This medicine is now known as resamirigene bilparvovec.

On 10 August 2015, orphan designation (EU/3/15/1539) was granted by the European Commission to Audentes Therapeutics UK Limited, United Kingdom, for adeno-associated viral vector serotype 8 containing the human MTM1 gene for the treatment of X-linked myotubular myopathy.

The sponsorship was transferred to Audentes Therapeutics Netherlands B.V., The Netherlands, in April 2019.

The sponsorship was transferred to Astellas Pharma Europe B.V. in September 2020.

Key facts

Active substance
Adeno-associated viral vector serotype 8 containing the human MTM1 gene (resamirigene bilparvovec)
Disease / condition
Treatment of X-linked myotubular myopathy
Date of first decision
EU designation number

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

Astellas Pharma Europe B.V
Sylviusweg 62
2333 BE Leiden
Tel. +31 (0)61 502 1591

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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