EU/3/17/1932 - orphan designation for treatment of Prader-Willi syndrome

synthetic cyclic 8 amino acid analogue of human unacylated ghrelin

Overview

On 16 October 2017, orphan designation (EU/3/17/1932) was granted by the European Commission to Alizé Pharma, France, for synthetic cyclic 8 amino acid analogue of human unacylated ghrelin (also known as AZP-531) for the treatment of Prader-Willi syndrome.

The sponsorship was transferred to Millendo Therapeutics SAS, France, in July 2018.

What is Prader-Willi syndrome?

Prader-Willi syndrome is an inherited condition caused by defects in specific regions of chromosome 15. This causes a wide range of symptoms, some of which can appear at birth, such as feeding problems, small size and reduced muscle strength. During childhood further symptoms develop, including increased appetite leading to constant eating and severe obesity, short stature, incomplete sexual development, learning difficulties and behavioural problems, such as aggression and stubbornness.

Prader-Willi syndrome is a life-long debilitating and life-threatening disease because of its serious symptoms, particularly learning difficulties, behavioural problems and obesity.

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

At the time of designation, Prader-Willi syndrome affected approximately 0.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 15,000 people¹, 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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¹Disclaimer: 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, growth hormone was authorised in the EU for treating Prader-Willi syndrome. In addition, symptoms were treated or managed in various ways, including supervised access to food to prevent obesity.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with Prader-Willi syndrome. Early studies show that it may reduce the excessive food intake – benefit not seen with current therapy. 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?

The medicine is thought to work by counteracting the activity of a hormone called ghrelin, which is produced mainly by the stomach. Ghrelin has an effect on appetite by regulating how fats and glucose (sugar) are used by the body. By counteracting the activity of ghrelin, this medicine is expected to reduce the patients' appetite and thus their food intake.

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, clinical trials with the medicine in patients with Prader-Willi syndrome were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for Prader-Willi syndrome 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 7 September 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/3/17/1932: Public summary of opinion on orphan designation : Synthetic cyclic 8 amino acid analogue of human unacylated ghrelin for the treatment of Prader-Willi syndrome

Reference Number: EMA/COMP/632552/2017

Key facts

• Active substance:
  synthetic cyclic 8 amino acid analogue of human unacylated ghrelin
• Intended use:
  Treatment of Prader-Willi syndrome
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/17/1932
• Date of designation:
  16/10/2017
• Sponsor:

  Millendo Therapeutics SAS; 15 Chemin du Saquin; Espace Européen; 69130 Écully; France; Tel. [phone]; 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.