EU/3/08/574 - orphan designation for treatment of spinal cord injury

autologous urothelial and smooth muscle cells

Overview

Please note that this product was withdrawn from the Community Register of designated orphan products in May 2012 on request of the sponsor.

On 7 November 2008, orphan designation (EU/3/08/574) was granted by the European Commission to Choice Pharma Limited, United Kingdom, for autologous urothelial and smooth muscle cells, for the treatment of spinal cord injury.

The sponsorship was transferred to European Medical Advisory Services Limited, United Kingdom, in May 2011.

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 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. When the injury is to the lower part of the spinal cord, the bladder can be affected.

Spinal cord injury is life-threatening and chronically debilitating. 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). Kidney and urinary tract infections are more common in patients whose bladder has been affected by the spinal cord injury.

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

At the time of designation, spinal cord injury affected between 2.2 to 4.2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of between 111,000 to 211,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000. 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 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 was authorised for the treatment of spinal cord injury in some member states. If the bladder is affected by the spinal cord injury and becomes non-functional, a type of surgery called 'bladder augmentation' is sometimes used. This usually involves implanting tissue from the gut into the bladder to improve the function of it.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that the autologous urothelial and smooth muscle cells may be of significant benefit for the treatment of non-functional bladder in spinal cord injury. 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 medice expected to work?

Autologous urothelial and smooth muscle cells are obtained from the patient's (autologous) bladder. The cells are treated in a manufacturing process to create an artificial bladder around a synthetic scaffold. This is intended to substitute the non functional bladder and to avoid the need to use gastrointestinal segments during surgical augmentation of the bladder. It is thought that the cells will support and improve the function of the bladder.

What is the stage of development of this medicine?

The effects of autologous urothelial and smooth muscle cells were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with spinal cord injury had not been initiated.

Autologous urothelial and smooth muscle cells was not authorised anywhere worldwide for treatment of spinal cord injury or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 September 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.

EU/3/08/574: Public summary of positive opinion for orphan designation of autologous urothelial and smooth muscle cells for the treatment of spinal cord injury

Adopted Reference Number: EMEA/COMP/480031/2008 Rev. 1

Key facts

• Active substance:
  autologous urothelial and smooth muscle cells
• Intended use:
  Treatment of spinal cord injury
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/08/574
• Date of designation:
  08/11/2008
• Sponsor:

  European Medical Advisory Services Limited; 71 Knowl Piece Wilbury Way Hitchin Hertfordshire SG4 0TY United Kingdom; Telephone: [phone]; Telefax: [phone]; Contact: www.emas-medical.com