EU/3/18/2041

Table of contents

About

On 31 July 2018, orphan designation (EU/3/18/2041) was granted by the European Commission to Ionis USA Ltd, United Kingdom, for 2ʹ-O-(2-methoxyethyl) antisense oligonucleotide targeting microtubule-associated protein tau pre-mRNA for the treatment of behavioural variant frontotemporal dementia.

Key facts

Active substance
2ʹ-O-(2-methoxyethyl) antisense oligonucleotide targeting microtubule-associated protein tau pre-mRNA
Disease / condition
treatment of behavioural variant frontotemporal dementia
Date of decision
31/07/2018
Outcome
Positive
Orphan decision number
EU/3/18/2041

Sponsor's contact details

Ionis USA Ltd
Tower 42, Level 30
International Finance Centre
25 Old Broad Street
London EC2N 1HQ
United Kingdom
Tel. +44 (0)20 7786 6104
E-mail: info@ionisph.com

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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