EU/3/14/1259: Orphan designation for the treatment of nontuberculous mycobacterial lung disease

Amikacin sulfate


On 8 April 2014, orphan designation (EU/3/14/1259) was granted by the European Commission to Insmed Limited, United Kingdom, for amikacin sulfate for the treatment of nontuberculous mycobacterial lung disease.

The sponsor’s address was updated in February 2019.

The sponsorship was transferred to Insmed Netherlands B.V., Netherlands, in February 2019.

The sponsor’s address was updated in October 2020.

Amikacin sulfate has been authorised in the EU as Arikayce liposomal since 27 October 2020. 

Key facts

Active substance
Amikacin sulfate
Intended use
Treatment of nontuberculous mycobacterial lung disease
Orphan designation status
EU designation number
Date of designation

Insmed Netherlands B.V.
Stadsplateau 7
3521 AZ Utrecht
Tel: +31302919894


Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Arikayce liposomal at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF icon orphan medicine assessment report .

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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