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Orphan designation

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in April 2014 on request of the Sponsor.

On 11 April 2005, orphan designation (EU/3/05/274) was granted by the European Commission to ICON Consulting, United Kingdom, for melatonin for the treatment of non-24-hour sleep-wake disorders in blind people with no light perception.

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

What is non-24-hour sleep-wake disorders in blind people with no light perception?

The 24-hour rhythm in sleep and wakefulness is regulated by a “body clock”. This clock is essential for the timing of various physiological rhythms. The individual cycle of this body clock is usually slightly longer than 24 hours and therefore it is reset on a daily basis to fit 24 hours. This occurs due to changes in the blood concentration of a certain substance called melatonin. Melatonin is a naturally occurring substance produced by a small flat gland localised within the brain. The production of melatonin is normally regulated by the perception of light and darkness and helps to regulate the body clock. In blind people, who cannot perceive light, this clock cannot be reset and will therefore not be synchronised with real time. These people experience alternating periods of difficulties to initiate sleep at night with difficulties to wake-up in the morning, or have early sleep onset with early awakenings. Non-24-hour sleep-wake disorders in blind people with no light perception is a chronically debilitating condition.

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

At the time of designation, non-24-hour sleep-wake disorders in blind people with no light perception affected approximately 2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 93,000 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?

The concentration of melatonin in the blood needed to follow the daily sleep-wake rhythm will be mimicked by external administration and therefore, when given to blind individuals who cannot perceive light may adjust the daily sleep-wake rhythm and reduce the number and duration of daytime naps. 

What is the stage of development of this medicine?

The effects of melatonin were evaluated in experimental models. 

At the time of submission of the application for orphan designation, clinical trials in patients with non-24-hour sleep-wake disorders in blind people were ongoing. 

The medicinal product was not marketed anywhere worldwide for non-24-hour sleep-wake disorders in blind people with no light perception, at the time of submission. Orphan designation of melatonin was granted in the United States for treatment of circadian rhythm sleep disorders in blind people with no perception of light. 

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 March 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 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>Melatonin</p>
Active substanceMelatonin
Medicine Name
Disease/conditionTreatment of non-24-hour sleep-wake disorders in blind people with no light perception
Date of decision11/04/2005
Orphan decision numberEU/3/05/274

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:

ICON Consulting
2 Globeside
Globeside Business Park
Buckinghamshire SL7 1TB
United Kingdom
Tel. +44 (0)16 2849 6315
Fax +44 (0)16 2849 6301

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.