Atidarsagene autotemcel



Atidarsagene autotemcel is a gene therapy comprising autologous CD34+ enriched stem cells encoding the human arylsulfatase A (ARSA) gene used to treat metachromatic leukodystrophy (MLD).

Brand Names
Generic Name
Atidarsagene autotemcel
DrugBank Accession Number

Atidarsagene autotemcel, sold under the brand name Libmeldy, is a gene therapy comprising genetically modified autologous CD34+ enriched hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene.3

Libmeldy was granted orphan designation by the EMA in April 2007, and was issued a marketing authorization in the EU in December 2020 for the treatment of certain manifestations of metachromatic leukodystrophy (MLD).4 It was granted FDA approval under the brand name Lenmeldy in March 2024.6

Biologic Classification
Gene Therapies
Other gene therapies
  • Atidarsagene autotemcel
  • Autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene
  • Autologous CD34+ cells transduced with lentiviral vector which encodes for the aryl Sulfatase A complimentary deoxyribonucleic acid sequence
External IDs
  • GSK 2696274
  • OTL-200



Atidarsagene autotemcel is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arylsulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity in children with:3,5

  • late infantile or early juvenile forms, without clinical manifestations of the disease (i.e. pre-symptomatic), or
  • early juvenile form, with early clinical manifestations of the disease (i.e. symptomatic), who still have the ability to walk independently and before the onset of cognitive decline
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Associated Conditions
Indication TypeIndicationCombined Product DetailsApproval LevelAge GroupPatient CharacteristicsDose Form
Treatment ofEarly symptomatic early juvenile metachromatic leukodystrophy (mld)•••••••••••••••••••••••••••••
Treatment ofMetachromatic leukodystrophy (mld)•••••••••••••••••••••••••••••••• ••••• •••••••• ••••••••••••• •••••••••••••• ••••••••••••••
Treatment ofMetachromatic leukodystrophy (mld)•••••••••••••••••••••••••••••••• •••• ••••••••• ••••••••••••• •••••••••••••• ••••••••••••••
Treatment ofMetachromatic leukodystrophy (mld)••••••••••••••••••••••••••• •• •••• •••••••••••••• ••••••••••• ••••• •••••••• ••••••••••••• •••••••••••••• ••••••••••••••
Treatment ofPre-symptomatic early juvenile metachromatic leukodystrophy (mld)•••••••••••••••••••••••••••••
Contraindications & Blackbox Warnings
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The reconstitution of ARSA activity in the hematopoietic system was observed in all MLD patients treated with atidarsagene autotemcel.3 ARSA activity in the CSF grew from undetectable at baseline to detectable in all patients by month 6 post-treatment, and finally to reference range levels at year 1 post-treatment.3

Mechanism of action

Metachromatic leukodystrophy (MLD) is an autosomal recessive hereditary disorder characterized by an inborn metabolic error in the lysosomal enzyme arylsulfatase A (ARSA).1 The resulting accumulation of sulfatides causes myelin sheaths in the central and peripheral nervous systems to become dysfunctional and degrade, leading to a gradual decline in neurodevelopmental and neurocognitive abilities.1

Atidarsagene autotemcel uses autologous CD34+ enriched stem cells transduced with a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. Following infusion and engraftment of the stem cells, the genetically modified cells produce and secrete a functional version of ARSA.3

AHuman Arylsulfatase A mRNA
gene replacement

Not Available

Volume of distribution

Not Available

Protein binding

Not Available

Not Available
Route of elimination

Not Available


Not Available


Not Available

Adverse Effects
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There are no data regarding overdosage with atidarsagene autotemcel.

Not Available
Pharmacogenomic Effects/ADRs
Not Available


Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
ElvitegravirThe therapeutic efficacy of Atidarsagene autotemcel can be decreased when used in combination with Elvitegravir.
FostemsavirThe therapeutic efficacy of Atidarsagene autotemcel can be decreased when used in combination with Fostemsavir.
IbalizumabThe therapeutic efficacy of Atidarsagene autotemcel can be decreased when used in combination with Ibalizumab.
Food Interactions
No interactions found.


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Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
LenmeldySuspension11800000 1/1IntravenousOrchard Therapeutics (Europe) Ltd2024-04-26Not applicableUS flag
Libmeldy6000000 cells/mLIntravenousOrchard Therapeutics (Netherlands) Bv2021-02-11Not applicableEU flag


ATC Codes
A16AB21 — Atidarsagene autotemcel
Drug Categories
Not classified
Affected organisms
  • Humans and other mammals

Chemical Identifiers

CAS number
Not Available


General References
  1. Rodrigues KF, Yong WTL, Bhuiyan MSA, Siddiquee S, Shah MD, Venmathi Maran BA: Current Understanding on the Genetic Basis of Key Metabolic Disorders: A Review. Biology (Basel). 2022 Sep 2;11(9):1308. doi: 10.3390/biology11091308. [Article]
  2. Messina M, Gissen P: Atidarsagene autotemcel for metachromatic leukodystrophy. Drugs Today (Barc). 2023 Feb;59(2):63-70. doi: 10.1358/dot.2023.59.2.3461911. [Article]
  3. EMA Summary of Product Characteristics: Libmeldy (atidarsagene autotemcel) dispersion for intravenous infusion [Link]
  4. EMA EPAR: Libmeldy (atidarsagene autotemcel) [Link]
  5. FDA Approved Drug Products: Lenmeldy (atidarsagene autotemcel) suspension for intravenous infusion [Link]
  6. FDA Press Announcement: FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy [Link]

Clinical Trials

Clinical Trials


Not Available
Not Available
Dosage Forms
SuspensionIntravenous11800000 1/1
Not Available
Not Available


Experimental Properties
Not Available


Drug created at February 15, 2023 18:57 / Updated at March 27, 2024 05:12