EU/3/16/1622: Orphan designation for the treatment of haemophilia A

Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene

Overview

On 21 March 2016, orphan designation (EU/3/16/1622) was granted by the European Commission to BioMarin Europe Ltd., United Kingdom, for adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene (also called BMN 270) for the treatment of haemophilia A.

The sponsorship was transferred to BioMarin International Limited, Ireland in September 2018.

The medicinal product has been authorised in the EU as Roctavian since 24 August 2022.

Key facts

Active substance
Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene
Intended use
Treatment of haemophilia A
Orphan designation status
Positive
EU designation number
EU/3/16/1622
Date of designation
21/03/2016
Sponsor

BioMarin International Limited
Shanbally
Ringaskiddy
County Cork P43 R298
Ireland
Tel. +353 1479430
E-mail: shanbally@bmrn.com

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

The Committee for Orphan Medicinal Products reviewed the orphan designation of Roctavian at the time of marketing authorisation, and confirmed that the orphan designation should be maintained. More information is available in the 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:

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