EU/3/18/2059 - orphan designation for treatment of spinocerebellar ataxia

acetylleucine

Overview

On 22 November 2018, orphan designation (EU/3/18/2059) was granted by the European Commission to IntraBio Ltd, United Kingdom, for acetylleucine for the treatment of spinocerebellar ataxia.

The sponsorship was transferred to IntraBio Ireland Ltd, Ireland, in April 2019.

What is spinocerebellar ataxia?

Spinocerebellar ataxia is a group of genetic conditions of the nervous system whereby cells in certain areas of the brain get damaged and die. This leads to progressive problems with movement, coordination and balance (ataxia). Depending on the type of spinocerebellar ataxia, people may develop different signs and symptoms, such as speech and swallowing difficulties, muscle stiffness, weakness in the muscles that control eye movement and cognitive (mental) impairment. Symptoms usually appear during adulthood.

Spinocerebellar ataxia is a long-term debilitating condition involving progressive slowing of gait, often associated with poor coordination of speech, hands and eye movement.

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

At the time of designation, spinocerebellar ataxia 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, no satisfactory methods were authorised in the EU for the treatment of spinocerebellar ataxia. Patients were mainly given supportive treatment including physiotherapy, occupational therapy, and speech therapy aimed at easing the symptoms of the condition.

How is this medicine expected to work?

Acetylleucine has been authorised in some countries for the treatment of vertigo (a spinning sensation that affects balance). The way acetylleucine works in spinocerebellar ataxia is not clear, but it is thought to stabilise the nerve cells responsible for balance and for coordinating movement. This is expected to improve movement control and coordination in patients with 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 spinocerebellar ataxia were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for spinocerebellar ataxia. 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 19 July 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/3/18/2059: Public summary of opinion on orphan designation: Acetylleucine for the treatment of spinocerebellar ataxia

AdoptedReference Number: EMA/530908/2018

Key facts

• Active substance:
  acetylleucine
• Intended use:
  Treatment of spinocerebellar ataxia
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2059
• Date of designation:
  22/11/2018
• Sponsor:

  IntraBio Ireland Ltd; 10 Earlsfort Terrace; Dublin 2; Co. Dublin D02 T380; Ireland; 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.