EU/3/05/288

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.
The sponsor changed it’s name to NeuroSearch Sweden AB in June 2007.
The sponsorship was transferred to NSAB, Filial af NeuroSearch Sweden AB, Sverige, Denmark in July 2009.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 12 may 2005 a positive opinion recommending the grant of the above-mentioned designation.

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 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 neurons, the nervous system cannot regulate properly the target organs 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 is equivalent to a total of between 18,000 and 37,000 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 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. This represents a population of 459,700,000 (Eurostat 2004).

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.

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.