EU/3/09/625

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Orphan designation

On 29 April 2009, orphan designation (EU/3/09/625) was granted by the European Commission to Acure Pharma AB, Sweden, for guanabenz for the treatment of traumatic spinal cord injury.

What is traumatic spinal cord injury?

Traumatic spinal cord injury is damage to the spinal cord caused by an accident, such as a blow to the back. 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.
The development of traumatic spinal cord injury is divided in two phases, the acute phase and the recovery phase. During the acute phase (lasting for a few weeks after the injury) a process of inflammation starts, in which the damage spreads to the nerve cells surrounding the original site of injury. In the recovery phase, the surviving nerve cells recover part of their function. The improvement usually continues for up to a year, after which the patient’s condition does not improve any further.
Traumatic spinal cord injury is a life-threatening and long-term debilitating disease 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, traumatic spinal cord injury affected approximately 0.6 people in 10,000 per year in the European Union (EU)*. This is equivalent to a total of around 30,000 people per year, which was considered to be below the threshold for orphan designation. This is based on the information provided by the sponsor and 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 27), Norway, Iceland and Liechtenstein. This represents a population of 504,800,000 (Eurostat 2009).

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 injury in some countries in the EU. Methylprednisolone reduces the inflammation and pressure on the spinal cord that can happen after it is damaged. Patients with spinal cord injury can also have surgery to reduce the pressure on the spine.
The sponsor has provided sufficient information to show that guanabenz might be of significant benefit for the patients because it works in a different way to existing 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?

Guanabenz has been available as a medicine to treat high blood pressure since the 1980s. In traumatic spinal cord injury, it is expected to work during the acute phase of the injury by reducing inflammation. It is thought to work by blocking enzymes called cyclo-oxygenase, lipoxygenase and nitric oxide synthase, which produce substances that are involved in the inflammation process. Guanabenz is not expected to work in the recovery phase of the disease because it has no effect on the regeneration of nerve cells.

What is the stage of development of this medicine?

The effects of guanabenz have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials in patients with traumatic spinal cord injury had been started.
At the time of submission, guanabenz was not authorised anywhere in the EU for traumatic 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 4 March 2009 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 Community) 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>Guanabenz</p>
Active substanceGuanabenz
Medicine Name
Disease/conditionTreatment of traumatic spinal cord injury
Date of decision29/04/2009
OutcomePositive
Orphan decision numberEU/3/09/625

Review of designation

Sponsor’s contact details:

Acure Pharma AB
Ulleråkersvägen 38
75643 Uppsala
Sweden
Telephone: +46 18 50 55 00
Telefax: +46 18 51 09 10
E-mail: info@acurepharma.com

Patient 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