EU/3/17/1964 - orphan designation for treatment of graft-versus-host disease
Itacitinib
OrphanHuman
Overview
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.
The sponsorship was transferred to Incyte Biosciences Distribution B.V., The Netherlands, in November 2018.
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.
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).
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.
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.
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.
- 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
- Active substance
- Itacitinib
- Intended use
- Treatment of graft-versus-host disease
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1964
- Date of designation
- Sponsor
Incyte Biosciences Distribution B.V.
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:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
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: