EU/3/20/2311 - orphan designation for treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes
Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in February 2024 on request of the Sponsor.
On 21 August 2020, orphan designation EU/3/20/2311 was granted by the European Commission to Scendea (NL) B.V., Netherlands, for sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate (also known as REN001) for the treatment of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS).
Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS) is an inherited disease caused by genetic abnormalities in the mitochondria, the energy-producing components within cells. The disease causes reduced energy production in the cells and can lead to symptoms such as muscle weakness and pain, headaches, loss of appetite, vomiting and seizures (fits) usually in childhood. By the age of 40, most patients experience stroke-like episodes that can lead to vision loss, problems with movement and loss of intellectual function.
The condition is life threatening and debilitating in the long term due to symptoms such as seizures, stroke-like episodes, and the recurring headaches, vomiting and weakness.
At the time of designation, MELAS affected less than 0.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 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).
*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).
No satisfactory methods of treatment were authorised in the EU at the time of orphan designation. Patients received supportive therapy to deal with the symptoms.
The medicine is expected to activate a protein known as PGC-1 alpha, which is involved in the production of mitochondria. By activating this protein, it will be able to increase the number of mitochondria in the cells. As a result, it is expected to improve energy production within the cells and improve symptoms of the disease.
The effects of this medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials in patients with MELAS were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of MELAS or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 16 July 2020, 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
- Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate
- Intended use
- Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/20/2311
- Date of designation
- Sponsor
Scendea (NL) B.V.
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: