EU/3/08/532

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Orphan designation

On 1 April 2008, orphan designation (EU/3/08/532) was granted by the European Commission to Sygnis Bioscience GmbH & Co. KG, Germany, for filgrastim for the treatment of amyotrophic lateral sclerosis.

What is amyotrophic lateral sclerosis?

Amyotrophic lateral sclerosis is a progressive disease of the nervous system. Amyotrophic lateral sclerosis occurs when specific nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate. The loss of these so-called motor neurons causes the muscles under their control to weaken and waste away, leading to paralysis. Amyotrophic lateral sclerosis varies from patient to patient, depending on which muscles weaken first. Symptoms may include tripping and falling, loss of motor control in hands and arms, difficulty in speaking, swallowing and/or breathing, persistent fatigue, and twitching and cramping. Amyotrophic lateral sclerosis strikes in mid-life. Men are about one-and-a-half times more likely to have the disease as women. Amyotrophic lateral sclerosis is chronically debilitating and life-threatening.

What are the methods of treatment available?

A medicinal product called riluzole was authorised for the condition in the Community at the time of submission of the application for orphan drug designation. Filgrastim might be of potential significant benefit for the treatment of amyotrophic lateral sclerosis. The benefit will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

What is the estimated number of patients affected by amyotrophic lateral sclerosis*?

Based on the information provided by the sponsor and previous knowledge of the Committee, amyotrophic lateral sclerosis was considered to affect approximately 0.7 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 35,000 persons.

* Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 27), Norway, Iceland and Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

How is this medicinal product expected to act?

Granulocyte-colony stimulating factor acts as a growth factor in the body. Growth factors stimulate cells to proliferate or to gain specific functions. Filgrastim stimulates building and function of white blood cells called granulocytes. Granulocytes are important for the immune system as they are the cells responsible for destroying bacteria and external agents that enter the body.

The mechanism of action of filgrastim in amyotrophic lateral sclerosis is not fully understood, but it is thought that it would protect nerve cells and prevent nerve cells from deteriorating as seen in amyotrophic lateral sclerosis patients.

What is the stage of development of this medicinal product?

The evaluation of the effects of filgrastim in experimental models is ongoing.

At the time of submission of the application for orphan designation, no clinical trials in patients with amyotrophic lateral sclerosis were initiated.

Filgrastim was not authorised anywhere worldwide for treatment of amyotrophic lateral sclerosis or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 6 February 2008 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition, 
  • the existence or not of alternative methods of diagnosis, prevention or treatment and 
  • either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.

Key facts

Product details for <p>Filgrastim</p>
Active substanceFilgrastim
Medicine Name
Disease/conditionTreatment of amyotrophic lateral sclerosis
Date of decision01/04/2008
OutcomePositive
Orphan decision numberEU/3/08/532

Review of designation

Sponsor’s contact details:

Sygnis Bioscience GmbH & Co. KG
Im Neuenheimer Feld 515
D-69120 Heidelberg
Germany
Telephone: +49 (0) 6221 454-6
Telefax: + 49 (0) 6221 454 700
E-mail: contact@sygnis.de

Patients’ associations contact points:

Irish Motor Neurone Disease Association
Coleraine House
Coleraine Street
Dublin 7 Dublin
Ireland
Telephone: +353 1 87 30 422
Telefax: +353 1 84 31 409
E-mail: info@imnda.ie

Association pour la Recherche sur la Sclérose Latérale Amyotrophique et autres maladies du motoneurone
75 Avenue de la République
75011 Paris
France
Telephone: +33 1 43 38 99 89
Telefax: +33 1 43 38 31 59
E-mail: ars@wanadoo.fr

Deutsche Muskelschwund - Hilfe e.V.
Alstertor 20,
20095 Hamburg
Germany
Telephone: +49 40 32 32 310
Telefax: +49 40 32 32 31 31
E-mail: info@muskelschwund.de