EU/3/19/2171 - orphan designation for treatment of centronuclear myopathies

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)

Overview

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.

What are 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.

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

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).

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*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).

What treatments are available?

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. 

How is this medicine expected to work?

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.

What is the stage of development of this medicine?

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.
 

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 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.

EU/3/19/2171: Public summary of opinion on orphan designation: 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' for the treatment of centronuclear myopathies

AdoptedReference Number: EMADOC-628903358-1035

Key facts

• Active substance:
  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)
• Intended use:
  Treatment of centronuclear myopathies
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/19/2171
• Date of designation:
  28/06/2019
• Sponsor:

  Dynacure S.A.S.; 300 Boulevard Sebastien Brant; 67400 Illkirch Graffenstaden; France; Tel. [phone]; E-mail: [e-mail]