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Orphan designation

On 26 August 2005, orphan designation (EU/3/05/303) was granted by the European Commission to Cellerix S.L., Spain, for human autologous mesenchymal adult stem cells extracted from adipose tissue for the treatment of anal fistula.

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

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

What is anal fistula?

An anal fistula is an abnormal passageway that develops between the rectum (the lower part of the large intestine that stores faeces) and the outside of the body. This results in abnormal discharge of faeces through an opening other than the anus.

Anal fistulae are usually caused by an infection or an abscess (collection of pus) in the anus. They can also result from other diseases that cause long-term inflammation of the bowel. Patients with an anal fistula have constant pain, sometimes accompanied by swelling and irritation of skin around the anus, leakage of pus, diarrhoea and fever.

Anal fistula is a long-term debilitating disease because it can lead to incontinence (a lack of control over the opening of the bowels) and sepsis (blood infection).

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

At the time of designation, anal fistula affected approximately 1.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 84,000 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 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 466,600,000 (Eurostat 2005).

What treatments are available?

At the time of submission of the application for orphan designation, no satisfactory method had been authorised in the European Union for treatment of the condition. Certain anal fistulas heal spontaneously, however fistula surgery is usually necessary. Fistula surgery involves cutting a small portion of the anal sphincter (a ring of muscle fibers controlling the opening and closing of the anus) to open the tunnel, joining the external and internal opening and converting the tunnel into a groove that will then heal from within outward.

How is this medicine expected to work?

This medicine is made up of ‘mesenchymal stem cells’ that are extracted from the patient’s own adipose (fat) tissue. To make this medicine, the cells are isolated and cultivated using a technique called ex vivo expansion to increase their number. When these cells are injected into the walls of the fistula, they are expected to send signals that reduce the activity of the immune system and inflammation. Once the inflammation in the fistula has subsided, new tissue can start to grow, helping the fistula to heal.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of human autologous mesenchymal adult stem cells extracted from adipose tissue in experimental models was ongoing.
At the time of submission of the application for orphan designation, clinical trials in patients with anal fistula were ongoing.

Human autologous mesenchymal adult stem cells extracted from adipose tissue was not authorised anywhere worldwide for treatment of anal fistula 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 13 July 2005 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>Human autologous mesenchymal adult stem cells extracted from adipose tissue</p>
Active substanceHuman autologous mesenchymal adult stem cells extracted from adipose tissue
Medicine Name
Disease/conditionTreatment of anal fistula
Date of decision26/08/2005
Orphan decision numberEU/3/05/303

Review of designation

Sponsor’s contact details

TiGenix S.A.U.
Marconi 1, Parque Tecnológico de Madrid
Tres Cantos
28760 Madrid
Tel. +34 91 804 92 64
Fax +34 91 804 92 63

Patients’ associations

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.