EU/3/17/1903 - orphan designation for treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

Odiparcil

Overview

On 23 August 2017, orphan designation (EU/3/17/1903) was granted by the European Commission to Inventiva, France, for odiparcil for the treatment of mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome).

What is mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome)?

Mucopolysaccharidosis type VI (also known as Maroteaux-Lamy syndrome) is an inherited disease that is caused by the lack of an enzyme called arylsulfatase B (ARSB). This enzyme is needed to break down substances in the body called glycosaminoglycans (GAGs). If the enzyme is not present, GAGs cannot be broken down and they build up in the cells and damage them. This causes a wide range of symptoms, the most noticeable being a short body, a large head, difficulty moving about, clouding of the eyes and hearing loss. The disease is usually diagnosed in children between one and five years of age.

Mucopolysaccharidosis VI is a long-lasting and life-threatening disease because of the damage to various parts of the body, particularly spine, heart and lungs.

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

At the time of designation, mucopolysaccharidosis VI affected less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 52,000 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).

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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 515,700,000 (Eurostat 2017).

What treatments are available?

At the time of designation, galsulfase was authorised in the EU for the treatment of mucopolysaccharidosis VI. This is an 'enzyme replacement therapy' which works by providing patients with the enzyme they are lacking. Some patients underwent transplantation to receive haematopoietic (blood) stem cells that are able to produce the missing enzyme.

The sponsor has provided sufficient information to show that odiparcil might be of significant benefit for patients with mucopolysaccharidosis VI. Laboratory studies indicate that odiparcil may improve symptoms not managed by the currently authorised medicine, such as clouding of the eyes. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Odiparcil works by attaching to an enzyme called galactosyl transferase I, which is responsible for starting production of GAGs. By attaching to this enzyme, odiparcil is expected to reduce production of GAGs, thereby relieving the symptoms of the disease.

What is the stage of development of this medicine?

The effects of odiparcil have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with odiparcil in patients with mucopolysaccharidosis VI were ongoing.

At the time of submission, odiparcil was not authorised anywhere in the EU for mucopolysaccharidosis VI or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 July 2017 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/3/17/1903: Public summary of opinion on orphan designation: Odiparcil for the treatment of mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome)

AdoptedReference Number: EMA/476085/2017

Key facts

• Active substance:
  Odiparcil
• Medicine name:
  -
• Intended use:
  Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/17/1903
• Date of designation:
  23/08/2017
• Sponsor:

  Inventiva; 50 rue de Dijon; 21121 Daix; France; Tel. + 33 3 80 44 75 00; 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.