EU/3/08/597

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Orphan designation

On 20 January 2009, orphan designation (EU/3/08/597) was granted by the European Commission to Innovative Drug European Associates Limited, United Kingdom, for 2,3,4,5 tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride for the treatment of Huntington’s disease.

In December 2012, Innovative Drug European Associates Limited changed name to IDEA Innovative Drug European Associates Limited.

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

What is Huntington’s disease?

Huntington’s disease is a hereditary disease where the cells (neurons) of specific areas of the brain (the so-called basal ganglia and cerebral cortex) degenerate. When activated, normal neurons release several substances known as neurotransmitters. Once released, neurotransmitters activate or inhibit the target cells in the different body organs. In Huntington’s disease, because of extensive degeneration of neurons, the nervous system cannot regulate properly the target organs anymore. This leads to symptoms typical of Huntington’s, such as involuntary movements, behavioural disturbances and mental deterioration. The disease progresses over time and is long-lasting with potentially life-threatening complications.

What is the estimated number of patients affected by Huntington’s disease?

At the time of designation, Huntington’s disease affected between 0.4 and 0.8 in 10,000 people in the European Union (EU). This was equivalent to a total of between 20,000 and 40,000 people*, and is below the ceiling 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 504,800,000 (Eurostat 2009).

What treatments are available?

At the time of submission of the orphan drug designation application, some products were authorised for the symptomatic treatment of the Huntington’s disease. In some member states, tetrabenazine, haloperidol, pimozide, and tiapride were authorised.

The sponsor has provided sufficient information to show that 2,3,4,5 tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride might be of potential significant benefit for the patients because its new mechanism of action could lead to an improvent of the overall outcome of the patients. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status. 

How is this medicine expected to work?

The mechanism of action of 2,3,4,5 tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride is not fully understood. However, this medicinal product is expected to have a neuroprotective effect (protect neurons from degenerating) in patients with Huntington’s disease.

What is the stage of development of this medicine?

The effects of 2,3,4,5 tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials in patients with Huntington’s disease were ongoing.

At the time of submission, 2,3,4,5 tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride was not authorised anywhere in the world for Huntington’s disease or designated as 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 5 November 2008 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 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

Product details for <p>2,3,4,5 Tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride</p>
Active substance2,3,4,5 Tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride
Medicine Name
Disease/conditionTreatment of Huntington's disease
Date of decision20/01/2009
OutcomePositive
Orphan decision numberEU/3/08/597

Review of designation

Sponsor’s contact details

IDEA Innovative Drug European Associates Limited
The Jeffrey’s Building
St John’s Innovation Park
Cowley Road
Cambridge CB4 0DS
United Kingdom
Tel. + 44 (0)12 23 40 26 66
Fax + 44 (0)12 23 413 689
E-mail: info@eurepresentative.com

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.