EU/3/15/1560 - orphan designation for treatment of Duchenne muscular dystrophy
N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide (edasalonexent)
OrphanHuman
Overview
On 9 October 2015, orphan designation (EU/3/15/1560) was granted by the European Commission to FGK Representative Service GmbH, Germany, for N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide for the treatment of Duchenne muscular dystrophy.
This medicine is now known as edasalonexent.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in September 2021 upon request of the Sponsor.
Duchenne muscular dystrophy (DMD) is a genetic disease that gradually causes weakness and atrophy (wasting) of the muscles. It mainly affects boys, and is usually diagnosed before the age of six years. The muscle weakness usually starts in the hips and legs, before affecting the arms, chest and the heart. Patients with DMD lack normal dystrophin, a protein found in muscles. Because this protein helps to protect muscles from injury as muscles contract and relax, in patients with DMD the muscles become weaker and eventually stop working.
DMD causes long-term disability and is life threatening because of its effects on the heart and the respiratory muscles (muscles that are used to breathe). The disease usually leads to death in adolescence or early adulthood.
At the time of designation, DMD affected approximately 0.5 in 10,000 people in the European Union (EU). This was equivalent to a total of 26,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 28), Norway, Iceland and Liechtenstein. This represents a population of 512,900,000 (Eurostat 2015).
At the time of designation, Translarna (ataluren) was authorised in the EU to treat patients with DMD who have a specific type of mutation (change) called a nonsense mutation in their dystrophin gene. Patients also received supportive treatment such as physiotherapy.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with DMD; early studies in experimental models indicate that the medicine would not be limited to patients with a specific mutation and would therefore target a wider patient population compared with authorised treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
It is thought that inflammation, particularly inflammation caused by the activity of a protein called 'NF??B', leads to muscle damage and prevention of muscle regeneration seen in patients with DMD. This medicine is expected to work by reducing NF-?B activity. This is expected to reduce the muscle damage seen in DMD and enable muscle regeneration.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with DMD had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for DMD. Orphan designation of the medicine has been granted in the United States for the treatment of DMD.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 September 2015 recommending the granting of this 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 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
- Active substance
- N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide (edasalonexent)
- Intended use
- Treatment of Duchenne muscular dystrophy
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/15/1560
- Date of designation
- Sponsor
FGK Representative Service GmbH
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
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: