EU/3/10/807

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Orphan designation

On 26 November 2010, orphan designation (EU/3/10/807) was granted by the European Commission to Dr Hans Moebius, United Kingdom, for methylthioninium for the treatment of frontotemporal dementia with parkinsonism-17.

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

For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.

What is frontotemporal dementia with parkinsonism-17?

Frontotemporal dementia with parkinsonism-17 is a brain disorder with a wide range of symptoms that worsen over time. Patients may lose the ability to control or adjust their behaviour in different situations, leading to inappropriate behaviour. Language skills may also be affected, with the patient losing the ability to speak correctly, to pronounce words properly or to remember the right words. Patients may also show signs of Parkinson’s disease such as shaking, slow movement and muscular stiffness.

The exact cause of the disease is unclear, but 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.

Frontotemporal dementia with parkinsonism-17 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 frontotemporal dementia with parkinsonism-17?

At the time of designation, frontotemporal dementia with parkinsonism-17 affected approximately 0.03 in 10,000 people in the European Union (EU). This was equivalent to a total of around 2,000 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 at the time of application. Patients were supported in their day-to-day activities by caregivers with help from experts such as speech therapists, psychologists, and physiotherapists.

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 frontotemporal dementia with parkinsonism-17, 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 frontotemporal dementia with parkinsonism-17 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 frontotemporal dementia with parkinsonism-17 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 frontotemporal dementia with parkinsonism-17
Date of decision26/11/2010
OutcomePositive
Orphan decision numberEU/3/10/807

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

Prof. Claude Wischik
TauRx Therapeutics 
Institute of Medical Sciences 
University of Aberdeen 
Aberdeen AB25 2ZD 
United Kingdom
Tel. +44 (0)1224 555191
Fax +44 (0)1224 555173
E-mail: c.m.wischik@abdn.ac.uk

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.