On 27 June 2016, orphan designation (EU/3/16/1671) was granted by the European Commission to Coté Orphan Consulting UK Limited, United Kingdom, for 2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting the growth hormone receptor (also known as ATL1103) for the treatment of acromegaly.
The sponsorship was transferred to Quintiles Ireland Limited, Ireland, in March 2018.
In May 2018 the sponsor, Quintiles Ireland Limited changed name to IQVIA RDS Ireland Limited.
2'-O-(2-Methoxyethyl) phosphorothioate antisense oligonucleotide targeting the growth hormone receptor
Treatment of acromegaly
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IQVIA RDS Ireland Limited
Documents related to this orphan designation evaluation
EU/3/16/1671: Public summary of opinion on orphan designation: C2'-O-(2-Methoxyethyl) phosphorothioate antisense oligonucleotide targeting the growth hormone receptor for the treatment of acromegaly (PDF/125.19 KB)
First published: 26/07/2016
Last updated: 26/07/2016
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
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: