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On 31 May 2018, the Committee for Medicinal Products for Human Use (CHMP) adopted a negative opinion, recommending the refusal of the marketing authorisation for the medicinal product Exondys, intended for the treatment of Duchenne muscular dystrophy. The company that applied for authorisation, AVI Biopharma International Ltd, requested a re-examination of the CHMP's opinion on 1 June 2018.
After considering the grounds for this request, the CHMP re-examined the opinion, and confirmed the refusal of the marketing authorisation on 20 September 2018.
Questions and answers on refusal of the marketing authorisation for Exondys (eteplirsen) - Outcome of re-examination (PDF/73.99 KB)
First published: 01/07/2018
Last updated: 21/09/2018
|International non-proprietary name (INN) or common name||
|Therapeutic area (MeSH)||
Muscular Dystrophy, Duchenne
|Anatomical therapeutic chemical (ATC) code||
This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.
AVI Biopharma International Ltd
|Date of opinion||
|Date of refusal of marketing authorisation||