EU/3/06/369 - orphan designation for treatment of epidermolysis bullosa

bilayer engineered skin composed of keratinocytes from the patient (autologous) and fibroblasts from a donor (allogeneic) embedded in a plasma matrix

Overview

On 22 May 2006, orphan designation (EU/3/06/369) was granted by the European Commission to Cellerix S.L., Spain, for bilayer engineered skin composed of keratinocytes from the patient (autologous) and fibroblasts from a donor (allogeneic) embedded in a plasma matrix for the treatment of epidermolysis bullosa.

The sponsor changed name to Cellerix S.A. in September 2008 and then to TiGenix S.A.U. in February 2013.

The sponsorship was transferred to Biodan Yelah S.L., Spain, in December 2014.

For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.

What is epidermolysis bullosa?

Epidermolysis bullosa describes a group of diseases of the skin and mucous membranes. Patients with epidermolysis bullosa have extremely fragile skin and recurrent blister formation, resulting from minor mechanical friction or injury. Epidermolysis bullosa is caused by defective production of collagen. Collagen is the core substance that makes up the matrix that surrounds cells (extracellular matrix) in the body. It is essential for the support of tissues and gives cells structure from the outside. There are several types of collagen, each with a unique function. Epidermolysis-bullosa patients have a change in their genetic material (the gene coding for collagen VII has a mutation) and cannot produce collagen VII, a type of collagen essential for the connections of cells of the skin.

There are several forms of hereditary epidermolysis bullosa. Some forms of the condition can be present at birth, while acquired forms occur in adults. Dystrophic epidermolysis bullosa present at birth is characterised by blistering, growing skin and mucous lesions. Adults suffering from the condition may have mitten-like deformities of fingers and toes. Epidermolysis bullosa is chronically debilitating and life-threatening.

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

At the time of designation, epidermolysis bullosa affected less than 0.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 23,000 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).

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*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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 468,900,000 (Eurostat 2006).

What treatments are available?

At the time of submission of the application for the orphan drug designation there was no treatment authorised in the European Union. Treatment of epidermolysis bullosa included supportive care and antibiotics for the treatment of infections on the skin and, in some cases, surgery.

How is this medicine expected to work?

The bilayer engineered skin composed of keratinocytes (cells from the upper layer of the skin) from the patient (autologous) and fibroblasts (a fibroblast is a type of cell that synthesises the extracellular matrix that acts as a frame around organs and gives them support) from a donor (allogeneic) embedded in a plasma matrix are expected to provide the missing collagen VII of the skin of patients with epidermolysis bullosa and thus prevent the formation of blisters.

What is the stage of development of this medicine?

The evaluation of the effects of bilayer engineered skin composed of keratinocytes from the patient (autologous) and fibroblasts from a donor (allogeneic) embedded in a plasma matrix in experimental models was ongoing. At the time of submission of the application for orphan designation, clinical trials in patients with epidermolysis bullosa were ongoing.

Bilayer engineered skin composed of keratinocytes from the patient (autologous) and fibroblasts from a donor (allogeneic) embedded in a plasma matrix was not authorised anywhere worldwide for epidermolysis bullosa or designated as an 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 5 April 2006 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/3/06/369: Public summary of positive opinion for orphan designation of bilayer engineered skin composed of keratinocytes from the patient (autologous) and fibroblasts from a donor (allogeneic) embedded in a plasma ma...

Adopted Reference Number: EMEA/COMP/126215/2006 Rev.2

Key facts

• Active substance:
  bilayer engineered skin composed of keratinocytes from the patient (autologous) and fibroblasts from a donor (allogeneic) embedded in a plasma matrix
• Intended use:
  Treatment of epidermolysis bullosa
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/06/369
• Date of designation:
  22/05/2006
• Sponsor:

  Biodan Yelah S.L.; C/Faraday 7; Scientific Park; 28049 Madrid; Spain; Tel. [phone]; Fax [phone]; E-mail: [e-mail]

Review of designation

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