the blood from brain tissue. However, in patients with glioma the proteins that are important for maintaining a tight barrier are no longer functioning properly, leading to gaps in the barrier. This means that tetrofosmin can enter the brain in areas where the cancer is active. It is then taken up by the glial cells and the radioactivity can then be picked up by the scanner. The images obtained are expected to help show the extent of the cancer. The small amount of radioactivity in the medicine disappears quickly and has little effect on the body.
The sponsor has provided non-clinical and clinical data from the published literature to support its application for orphan designation.
At the time of submission, tetrofosmin was authorised in the EU for heart and breast tumour imaging.
At the time of submission, tetrofosmin was not authorised anywhere in the EU for diagnosis of 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 8 September 2016 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
- tetrofosmin
- Intended use
- Diagnosis of glioma
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1764
- Date of designation
- Sponsor
ProActina
Greece
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:
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.
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: