European Medicines Agency
Pre-authorisation Evaluation of Medicines for Human Use
Document Date: London, 27 October 2005
Doc.Ref.: EMEA/COMP/138419/2005
COMMITTEE FOR ORPHAN MEDICINAL PRODUCTS
PUBLIC SUMMARY OF
POSITIVE OPINION FOR ORPHAN DESIGNATION
OF melatonin for the treatment of non-24-hour sleep-wake disorders in blind people with no light perception
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.
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 of development of this medicinal product?
The effects of melatonin were evaluated in experimental models.
7 Westferry Circus, Canary Wharf, London, E14 4HB, UK
Tel. (44-20) 74 18 84 00 Fax (44-20) 75 23 70 40
E-mail: orphandrugs@emea.eu.int www.emea.eu.int
.EMEA 2005 Reproduction and/or distribution of this document is authorised for non-commercial purposes only provided the EMEA is acknowledged
At the time of submission of the application for orphan designation, clinical trials in patients with non24-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:
(i) the seriousness of the condition,
(ii) the existence or not of alternative methods of diagnosis, prevention or treatment.
(iii) Either the rarity of the condition (considered to affect not more than five in tenthousand 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.
For more information:
Sponsor's contact details:
ICON Consulting
2 Globeside, Globeside Business Park
Marlow, Buckinghamshire
SL7 1TB
United Kingdom
Telephne: +44 (0)16 28 49 63 15
Telefax: +44 (0)16 28 49 63 01
E-mail: ledug@iconuk.com
COOKING FOR ONE THE EASY WAY.
Starting to learn to cook and prepare food for yourself and maybe other members of your family or friends can seem a daunting task if you are just starting out in your own home with a disability. Some things are just common sense, but you would be surprised at the lack of kitchen design when you are trying to reach up or down to cupboards.
Start by thinking about the essential items that you use every day and put them in an order that you know you can reach and access the working surface easily. make sure the cooker is at a height that you can work with while handling hot dishes.
If you have a problem with standing, make sure that you get yourself a leaning stool. This is a stool that you can adjust to the height you need to half sit to manage saucepans on the hob, or to prepare vegetables etcetera. The opposite is needed if you are in a wheelchair you need your cooker at a level you are comfortable with. (higher up and with a door that opens sideways is easier to transfer food in and out of the oven).
Store cupboard items.
make a list of all the store cupboard items that you use regularly, and keep them within easy reach everybody uses items like Sugar Flour Bread Pasta Rice Spices Cooking oil vinegar cereal stock cubes and gravy granules. I expect you can think of more that you regularly use.
If you have a blind person in the house, try to acquire cupboards that have doors that lift or roll up out of the way, ( I have lost count of the times my partner has walked into an open cupboard door!)
One pot meals are brilliant for cooking for one or two as you can throw everything in and serve up with salad or bread and there is not too much washing up! a bit of research at the supermarket and on line for easy meals that are healthy and nutritious pay dividends for the future. Would you like to see recipes here or websites to try? one I have used is sofeminine.com Men don't be put off by the name, it's a really good site for easy meals.