EU/3/08/605

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Orphan designation

On 19 January 2009, orphan designation (EU/3/08/605) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for recombinant human monoclonal antibody to human Nogo-A protein of the IgG4/k class for the treatment of spinal cord injury.

What is acute 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 the nerves 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.

The complications include paralysis of the legs with or without paralysis of the arms, breathing problems, blood clots in the veins and the lungs, and repeated infections of the lungs, the airways, the kidneys and the urinary tract (the structures that carry urine). Spinal cord injury is life-threatening and chronically debilitating.

What is the estimated number of patients affected by the acute spinal cord injury*?

At the time of designation spinal cord injury affected approximately 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 between 12,000 to 22,000 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?

Surgical intervention is often performed to decrease the pressure over the spine (decompression), but its role is controversial. Methylprednisolone sodium succinate is approved in many European countries for the treatment of spinal cord injury.

The sponsor has provided sufficient information to show that recombinant human monoclonal antibody to human Nogo-A protein of the IgG4/k class for the treatment of acute spinal cord injury might be of significant benefit for the patients because its different mechanism of action is expected to improve the overall outcome in patients. 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?

The product is expected to work by inactivating Nogo-A, which is believed to play role in inhibiting nerve cells from regrowing after they have been damaged. By blocking the activity of Nogo-A, this medicine may allow the nerve cells to repair themselves and regrow their damaged axons (the long processes of nerve cells along which nerve impulses pass), and thus improve the symptoms of spinal cord injury.

What is the stage of development of this medicine?

The effects of the recombinant human monoclonal antibody to human Nogo-A protein of the IgG4/k class have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with acute spinal cord injury were ongoing.

At the time of submission, the recombinant human monoclonal antibody to human Nogo-A protein of the IgG4/k class was not authorised anywhere in the world for acute spinal cord injury or designated as orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 November 2008 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;
  • 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>Recombinant human monoclonal antibody to human Nogo-A protein of the IgG4/kappa class</p>
Active substanceRecombinant human monoclonal antibody to human Nogo-A protein of the IgG4/kappa class
Medicine Name
Disease/conditionTreatment of spinal cord injury
Date of decision18/01/2009
OutcomePositive
Orphan decision numberEU/3/08/605

Review of designation

Sponsor’s contact details:

Novartis Europharm Limited
Wimblehurst Road
Horsham
West Sussex RH12 5AB
United Kingdom
Telephone: +44 1403 323542
Telefax: +44 1403 323259
E-mail: carol.beauchamp@novartis.com

Patients’ associations contact points:

Associazione Luca Coscioni
via di Torre Argentina
76 - 00186 Roma
Italy
Telephone: +39 06 68 97 91
Telefax: +39 06 68 80 53 96

Danish Spinal Cord Injuries Association
Hans Knudsens Plads 1 A
DK 2100 København Ø
Denmark
Telephone: +45 39 29 35 55
Telefax: +45 39 29 39 48
E-mail: info@ryk.dk

I.W.A. – Irish Wheelchair Association
National headquarters
Áras Chúchulainn
Blackheath Drive
Clontarf
Dublin 3
Ireland
Telephone: +353 01 8186 400
Telefax: +33 01 8333 873
E-mail: info@iwa.ie