On 29 July 2004, orphan designation (EU/3/04/214) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for midostaurin for the treatment of acute myeloid leukaemia.
Midostaurin for treatment of acute myeloid leukaemia has been authorised in the EU as Rydapt since 18 September 2017.
The sponsorship was transferred to Novartis Europharm Limited, Ireland, in May 2018.
Treatment of acute myeloid leukaemia
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Review of designation
On 25 July 2017, the Committee for Orphan Medicinal Products (COMP) completed a review of the designations EU/3/04/214 and EU/3/10/765 for Rydapt (midostaurin) as an orphan medicinal product for the treatment of acute myeloid leukaemia (AML) and mastocytosis. 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 conditions, 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 AML and mastocytosis. The COMP recommended that the orphan designations of the medicine be maintained.
 The 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 the same therapeutic indication cannot be placed on the market.
Documents related to this orphan designation evaluation
Recommendation for maintenance of orphan designation at the time of marketing authorisation: Rydapt (midostaurin) for the treatment of acute myeloid leukaemia and mastocytosis (PDF/88.34 KB)
First published: 25/10/2017
Last updated: 25/10/2017
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: