Overview

On 19 November 2018, orphan designation (EU/3/18/2083) was granted by the European Commission to MaaT Pharma, France, for allogeneic faecal microbiota, pooled for the treatment of graft-versus-host disease.

Graft-versus-host disease is a complication that can occur in patients who have had a transplant. In this disease, the transplanted cells recognise the patient’s body as ‘foreign’ and attack the patient’s organs, such as the stomach, gut, skin and liver, leading to organ damage. The disease may occur shortly after transplantation or later on, in which case more organs can be affected.

Graft-versus-host disease is a serious and life-threatening disease with a high mortality rate.

At the time of designation, graft-versus-host disease affected approximately 0.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 26,000 people1, 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).

 

1Disclaimer: 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 28), Norway, Iceland and Liechtenstein. This represents a population of 517,400,000 (Eurostat 2018).

At the time of designation, several medicines were authorised in the EU for the treatment of graft-versus-host disease, such as ciclosporin and corticosteroids. Treatment aimed to reduce the activity of transplanted cells involved in graft-versus-host disease, thereby reducing their ability to attack the patient's organs.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with graft-versus-host disease. Published studies showed that the medicine can reduce the effects of graft-versus-host disease when given together with standard treatment, and in patients who no longer respond to authorised treatments.

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

The human gut hosts several types of bacteria and other microorganisms, which together are known as microbiota. The microbiota plays an important role not only in the digestive process, but also in maintaining the correct functioning of the immune system (the body’s natural defences). A disruption of the balance within the microbiota, sometimes caused by medical treatment, can cause uncontrolled responses of the immune system and inflammation throughout the body.

This medicine is made of microbiota from healthy donors. Once it is given to a patient with graft-versus-host disease, the medicine is expected to help re-establish a balanced microbiota in the gut of the patient, reducing inflammation and regulating the immune system. This, in turn, is expected to help protect the patient from the effect of graft-versus-host disease.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with graft-versus-host disease were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for graft-versus-host disease 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 11 October 2018 recommending the granting of this designation.

  • 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/3/18/2083: Public summary of opinion on orphan designation: Allogeneic faecal microbiota, pooled for the treatment of graft-versus-host disease

Key facts

Active substance
Allogeneic faecal microbiota, pooled
Intended use
Treatment of graft-versus-host disease
Orphan designation status
Positive
EU designation number
EU/3/18/2083
Date of designation
Sponsor

MaaT PHARMA
 

 

Review of designation

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

Update history

DateUpdate
September 2022The sponsor's address was updated.

EMA list of opinions on orphan medicinal product designation

EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

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.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

How useful do you find this page?