EU/3/14/1399

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Orphan designation

On 16 December 2014, orphan designation (EU/3/14/1399) was granted by the European Commission to Treeway B.V., the Netherlands, for edaravone for the treatment of amyotrophic lateral sclerosis.

What is amyotrophic lateral sclerosis?

Amyotrophic lateral sclerosis (ALS) is a progressive disease of the nervous system, where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis. The exact causes are unknown but are believed to include genetic and environmental factors. The symptoms of ALS vary depending on which muscles weaken first, and include loss of balance, loss of control of hand and arm movement, and difficulty speaking, swallowing and breathing. ALS usually starts in mid-life and men are more likely to develop the disease than women.

ALS is a long-term debilitating and life-threatening disease because of the gradual loss of function and its paralysing effect on muscles used for breathing which usually leads to death due to respiratory failure.

What is the estimated number of patients?

At the time of designation, ALS affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 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).


*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 511,100,000 (Eurostat 2014).

What treatments are available?

At the time of designation, riluzole was authorised in the EU to treat ALS. Patients also received supportive treatment to temporarily relieve the symptoms of the disease, such as physiotherapy and speech therapy.

The sponsor has provided sufficient information to show that edaravone might be of significant benefit for patients with the condition based on data from early studies that showed potential favourable effects of using edaravone in combination with riluzole treatment. 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?

Damage to nerve cells in ALS appears to have several causes but there is evidence that it may involve damage to nerves caused by toxic molecules containing oxygen. In some patients this is associated with a defect in the gene responsible for producing the enzyme called superoxide dismutase (SOD), which causes the enzyme to clump together inside nerve cells. This leads to inflammation and kills the affected nerve cells. Edaravone is expected to act as an antioxidant, a molecule that can prevent damage to nerve cells caused by oxygen-containing molecules, and also blocks the clumping together of SOD in the nerves and so reduces inflammation.

Edaravone has been authorised in Japan to reduce nerve damage caused by acute ischaemic stroke (stroke caused by failure of the blood supply to part of the brain).

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of edaravone in experimental models was ongoing.

At the time of submission, no clinical trials with edaravone in patients with ALS were ongoing.

At the time of submission, edaravone was not authorised anywhere in the EU for ALS. Orphan designation had been granted in Japan for ALS.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 November 2014 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.

Key facts

Product details for <p>Edaravone</p>
Active substanceEdaravone
Medicine Name
Disease/conditionTreatment of amyotrophic lateral sclerosis
Date of decision16/12/2014
OutcomePositive
Orphan decision numberEU/3/14/1399

Review of designation

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

Sponsor’s contact details:

Treeway B.V.
Sevillawag 142
3047 AL Rotterdam
The Netherlands
Tel. +31 10 298 88 88
Fax +31 10 298 88 89
E-mail: info@treeway.nl

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.