EU/3/04/225 - orphan designation for treatment of systemic monochloroacetate poisoning
sodium dichloroacetate
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in December 2008 on request of the Sponsor.
On 2 September 2004, orphan designation (EU/3/04/225) was granted by the European Commission to Dr Anthony W. Fox, Germany, for sodium dichloroacetate for the treatment of systemic monochloroacetate poisoning.
Industrial activities are a potential source of accidental exposure to a substance called monochloracetate. Monochloracetate might enter the body through the skin (most importantly) but also by breathing or eating.
Systemic monochloroacetate poisoning is characterised by accumulation of an organic acid (lactate) in the blood, which leads to nausea, vomiting, difficulties in breathing, low blood pressure, seizures and confusion. The condition is life-threatening.
At the time of designation, systemic monochloroacetate poisoning affected not more than 0.01 in 10,000 people in the European Union (EU)*. This is equivalent to a total of not more than 500 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 based on data from the European Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004).
Methods that were used at the time of application consisted of decontamination of skin through chemical neutralisation of the poison and general supportive measures. No satisfactory methods exist that were authorised at the time of application.
Sodium dichloroacetate would act as an antidote (counteracting the effects of the poison) and directly oppose the harmful systemic action of monochloroacteate by inducing breakdown of the accumulated lactate.
The effects of sodium dichloroacetate were evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials in patients with systemic monochloroacetate poisoning were initiated.
The medicinal product was not marketed anywhere worldwide for systemic monochloroacetate poisoning, at the time of submission. Orphan designation of sodium dichloroacetate was granted in the United States as an antidote in the management of systemic monochloroacetic acid poisoning.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 22 July 2004 a positive opinion recommending the grant of the above-mentioned designation.
- 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
- Active substance
- sodium dichloroacetate
- Intended use
- Treatment of systemic monochloroacetate poisoning
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/04/225
- Date of designation
- Sponsor
Dr Anthony W. Fox
EBD Group
Neuhauser Strasse 3
D-80331 München
Germany
Telephone: +49 89 51 39 98 03
Telefax: +49 89 54 42 02 14
E-mail: awfox@ebdgroup.com
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: