This medicine is now known as ibrutinib.
On 26 April 2012, orphan designation (EU/3/12/984) was granted by the European Commission to Nexus Oncology Ltd, United Kingdom, for 1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one for the treatment of chronic lymphocytic leukaemia.
The sponsorship was transferred to Janssen-Cilag International N.V, Belgium, in January 2013.
1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one for treatment of chronic lymphocytic leukaemia has been authorised in the EU as Imbruvica since 21 October 2014.
1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one (ibrutinib)
Treatment of chronic lymphocytic leukaemia
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
During its meeting of 2 to 4 September 2014, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/12/984 for Imbruvica (ibrutinib1) as an orphan medicinal product for the treatment of chronic lymphocytic leukaemia (CLL). The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with CLL. The COMP recommended that the orphan designation of the medicine be maintained2.
1Previously known as 1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H- pyrazolo [3,4-d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one.
2The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.
Documents related to this orphan designation evaluation
EU/3/12/984: Public summary of opinion on orphan designation: 1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one for the treatment of chronic lymphocytic leukaemia (PDF/126.75 KB)
First published: 18/06/2012
Last updated: 10/03/2015
EMA/COMP/219979/2012 Rev. 2
Recommendation for maintenance of orphan designation at the time of marketing authorisation: Imbruvica (ibrutinib) for the treatment of chronic lymphocytic leukaemia (PDF/80.78 KB)
First published: 01/12/2014
Last updated: 01/12/2014
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.
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: