EU/3/05/288

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Orphan designation

On 20 June 2005, orphan designation (EU/3/05/288) was granted by the European Commission to A Carlsson Research AB, Sweden, for 4-[3-(methylsulfonyl)phenyl]-1-propylpiperidine x HC1 for the treatment of Huntington's disease.

In June 2007, A Carlsson Research AB changed name to NeuroSearch Sweden AB.

The sponsorship was transferred to NSAB, Filial af NeuroSearch Sweden AB, Sverige, Denmark in July 2009 and subsequently to Teva Pharma GmbH, Germany, in April 2013.

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 basal ganglia and cerebral cortex) degenerate. When activated, the neurons release a group of substances, the so-called neurotransmitters. Once released, the neurotransmitters activate or inhibit the target cells in the different body organs. In Huntington’s disease, due to the extensive degeneration of neurones, the nervous system cannot regulate the target organs properly anymore. This leads to the typical symptoms of the disease, namely involuntary movements, behavioural disturbances and mental deterioration. The disease progresses over time and is chronically debilitating with potentially life-threatening complications.

What is the estimated number of patients affected by the condition?

At the time of designation, Huntington's disease affected between 0.4 to 0.8 in 10,000 people in the European Union (EU). This was equivalent to a total of between 19,000 and 37,000 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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 466,600,000 (Eurostat 2005).

What treatments are available?

No satisfactory methods exist that were authorised at the time of application.

How is this medicine expected to work?

4-[3-(Methylsulfonyl)phenyl]-1-propylpiperidine x HC1 acts by stabilising the effects of a neurotransmitter called dopamine, which might result in some improvement in the clinical symptoms associated with Huntington’s disease.

What is the stage of development of this medicine?

The evaluation of the effects of 4-[3-(methylsulfonyl)phenyl]-1-propylpiperidine x HC1 in experimental models is ongoing. At the time of submission of the application for orphan designation, clinical trials in patients with Huntington's disease were ongoing.

The medicinal product was not marketed anywhere worldwide for Huntington's disease or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 May 2005 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 Community) 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>4-[3-(Methylsulfonyl)phenyl]-1-propylpiperidine x HC1</p>
Active substance4-[3-(Methylsulfonyl)phenyl]-1-propylpiperidine x HC1
Medicine Name
Disease/conditionTreatment of Huntington's disease
Date of decision20/06/2005
OutcomePositive
Orphan decision numberEU/3/05/288

Review of designation

Sponsor’s contact details:

Teva Pharma GmbH
Graf-Arco-Str. 3
89079 Ulm
Germany
Tel. +49 6105 97 676 17
Fax +49 6105 97 767 60
Schreiben Sie uns!

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.