EU/3/10/806

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Orphan designation

On 26 November 2010, orphan designation (EU/3/10/806) was granted by the European Commission to Dr Hans Moebius, United Kingdom, for methylthioninium for the treatment of progressive non-fluent aphasia.

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 progressive non-fluent aphasia?

Progressive non-fluent aphasia is a brain disorder that affects the patient’s ability to speak fluently. The patient may lose the ability to speak correctly, start mispronouncing words or find it difficult to remember the right words. This worsens over time until the patient is unable to speak. Behaviour may also change, with the patient losing the ability to control or adjust their behaviour in different situations.

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 parts of the brain. The parts of the brain that are affected are the frontal and temporal (side) lobes.

Progressive non-fluent aphasia 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 progressive non-fluent aphasia?

At the time of designation, progressive non-fluent aphasia affected approximately 0.25 in 10,000 people in the European Union (EU). This was equivalent to a total of around 13,000 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 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 progressive non-fluent aphasia, 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 progressive non-fluent aphasia 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 progressive non-fluent aphasia 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 progressive non-fluent aphasia
Date of decision26/11/2010
OutcomePositive
Orphan decision numberEU/3/10/806

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.