EU/3/20/2261: Orphan designation for the treatment of chronic myeloid leukaemia



On 24 March 2020, orphan designation EU/3/20/2261 was granted by the European Commission to Novartis Europharm Limited, Ireland, for asciminib for the treatment of chronic myeloid leukaemia.

The medicinal product has been authorised in the EU as Scemblix since 25 August 2022.

Key facts

Active substance
Intended use
Treatment of chronic myeloid leukaemia
Orphan designation status
EU designation number
Date of designation

Novartis Europharm Limited
Vista Building
Elm Park       
Merrion Road
Dublin 4 D04 A9N6
Tel. +41 613 241 111

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Scemblix at the time of marketing authorisation, and confirmed that the orphan designation should be maintained. More information is available in the PDF icon orphan medicine assessment report

Documents related to this orphan designation evaluation

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:

Related content

How useful was this page?

Add your rating
1 rating