EU/3/21/2447: Orphan designation for the treatment of neuronal ceroid lipofuscinosis

Adeno-associated virus serotype 9 expressing human CLN5

Table of contents


This medicine was designated as an orphan medicine for the treatment of neuronal ceroid lipofuscinosis in the European Union on 21 June 2021.

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 9 expressing human CLN5
Intended use
Treatment of neuronal ceroid lipofuscinosis
Orphan designation status
EU designation number
Date of designation

Real Regulatory Limited
Unit 9A
Plato Business Park
Dublin 15 D15 PA4C

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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