EU/3/15/1509: Orphan designation for the treatment of spinal muscular atrophy

Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec)

Overview

On 19 June 2015, orphan designation (EU/3/15/1509) was granted by the European Commission to AveXis EU, Ltd, Ireland, for adeno-associated viral vector serotype 9 containing the human SMN gene for the treatment of spinal muscular atrophy.

The sponsorship was transferred to Avexis Netherlands B.V., The Netherlands, in September 2018.

The sponsorship was transferred to AveXis EU Limited, Ireland, in September 2019.

Adeno-associated viral vector serotype 9 containing the human SMN gene has been authorised in the EU as Zolgensma since 18 May 2020.

In December 2020, AveXis EU Limited changed name to Novartis Gene Therapies EU Limited.

The sponsorship was transferred to Novartis Europharm Limited, Ireland in July 2022.

This medicine is also known as onasemnogene abeparvovec.

Key facts

Active substance
Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec)
Medicine name
Zolgensma
Intended use
Treatment of spinal muscular atrophy
Orphan designation status
Positive
EU designation number
EU/3/15/1509
Date of designation
19/06/2015
Sponsor

Novartis Europharm Limited
Vista Building
Elmpark
Merrion Road
Dublin 4
D04 A9N6
Ireland

E-mail: orphan.enquiries@novartis.com

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Zolgensma 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 .

Update history

DATE Update
July 2022 The sponsorship was transferred to Novartis Europharm Limited, Ireland in July 2022.

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:

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