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Orphan designation

On 17 January 2018, orphan designation (EU/3/17/1964) was granted by the European Commission to Incyte Biosciences UK Ltd, United Kingdom, for itacitinib for the treatment of graft-versus-host disease.

What is 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 a wider range of organs can be involved.

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

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

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 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 28), Norway, Iceland and Liechtenstein. This represents a population of 515,700,000 (Eurostat 2017).

What treatments are available?

At the time of designation, several medicines were authorised in the European Union (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 itacitinib might be of significant benefit for patients with graft-versus-host disease, with early studies showing an improved outcome when it is given together with corticosteroids. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Itacitinib blocks an enzyme known as JAK1, which plays an important role in the inflammation and tissue damage that occur in graft-versus-host disease. By blocking this enzyme, the medicine is expected to help reduce the tissue damage and thereby relieve symptoms of the condition.

What is the stage of development of this medicine?

The effects of itacitinib have been evaluated in experimental models.

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

At the time of submission, itacitinib was not authorised anywhere in the EU for graft-versus-host disease. Orphan designation had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 December 2017 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>Itacitinib</p>
Active substanceItacitinib
Medicine Name
Disease/conditionTreatment of graft-versus-host disease
Date of decision17/01/2018
Orphan decision numberEU/3/17/1964

Review of designation

Sponsor’s contact details

Incyte Biosciences UK Ltd
Riverbridge House
Guildford Road
Surrey KT22 9AD
United Kingdom
Tel. +44 (0)1372 365900

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;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.