On 26 November 2010, orphan designation (EU/3/10/810) was granted by the European Commission to Dr Ulrich Granzer, Germany, for N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate for the treatment of post-essential thrombocythaemia myelofibrosis.

The sponsorship was transferred to Sanofi Aventis, France, in February 2011. In October 2012, Sanofi Aventis changed name to Sanofi-Aventis Groupe.

The sponsorship was transferred to SynteractHCR Deutschland GmbH, Germany, in May 2017.

The sponsorship was transferred to Celgene Europe B.V., the Netherlands, in June 2018.

Key facts

Active substance
N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate
Disease / condition
Treatment of post-essential thrombocythaemia myelofibrosis
Date of first decision
EU designation number

Review of designation

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

Sponsor's contact details

Celgene Europe B.V.
Winthontlaan 6 N
3526 KV Utrecht
The Netherlands
Tel. +31 302844547

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.

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