EU/3/08/537 - orphan designation for treatment of spina bifida

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Please note that this product was withdrawn from the Community Register of designated orphan products in May 2012 on request of the sponsor.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 17 March 2008, orphan designation (EU/3/08/537) was granted by the European Commission to Choice Pharma Limited, United Kingdom, for autologous urothelial and smooth muscle cells for treatment of spina bifida.

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

What is spina bifida?

The neural tube is the part of the embryo that gives origin to the nervous system. Its shape is similar to a tube and it is completely closed at a certain point of the embryonic development. Spina bifida is caused by the failure of the neural tube to close during the first month of embryonic development. This causes malfunction of some body functions controlled by the nervous system and include seizures, paralysis, limb deformity, bowel, bladder and sexual dysfunction. Spina bifida is chronically debilitating and life-threatening.

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

At the time of designation, spina bifida affected approximately 2.2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of 110,000 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 Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What are the methods of treatment available?

There are no authorised medicinal products for the treatment of spina bifida in the Community. Treatment for the variety of effects of spina bifida may include surgery, medication, and physiotherapy. Surgery to close the newborn's spinal opening is generally performed within 24 hours after birth to minimize the risk of infection and to preserve existing function in the spinal cord.

Bladder function is normally controlled by some nerve roots from the spinal cord. In spina bifida, this control may be defective leading to a dysfunction of the urinary bladder (neurogenic bladder). Surgery such as bladder augmentation is sometimes used for the treatment of neurogenic bladder. A bladder augmentation is a surgical operation where tissue from gastrointestinal tract is used to improve the function of the bladder.

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 spina bifida. 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 medicine 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 spina bifida were initiated.

Autologous urothelial and smooth muscle cells was not authorised anywhere worldwide for treatment of spina bifida 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 6 February 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;
• 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

EU/3/08/537: Public summary of positive opinion for orphan designation of autologous urothelial and smooth muscle cells for the treatment of spina bifida

Adopted Reference Number: EMEA/COMP/49041/2008

Key facts

• Active substance:
  autologous urothelial and smooth muscle cells
• Intended use:
  Treatment of spina bifida
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/08/537
• Date of designation:
  18/03/2008
• 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