EU/3/12/1060: Orphan designation for the treatment of systemic light chain amyloidosis



On 8 November 2012, orphan designation (EU/3/12/1060) was granted by the European Commission to Takeda Global Research and Development Centre (Europe) Ltd, United Kingdom, for ixazomib for the treatment of systemic light chain amyloidosis.

In November 2013, Takeda Global Research and Development Centre (Europe) Ltd changed name to Takeda Development Centre Europe Ltd.

The sponsorship was transferred to Takeda Pharma A/S, Denmark, in March 2015.

The sponsor’s address was updated in August 2020.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in April 2021 on request of the Sponsor.

Key facts

Active substance
Intended use
Treatment of systemic light chain amyloidosis
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.

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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