EU/3/08/580

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Orphan designation

On 7 November 2008, orphan designation (EU/3/08/580) was granted by the European Commission to Sygnis Bioscience GmbH & Co. KG, Germany, for filgrastim for the treatment of spinal cord injury.

What is spinal cord injury?

The spinal cord can be injured through accidents, such as damage to the back, or by internal causes such as tumours or bleeding within the spine putting pressure on the spinal cord. Injury to the spinal cord can damage and kill the nerve cells that run through the cord and that branch out from it. This can stop the flow of nerve impulses between the brain and the body, resulting in the loss of feeling, paralysis and even death, depending upon the severity of the injury and where it is located.

Spinal cord injury is life-threatening and chronically debilitating because it can cause paralysis of the arms and legs and reduce life expectancy.

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

At the time of designation spinal cord injury affected between 2.2 and 4.2 in 10,000 people in the European Union (EU)*. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 110,000 to 211,000 people.

*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 Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).

What treatments are available?

At the time of submission of the application for orphan drug designation, methylprednisolone (a steroid) was authorised for the treatment of spinal cord in some countries in the Community. Methylprednisolone reduces inflammation and pressure on the spinal cord. At the time of submission of the application for orphan drug designation surgical intervention was often performed to decrease the pressure over the spine (decompression), but its role was controversial.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that filgrastim might be of potential significant benefit for the treatment of spinal cord injury because it has a new mechanism of action. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Filgrastim is a copy of a human protein called granulocyte colony stimulating factor (G-CSF), which may reduce cell death and promote cell division. In the treatment of spinal cord injury, filgrastim is expected to protect nerve cells in the spinal cord from death, thus limiting the effects of the injury.

What is the stage of development of this medicine?

The active ingredient is already authorised in the EU for treatment of other conditions in the field of haematology.

The effects of filgrastim have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with spinal cord injury had been initiated.

ilgrastim was not authorised anywhere worldwide for the treatment of spinal cord 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 a positive opinion on 10 September 2008 recommending the granting of the above-mentioned 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;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Filgrastim</p>
Active substanceFilgrastim
Medicine Name
Disease/conditionTreatment of spinal cord injury
Date of decision06/11/2008
OutcomePositive
Orphan decision numberEU/3/08/580

Review of designation

Sponsor’s contact details:

Sygnis Bioscience GmbH & Co. KG
Im Neuenheimer Feld 515
D-69120 Heidelberg
Germany
Telephone: + 49 62 21 45 46
Telefax: + 49 62 21 45 47 00
E-mail: contact@sygnis.de

Patients’ associations contact points:

AFM : Association Française contre les Myopathies
BP 59
1 Rue de l'Internationale
91002 Evry Cedex
France
Telephone: +33 1 69 47 28 28 / +33 81 08 11 088
Telefax: +33 1 60 77 12 16
E-mail: afm@afm.genethon.fr

ASEM ESPAÑA : Asociación Española contra las Enfermedades Neuromusculares
Gran Via de les Corts Catalanes, 562, pral. 2
08011 Barcelona
Spain
Telephone: +34 93 451 65 44
Telefax: +34 93 45 16 904
E-mail: asemcatalunya@terra.es

ASAMSI : Associazione Studio Atrofie Muscolari Spinali Infantili - ONLUS
Via Laderchi 3
48018 Faenza (RA)
Italy
Telephone: +39 338 99 51 232
Telefax: +39 05 46 27 206
E-mail: info@asamsi.org