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Orphan designation

On 26 November 2010, orphan designation (EU/3/10/805) was granted by the European Commission to Dr Hans Moebius, United Kingdom, for methylthioninium for the treatment of behavioural variant frontotemporal dementia. 

The sponsorship was transferred to Prof. Claude Wischik, United Kingdom, in February 2012.

The sponsorship was transferred to Prof. Claude Wischik, United Kingdom, in February 2012 and subsequently to TauRx Therapeutics Europe Ltd, United Kingdom, in August 2016.

What is behavioural variant frontotemporal dementia?

Behavioural variant frontotemporal dementia is a brain disorder in which patients gradually lose their ability to control or adjust their behaviour in different situations, leading to inappropriate behaviour. Language skills may also be affected, with the patient not being able to speak correctly, to pronounce words properly or to remember the right words.

The exact cause of the disease is unclear, but it is thought to be related to the abnormal clumping together of proteins in the brain called ‘tau’, damaging different areas of the brain. The parts of the brain that are affected are the frontal and temporal (side) lobes.

Behavioural variant frontotemporal dementia is a debilitating disease that is life threatening because of its damaging effects on the brain.

What is the estimated number of patients affected by behavioural variant frontotemporal dementia?

At the time of designation, behavioural variant frontotemporal dementia affected approximately 1.5 in 10,000 people in the European Union (EU). This is equivalent to a total of around 76,000 people*, and is below the threshold 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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 506,300,000 (Eurostat 2010).

What treatments are available?

No satisfactory methods of treatment were authorised in the EU at the time of designation. Patients were supported in their day-to-day activities by caregivers with help from experts such as psychologists, physiotherapists and speech therapists.

How is this medicine expected to work?

Methylthioninium is expected to work by dissolving the abnormal tangles of tau proteins in the brain of patients who have behavioural variant frontotemporal dementia, thereby slowing down or reversing the symptoms of the disease.

What is the stage of development of this medicine?

The effects of methylthioninium have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with methylthioninium in patients with behavioural variant frontotemporal dementia had been started.

At the time of submission, methylthioninium was used or authorised in several countries for the treatment of other diseases including urinary-tract infection, drug-induced methaemoglobinaemia, ifosfamide encephalopathy, and refractory shock syndromes.

At the time of submission, methylthioninium was not authorised anywhere in the EU for behavioural variant frontotemporal dementia 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 9 September 2010 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria
  • 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 European Union) 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

Product details for <p>Methylthioninium</p>
Active substanceMethylthioninium
Medicine Name
Disease/conditiontreatment of behavioural variant frontotemporal dementia
Date of decision26/11/2010
Orphan decision numberEU/3/10/805

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details

TauRx Therapeutics Europe Ltd
395 King Street
Aberdeen AB24 5RP
United Kingdom
Tel. +44 (0)1224 440902

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.