EU/3/18/2046 - orphan designation for treatment of Becker muscular dystrophy

givinostat

Overview

On 31 July 2018, orphan designation (EU/3/18/2046) was granted by the European Commission to Italfarmaco S.p.A., Italy, for givinostat for the treatment of Becker muscular dystrophy.

What is Becker muscular dystrophy?

Becker muscular dystrophy is an inherited disease that gradually causes the muscles to become weaker. The muscle weakness usually starts in the hips and legs and may later on involve the chest and the heart. The disease mainly affects boys, and usually starts between the ages of 10 and 15 years.

Becker muscular dystrophy is caused by abnormalities in the gene responsible for the production of dystrophin, a protein that forms an important component of muscle fibres. As the patients do not have enough of the functional protein, muscle fibres gradually break down leading to muscle weakness.

Becker muscular dystrophy causes long-term disability and is life-threatening because of its effects on the heart and the muscles that are used to breathe.

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

At the time of designation, Becker muscular dystrophy affected approximately 0.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 10,000 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 517,400,000 (Eurostat 2018).

What treatments are available?

At the time of designation, there were no treatments authorised in the EU for Becker muscular dystrophy. Patients received supportive treatment to relieve symptoms and improve the patient’s general condition. In addition, corticosteroids were used in an attempt to improve symptoms, although they were not authorised for use in this disease.

How is this medicine expected to work?

Givinostat is an ‘HDAC inhibitor’ medicine. This means that it blocks enzymes called histone deacetylases (HDACs), which are involved in turning genes ‘on’ and ‘off’ within cells. The activity of HDACs is increased in patients with Becker muscular dystrophy. By blocking HDAC enzymes, givinostat ‘switches on’ a gene that increases the amount of a protein called follistatin in muscle cells. Follistatin is expected to increase muscle mass and prevent muscle degeneration and thereby improve the symptoms of Becker muscular dystrophy.

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, a clinical trial with the medicine in patients with Becker muscular dystrophy was ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for Becker muscular dystrophy. Orphan designation of the medicine had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 21 June 2018 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/3/18/2046: Public summary of opinion on orphan designation: Givinostat for the treatment of Becker muscular dystrophy

Adopted Reference Number: EMA/465065/2018

Key facts

• Active substance:
  givinostat
• Intended use:
  Treatment of Becker muscular dystrophy
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2046
• Date of designation:
  31/07/2018
• Sponsor:

  Italfarmaco S.p.A.; Viale Fulvio Testi 330; 20126 Milan; MI; Italy; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.