Autologous CD34+ cells encoding ARSA gene

This medicine is authorised for use in the European Union.


Libmeldy is a medicine used to treat children with metachromatic leukodystrophy (MLD). MLD is a rare inherited disorder in which there is a change (mutation) in a gene needed to make an enzyme called arylsulfatase A (ARSA), which breaks down substances called sulfatides. As a result, sulfatides build up and damage the nervous system and other organs, causing symptoms such as walking difficulties, gradual mental deterioration and eventual death.

Libmeldy is used in children with MLD who have mutations in the ARSA gene. It is given to

  • those with late infantile or early juvenile forms of the disease who have not yet developed symptoms;
  • those with early juvenile MLD who have initial symptoms but can still walk independently and have not yet developed mental deterioration.

Libmeldy is a type of advanced therapy medicine called a ‘gene therapy’. This type of medicine works by delivering genes into the body. The active substance in Libmeldy is stem cells, (CD34+ cells), derived from the patient’s own bone marrow or blood, that have been modified to contain a copy of the gene to make ARSA and can divide to produce other sorts of blood cells.

MLD is rare, and Libmeldy was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 13 April 2007.

This EPAR was last updated on 09/03/2021

Authorisation details

Product details
Agency product number
Active substance
autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A gene
International non-proprietary name (INN) or common name
Autologous CD34+ cells encoding ARSA gene
Therapeutic area (MeSH)
Leukodystrophy, Metachromatic
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
Orchard Therapeutics (Netherlands) BV
Date of issue of marketing authorisation valid throughout the European Union
Contact address
Prins Bernhardplein 200
1097 JB Amsterdam
The Netherlands

Product information

13/01/2021 Libmeldy - EMEA/H/C/005321 - IAIN/0001/G


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Pharmacotherapeutic group

Other nervous system drugs

Therapeutic indication

Libmeldy is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity:

in children with late infantile or early juvenile forms, without clinical manifestations of the disease,

in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.

Assessment history

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