EU/3/18/2082: Orphan designation for the treatment of Kabuki syndrome

5-{(1R,2R)-2-[(cyclopropylmethyl)amino]cyclopropyl}-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide monohydrochloride


On 19 November 2018, orphan designation (EU/3/18/2082) was granted by the European Commission to Takeda Pharma A/S, Denmark, for 5-{(1R,2R)-2-[(cyclopropylmethyl)amino]cyclopropyl}-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide monohydrochloride (also known as TAK-418) for the treatment of Kabuki syndrome.

The sponsor’s address was updated in August 2020.

Key facts

Active substance
5-{(1R,2R)-2-[(cyclopropylmethyl)amino]cyclopropyl}-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide monohydrochloride
Intended use
Treatment of Kabuki syndrome
Orphan designation status
EU designation number
Date of designation

Takeda Pharma A/S
Delta Park 45
2665 Vallensbaek Strand
Tel: +45 4677 1111

Review of designation

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

Update history

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November 2022 Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2022 on request of the Sponsor.

Documents related to this orphan designation evaluation

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.

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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