EU/3/13/1132: Orphan designation for the treatment of malignant mesothelioma

N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride (defactinib)


On 7 June 2013, orphan designation (EU/3/13/1132) was granted by the European Commission to TMC Pharma Services Ltd, United Kingdom, for N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride for the treatment of malignant mesothelioma.

The sponsorship was transferred to TMC Pharma (EU) Limited, Ireland, in July 2019.

The sponsor’s address was updated in March 2021.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in February 2022 on request of the Sponsor.

Key facts

Active substance
N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride (defactinib)
Intended use
Treatment of malignant mesothelioma
Orphan designation status
EU designation number
Date of designation

TMC Pharma (EU) Limited 
Durands Court 45
7a Parnell Street
Co. Waterford
X91 P381
Tel. +353 1 901 5595

Review of designation

This medicine is now known as defactinib.

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

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:

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