Libmeldy
What Libmeldy is used for
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.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Advanced therapy: a gene, cell or tissue-engineered medicine.
Key facts
- Brand
- Libmeldy
- Active substance
- autologous CD34+ cells encoding ARSA gene
- Disease area
- Leukodystrophy, Metachromatic
- Medicine group
- Other nervous system drugs
- ATC code
- N07
- Marketing authorisation holder
- Orchard Therapeutics (Netherlands) B.V.
- Authorised in the EU since
- 2020
Official information
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Libmeldy used for?
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.
What is the active substance in Libmeldy?
Libmeldy contains autologous CD34+ cells encoding ARSA gene.
Who makes Libmeldy?
The EU marketing authorisation holder is Orchard Therapeutics (Netherlands) B.V..
Is Libmeldy an orphan medicine?
Yes. Libmeldy is designated an orphan medicine in the EU, for a rare disease (Leukodystrophy, Metachromatic).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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