EU/3/08/586

Table of contents

About

This medicine is now known as Eteplirsen.

On 3 December 2008, orphan designation (EU/3/08/586) was granted by the European Commission to AVI Biopharma International, United Kingdom, for RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5') (C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), P-[4[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl] N,N-dimethylaminophosphonamidate for the treatment of Duchenne muscular dystrophy.

The sponsorship was transferred to Sarepta Therapeutics Ireland Limited, Ireland, in January 2019.

Key facts

Active substance
RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5') (C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N dimethylaminophosphonamidate (Eteplirsen)
Disease / condition
Treatment of Duchenne muscular dystrophy
Date of first decision
03/12/2008
Outcome
Positive
EU designation number
EU/3/08/586

Sponsor's contact details

Regus House
Harcourt Centre, Harcourt Road
Dublin 2
D02 HW77
Ireland
E-mail: info@sarepta.com

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

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