On 24 April 2015, orphan designation (EU/3/15/1472) was granted by the European Commission to Blue Earth Diagnostics Ltd, United Kingdom, for fluciclovine (18F) for the diagnosis of glioma.
The sponsorship was transferred to to Blue Earth Diagnostics Ireland Limited, in November 2019
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, 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 1.5 people in 10,000 in the European Union (EU). This was equivalent to a total of around 77,000 people*, and is below the ceiling for orphan designation. 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 eligible for diagnosis of 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 512,900,000 (Eurostat 2015).
At the time of the application for orphan designation, 3 products are approved for the diagnosis of glioma: fludeoxyglucose (18F), 6-[18F]fluoro-L-3,4-dihydroxyphenylalanine and 5-aminolevulinic acid (Gliolan).
The sponsor has provided sufficient information to show that fluciclovine (18F) might be of significant benefit for patients with glioma because early studies suggest that it might be better than current methods at distinguishing gliomas from normal tissue.This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Fluciclovine (18F) is a radiopharmaceutical: it is made up of a substance similar to leucine (an amino acid), which has been labelled with a small amount of radioactivity. This medicine can enter cells through proteins called 'amino acid transporters' (AATs), and early studies have shown that it enters glioma cells more readily because AATs in these cells are more active.
When given as injection into patients with glioma, the medicine is therefore expected to build up in the glioma cancer cells. Its small radioactivity can then be picked up by a sensitive scanner (known as a PET scanner) and the images obtained will help show the extent of the cancer in the body, which may lead to more complete surgical removal of the cancer.
The effects of fluciclovine (18F) have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with fluciclovine (18F) in patients with glioma were ongoing.
At the time of submission, fluciclovine (18F) 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 25 March 2015 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.
EU/3/15/1472: Public summary of positive opinion for orphan designation of Fluciclovine (18F) for the diagnosis of glioma
- Active substance
- Fluciclovine (18F)
- Intended use
- Diagnosis of glioma
- Orphan designation status
- EU designation number
- Date of designation
Blue Earth Diagnostics Ireland Limited
2 Grand Canal Square
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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:
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform: