EU/3/07/458

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Orphan designation

On 10 July 2007, orphan designation (EU/3/07/458) was granted by the European Commission to Dr Ulrich Granzer, Germany, for fampridine for the treatment of Guillain-Barré syndrome.

What is Guillain-Barré syndrome?

Guillain-Barré syndrome is an inflammation of the peripheral nervous system (i.e., nerves outside the brain or the spinal cord), caused by activation of the immune system and production of specific antibodies. It is characterized by destruction or loss of the myelin sheath that surrounds most nerve cells. Numbness in the toes is often the first neurological symptom, followed within hours or days by weakness of the legs or limbs. The condition is chronically debilitating and life-threatening.

What are the methods of treatment available?

Plasmapheresis (taking the blood out of the patient, filtering it and re-injecting it into the veins) is used to eliminate the antibodies that cause the disease. Several drugs were authorised for the condition in some countries in the Community at the time of submission of the application for orphan drug designation. Fampridine may be of potential significant benefit in patients affected by Guillain-Barré syndrome. The assumption 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 the condition*?

According to the information provided by the sponsor, Guillain-Barré syndrome was considered to affect between 9,200 and 18,000 persons in the European Union.

* 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 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). 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?

The action of fampridine is believed to be through an improvement of the function of the affected nerve cells. The drug blocks specific proteins that transport potassium from outside to inside the cell which would hypothetically improve the conduction of the electrical impulse in the damaged nerve cells.

What is the stage of development of this medicinal product? 

The effects of fampridine were evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials in patients with Guillain-Barré syndrome were ongoing.

Fampridine was not authorised anywhere worldwide for the treatment of Guillain-Barré syndrome 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 31 May 2007 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>Fampridine</p>
Active substanceFampridine
Medicine Name
Disease/conditionTreatment of Guillain-Barré syndrome
Date of decision10/07/2007
OutcomePositive
Orphan decision numberEU/3/07/458

Review of designation

Sponsor’s contact details:

Dr Ulrich Granzer
Zielstattstrasse 44
81379 München
Germany
Telephone: +49 89 780 68 98 20
Telefax: +49 89 780 68 98 15
E-mail: granzer@granzer.biz

Patients’ association contact point:

Guillain-Barre Syndrome Ireland
Guillain-Barre Syndrome
Oakfield, Garryvadden
Blackwater
Enniscorthy, Co. Wexford
Ireland
Telephone: +44 800 374 803
Telefax: +44 1529 304 615

Fondazione Internazionale Sindrome di Guillain-Barré
Via Castagna 72
82034 San Lorenzo Maggiore (BN)
Italy
Telephone: +39 0824 813814
Telefax: +39 0824 813814
E-mail: info@istitutomentecorpo.it

AFL+: Association Française du Lupus et autres maladies auto-immunes
Escalier 50
4 Rue de Crimée
75019 Paris
France
Telephone: +33 1 42 02 23 20
Telefax: +33 1 42 02 23 20
E-mail: lupus.plus@free.fr