On 31 July 2018, orphan designation (EU/3/18/2051) was granted by the European Commission to Wave life Sciences Ireland Limited, Ireland, for synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA (also known as WVE 210201) for the treatment of Duchenne muscular dystrophy.

Key facts

Active substance
Synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA
Disease / condition
Treatment of Duchenne muscular dystrophy
Date of decision
Orphan decision 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

One Spencer Dock
North Wall Quay
Dublin 1
Tel. +44 787 57 37 516            

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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