EU/3/11/857: Orphan designation for the treatment of transthyretin-mediated amyloidosis

Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran)

Overview

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.

Key facts

Active substance
Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran)
Medicine name
Onpattro
Intended use
Treatment of transthyretin-mediated amyloidosis
Orphan designation status
Positive
EU designation number
EU/3/11/857
Date of designation
06/04/2017
Sponsor

Alnylam Netherlands B.V.
Floor 20th
Antonio Vivaldistraat 150
1083 HP Amsterdam
Nord-Holland
Netherlands
Tel. +31 203697861
E-mail: info@alnylam.com

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Onpattro at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF icon orphan medicine assessment report .

Documents related to this orphan designation evaluation

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.

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:

Related content

How useful was this page?

Add your rating
Average
3 ratings