EU/3/17/1920 - orphan designation for treatment of West syndrome

Cannabidiol

Overview

On 16 October 2017, orphan designation (EU/3/17/1920) was granted by the European Commission to GW Research Ltd, United Kingdom, for cannabidiol for the treatment of West syndrome.

What is West syndrome?

West syndrome is an epilepsy disorder in which young children have regular seizures (fits) called 'infantile spasms'. The seizures usually start in the first year of life and can affect different parts of the body. Usually when the seizures start occurring, the child's physical and mental development suffer. Children with West syndrome also have a typical abnormal pattern of electrical activity in the brain called 'hypsarrhythmia'. While the disorder resolves in some children, the majority will have varying degrees of long-term developmental problems.

West syndrome is a long-term debilitating and life-threatening disorder because the seizures can cause severe physical and mental problems.

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

At the time of designation, West syndrome affected approximately 0.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 31,000 people¹, 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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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, vigabatrin was authorised in the EU for the treatment of West syndrome; corticotropin (ACTH) or the related medicine tetracosactide, and corticosteroids such as prednisolone were also used. In addition, epilepsy medicines such as zonisamide and valproic acid might be given.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with West syndrome because early results showed a reduction in seizures in patients whose condition had not improved with epilepsy medicines.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?

Cannabidiol is a substance extracted from the Cannabis sativa plant. Although the way this medicine works is not fully understood, cannabidiol is thought to act on targets that play a role in the movement of calcium in the cells, which in turn is important for the transmission of electrical signals in some nerve cells. As seizures are caused by excessive electrical activity in the brain, altering the movement of calcium is expected to reduce or prevent the seizures in West syndrome. Cannabidiol is also thought to act on adenosine, a chemical messenger in the brain that plays an important role in suppressing seizures.

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 cannabidiol in patients with West syndrome were ongoing.

At the time of submission, cannabidiol was not authorised anywhere in the EU for West syndrome. Orphan designation of the medicine had been granted in the United States for treatment of infantile spasms and in both the EU and the United States for various other epilepsy syndromes such as Dravet syndrome and Lennox-Gastaut syndrome.

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/3/17/1920: Public summary of opinion on orphan designation : Cannabidiol for the treatment of West syndrome

Reference Number: EMA/625004/2017

Key facts

• Active substance:
  Cannabidiol
• Intended use:
  Treatment of West syndrome
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/17/1920
• Date of designation:
  16/10/2017
• Sponsor:

  Jazz Pharmaceuticals Ireland Ltd; Fifth Floor, Waterloo Exchange; Waterloo Road; Dublin 4; Ireland D04 E5W7; 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.