EU/3/11/857

About

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.

Update: 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.

Key facts

Active substance
Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran)
Medicine name
Onpattro
Disease / condition
Treatment of transthyretin-mediated amyloidosis
Date of first decision
06/04/2017
Outcome
Positive
EU designation number
EU/3/11/857

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 iconorphan medicine assessment report .

Sponsor's contact details

Alnylam Netherlands B.V.
Strawinskylaan 3051
1077ZX
Amsterdam
The Netherlands
Tel. + 31 20 369 0716
E-mail: info@alnylam.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;

How useful was this page?

Add your rating