On 28 June 2019, orphan designation EU/3/19/2171 was granted by the European Commission to Dynacure S.A.S., France, for 5'-cEtG-sp-cEt5MeU-sp-cEt5MeU-sp-dT-sp-dA-sp-dT-sp-dT-sp-dA-sp-dT-sp-dA-sp-dG-sp-dG-sp-dG-sp-cEt5MeC-sp-cEt5MeU-sp-cEt5MeU-3' (also known as DYN101) for the treatment of centronuclear myopathies.
Centronuclear myopathies are a group of inherited muscle-wasting diseases caused by mutations (changes) in genes involved in the working of muscles. Symptoms include muscle weakness and wasting, which can lead to difficulty walking and eating. Some people with centronuclear myopathy may also have breathing problems because of weakness in the muscles needed for breathing.
Centronuclear myopathies are long-term debilitating conditions because of problems caused by muscle weakness throughout the body.
At the time of designation, centronuclear myopathies affected less than 0.1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 5,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based 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 518,400,000 (Eurostat 2019).
At the time of submission, no satisfactory methods were authorised in the EU for the treatment of centronuclear myopathies. Patients received treatment to relieve their symptoms such as physiotherapy, and support for mobility, nutrition and breathing.
It is thought that centronuclear myopathies may be caused by the hyperactivity of a protein involved in muscle development called DNM2. The medicine is an anti-sense oligonucleotide (a type of genetic material) that attaches to genetic material of cells responsible for producing DNM2. This is expected to reduce production of this protein, thus relieving the symptoms of the disease.
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 centronuclear myopathies had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of centronuclear myopathies 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 23 May 2019, recommending the granting of this designation.
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.
Dynacure S.A.S.
300 Boulevard Sebastien Brant
67400 Illkirch Graffenstaden
France
Tel. +33 368 854486
E-mail: info@dynacure.com
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: