EU/3/16/1629 - orphan designation for treatment of idiopathic intracranial hypertension
exenatide
OrphanHuman
Overview
On 21 March 2016, orphan designation (EU/3/16/1629) was granted by the European Commission to Alan Boyd Consultants Ltd, United Kingdom, for exenatide for the treatment of idiopathic intracranial hypertension.
The sponsorship was transferred to Boyd Consultants Limited, Ireland, in March 2019.
The sponsor’s address was updated in September 2021.
Idiopathic intracranial hypertension is a condition where there is increased pressure in the cerebrospinal fluid (fluid around the brain) with no known cause. Idiopathic intracranial hypertension most often occurs in obese women of childbearing age and is a debilitating condition because symptoms include vision loss, which may be permanent, and severe headaches.
At the time of designation, idiopathic intracranial hypertension affected approximately 1.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 72,000 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 513,700,000 (Eurostat 2016).
At the time of designation, no medicines were authorised for the treatment of idiopathic intracranial hypertension. A carbonic anhydrase inhibitor, such as acetazolamide, was used to treat idiopathic intracranial hypertension, although it was not authorised for use in this disease. Surgery to reduce pressure on the brain was sometimes performed. Lumbar punctures (drawing out fluid from the spine) were used to improve symptoms, although effects usually lasted for a few days.
It is thought that a protein in the body called glucagon-like peptide (GLP-1) reduces the amount of cerebrovascular fluid. Exenatide acts in a similar way to GLP-1 and it is also expected to reduce the amount of cerebrovascular fluid. This is expected to reduce intracranial pressure.
The effects of exenatide have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with exenatide in patients with idiopathic intracranial hypertension had been started.
At the time of submission, exenatide was authorised in the EU and the United States of America for type-2 diabetes.
At the time of submission, exenatide was not authorised anywhere in the EU for idiopathic intracranial hypertension 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 18 February 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
- exenatide
- Intended use
- Treatment of idiopathic intracranial hypertension
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1629
- Date of designation
- Sponsor
Boyd Consultants Limited
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: