EU/3/06/385

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Orphan designation

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in December 2008 on request of the sponsor.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 25 July 2006, orphan designation (EU/3/06/385) was granted by the European Commission to VASTox Plc, United Kingdom, for 2-(4-(diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine for the treatment of Duchenne muscular dystrophy.

What is Duchenne muscular dystrophy?

Duchenne muscular dystrophy is an inherited genetic disease, which usually starts before the age of 6 years. It is characterised by progressive weakness of the muscles, first involving the hips and legs, and later also the muscles of the chest and arms. Genes located on structures present in each cell of the body (the chromosomes) carry the information that characterises each individual. In humans, the so-called X and Y-chromosomes determine the sex (males have one X and one Y, females have 2 Xs), but carry also other genetic information. Duchenne muscular dystrophy is caused by an abnormality of a gene located on the X chromosome. This gene is responsible for the production of a protein, dystrophin, in the muscle cells. This means that patients suffering from this condition do not produce the dystrophin protein or produce a non-functional dystrophin. As boys, contrary to girls, only have one X chromosome, and thus one single copy of dystrophin gene, they have a much higher probability of suffering from Duchenne muscular dystrophy. Duchenne muscular dystrophy is a debilitating and life-threatening disease.

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

At the time of designation Duchenne muscular dystrophy affected approximately 0.5 in 10,000 persons in 10,000 people in the European Union (EU)*. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 23,000 people.

* 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 Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What treatments are available?

At the time of submission of the application for orphan designation, no satisfactory method had been authorised in the European Union for treatment of the condition. Treatment of patients with Duchenne muscular dystrophy primarily involves physiotherapy as supportive treatments.

How is this medicine expected to work?

Utropin is a protein that has similar structure and function to that of dystrophin. It is thought that utrophin may replace the function of dystrophin that is lacking or non-functional in patients with Duchenne muscular dystrophy. The mechanism of action of 2-(4-(diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine was not fully demonstrated at the time of designation, but it is expected that this medicinal product increases the levels of utrophin and by doing that, decreases the symptoms of Duchenne muscular dystrophy.

What is the stage of development of this medicine?

The evaluation of the effects of 2-(4-(diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine in experimental models is ongoing.
At the time of submission of the application for orphan designation, no clinical trials in patients with Duchenne muscular dystrophy were initiated.
2-(4-(diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine was not authorised anywhere worldwide for Duchenne muscular dystrophy or designated as orphan medicinal product elsewhere for this condition, at the time of submission.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 15 June 2006 a positive opinion recommending the grant of the above-mentioned 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 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

Product details for <p>2-(4-(Diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine</p>
Active substance2-(4-(Diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine
Medicine Name
Disease/conditionTreatment of Duchenne muscular dystrophy
Date of decision25/07/2006
OutcomeWithdrawn
Orphan decision numberEU/3/06/385

Review of designation

Sponsor’s contact details:

VASTox Plc
91 Milton Park, Abingdon
Oxfordshire, OX14 4RY
United Kingdom
Telephone: +44 12 35 44 39 40
Telefax: +44 12 35 44 39 99

Patients’ associations contact points:

Association Française contre les Myopathies
1 Rue de l'Internationale, BP 59
91002 Evry Cedex
France
Telephone: + 33 1 69 47 28 28 / 0810811088
Telefax: +33 1 60 77 12 16

Action Duchenne (formerly PPUK)
41 West Street
London
E11 4JL
United Kingdom
Telephone: +44 20 85 56 99 55
E-mail: info@ppuk.org

ASEM Madrid - Asociación Española contra las Enfermedades Neuromusculares
c/ Fco. Navacerrada, 12, bajo izq
28028
Madrid
Spain
Telephone: +34 913 613 895
E-mail: info@asemmadrid.org