EU/3/22/2592: Orphan designation for the treatment of mucopolysaccharidosis type II (Hunter syndrome)

Adeno-associated virus serotype HSC 15 expressing human iduronate 2-sulfatase (Bevufenogene nofeparvovec)

Table of contents


This medicine was designated as an orphan medicine for the treatment of mucopolysaccharidosis type II (Hunter syndrome) (Bevufenogene nofeparvovec) in the European Union on 16 March 2022.

This medicine is now known as Bevufenogene nofeparvovec.

This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.

Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:

Key facts

Active substance
Adeno-associated virus serotype HSC 15 expressing human iduronate 2-sulfatase (Bevufenogene nofeparvovec)
Intended use
Treatment of mucopolysaccharidosis type II (Hunter Syndrome)
Orphan designation status
EU designation number
Date of designation

Propharma Group The Netherlands B.V.
Schipholweg 73
2316 ZL Leiden

Update history

October 2022The sponsorship was transferred from Diamond Pharma Services Ireland Limited, Ireland to Propharma Group The Netherlands B.V., Netherlands.
August 2023Please note that this product was withdrawn from the Union Register of orphan medicinal products in August 2023 on request of the Sponsor.

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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