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Orphan designation

On 1 October 2010, orphan designation (EU/3/10/791) was granted by the European Commission to NeuroVive Pharmaceutical AB, Sweden, for ciclosporin for the treatment of moderate and severe closed traumatic brain injury.

What is closed traumatic brain injury?

Closed traumatic brain injury is brain damage caused by a head injury (such as a blow to the head in a traffic accident or a fall). ‘Closed’ means that the skull remains intact. The initial injury to the head and brain usually goes on to cause ‘secondary’ problems, most frequently due to inflammation and swelling of the brain tissue increasing the pressure within the skull. Traumatic brain injury is classified as mild, moderate or severe according to the patient’s symptoms: patients with moderate injury are lethargic (lacking in energy) or stuporous (unaware of their surroundings), and those with severe injury are comatose (unconscious). People with moderate or severe traumatic brain injury need to be admitted to hospital for observation and examination, in case the condition gets worse.

Moderate and severe traumatic brain injuries are long-term debilitating and life threatening because they may lead to permanent disability and death.

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

At the time of designation, moderate and severe closed traumatic brain injury affected less than 4 people in 10,000 per year in the European Union (EU). This was equivalent to a total of fewer than 203,000 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 the 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. At the time of designation, this represented a population of 506,300,000 (Eurostat 2010).

What treatments are available?

At the time of designation, various methods were used to reduce the pressure within the skull in patients with moderate or severe closed traumatic brain injury, including medicines such as mannitol (a diuretic) and surgery

The sponsor has provided sufficient information to show that ciclosporin might be of significant benefit for patients with moderate or severe closed traumatic brain injury because this medicine is a new formulation of ciclosporin and early studies indicate that it might have a positive effect in protecting injured brain cells. 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?

Ciclosporin is an immunosuppressant medicine (a medicine that reduces the activity of the immune system) that has been used since the early 1980s. This medicine will be available as a solution for injection and is expected to be able to reach the injured brain tissue because the ‘blood-brain barrier’ that separates the bloodstream from the brain is usually damaged in patients with moderate and severe brain injuries. Once ciclosporin has reached the nerve cells in the brain, it is expected to act within the cells to protect their energy production, therefore reducing the loss of brain cells.

What is the stage of development of this medicine?

The effects of ciclosporin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with this formulation of ciclosporin in patients with moderate or severe closed traumatic brain injury had been planned.

At the time of submission, this formulation of ciclosporin was not authorised anywhere in the EU for the treatment of moderate and severe closed traumatic brain injury or designated as an 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 8 July 2010 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 EU) 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>Ciclosporin</p>
Active substanceCiclosporin
Medicine Name
Disease/conditionTreatment of moderate and severe closed traumatic brain injury
Date of decision01/10/2010
Orphan decision numberEU/3/10/791

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details:

NeuroVive Pharmaceutical AB
Medicon Village
Scheelegatan 2
SE-223 81 Lund
Tel. +46 275 62 20
Fax +46 888 83 48

Patients' organisations:

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.