On 6 April 2017, orphan designation (EU/3/11/857) was granted by the European Commission to Alnylam UK Limited, United Kingdom, for synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran) for the treatment of transthyretin-mediated amyloidosis.
Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA has been authorised in the EU as Onpattro since 27 August 2018.
This medicine is now known as Patisiran.
The sponsorship was transferred to Alnylam Netherlands B.V., The Netherlands in May 2018.
The sponsor’s address was updated in November 2020.
Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran)
Treatment of transthyretin-mediated amyloidosis
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Review of designation
Documents related to this orphan designation evaluation
EU/3/11/857: Public summary of positive opinion for orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA for the treatment of transthyretin-mediated amyloidosis (PDF/124.54 KB)
First published: 26/04/2011
Last updated: 05/05/2017
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.
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: