EU/3/18/2082

About

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.

Key facts

Active substance
5-{(1R,2R)-2-[(cyclopropylmethyl)amino]cyclopropyl}-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide monohydrochloride
Disease / condition
Treatment of Kabuki syndrome
Date of decision
19/11/2018
Outcome
Positive
Orphan decision number
EU/3/18/2082

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

Takeda Pharma A/S
Dybendal Alle 10
Taastrup
2630 Denmark
Tel. +45 46 77 1036
E-mail: info@takeda.dk

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;

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