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Orphan designation

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.

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 are the methods of treatment available?

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

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

According to the information provided by the sponsor, non-24-hour sleep-wake disorders in blind people with no light perception was considered to affect about 92,000 persons in the European Union. 

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

How is this medicinal product expected to act?

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 ofdevelopment of this medicinal product?

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. According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 3 March 2005 a positive opinion recommending the grant of the above-mentioned designation. 

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition,
  • the existence or not of alternative methods of diagnosis, prevention or treatment and 
  • either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments. 

Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this 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

Sponsor’s contact details:

ICON Consulting
2 Globeside, Globeside Business Park
Marlow, Buckinghamshire
United Kingdom
Telephne: +44 (0)16 28 49 63 15
Telefax: +44 (0)16 28 49 63 01

Patients’ associations contact points:

Action for Blind People
14-16 Verney Road
SE16 3DZ
United Kingdom
Telephone: +44 20 76 35 48 00
Telefax: +44 20 76 35 49 00

Fighting Blindness IR
Insight Counselling Centre
4 South Great George's Street
Dublin 2
Telephone: +35 1 67 46 496
Telefax: +35 1 67 46 497