Identification
- Summary
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).
- Generic Name
- Atidarsagene autotemcel
- DrugBank Accession Number
- DB17538
- Background
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
- Type
- Biotech
- Groups
- Approved
- Biologic Classification
- Gene Therapies
Other gene therapies - Synonyms
- 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
Pharmacology
- Indication
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
Reduce drug development failure ratesBuild, train, & validate machine-learning modelswith evidence-based and structured datasets.Build, train, & validate predictive machine-learning models with structured datasets.- Associated Conditions
Indication Type Indication Combined Product Details Approval Level Age Group Patient Characteristics Dose Form Treatment of Early symptomatic early juvenile metachromatic leukodystrophy (mld) •••••••••••• •••••••• ••••••••• Treatment of Metachromatic leukodystrophy (mld) •••••••••••• •••••••• •••••••••••• ••••• •••••••• ••••••••••••• •••••••••••••• ••••• ••••••••• Treatment of Metachromatic leukodystrophy (mld) •••••••••••• •••••••• •••••••••••• •••• ••••••••• ••••••••••••• •••••••••••••• ••••• ••••••••• Treatment of Metachromatic leukodystrophy (mld) •••••••••••• •••••••• ••••••• •• •••• •••••••••••••• ••••••••••• ••••• •••••••• ••••••••••••• •••••••••••••• ••••• ••••••••• Treatment of Pre-symptomatic early juvenile metachromatic leukodystrophy (mld) •••••••••••• •••••••• ••••••••• - Contraindications & Blackbox Warnings
Prevent Adverse Drug Events TodayTap into our Clinical API for life-saving information on contraindications & blackbox warnings, population restrictions, harmful risks, & more.Avoid life-threatening adverse drug events with our Clinical API- Pharmacodynamics
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
Target Actions Organism AHuman Arylsulfatase A mRNA gene replacementHumans - Absorption
Not Available
- Volume of distribution
Not Available
- Protein binding
Not Available
- Metabolism
- Not Available
- Route of elimination
Not Available
- Half-life
Not Available
- Clearance
Not Available
- Adverse Effects
Improve decision support & research outcomesWith structured adverse effects data, including: blackbox warnings, adverse reactions, warning & precautions, & incidence rates. View sample adverse effects data in our new Data Library!Improve decision support & research outcomes with our structured adverse effects data.- Toxicity
There are no data regarding overdosage with atidarsagene autotemcel.
- Pathways
- Not Available
- Pharmacogenomic Effects/ADRs
- Not Available
Interactions
- 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.
Drug Interaction Integrate drug-drug
interactions in your softwareElvitegravir The therapeutic efficacy of Atidarsagene autotemcel can be decreased when used in combination with Elvitegravir. Fostemsavir The therapeutic efficacy of Atidarsagene autotemcel can be decreased when used in combination with Fostemsavir. Ibalizumab The therapeutic efficacy of Atidarsagene autotemcel can be decreased when used in combination with Ibalizumab. - Food Interactions
- No interactions found.
Products
Drug product information from 10+ global regionsOur datasets provide approved product information including:dosage, form, labeller, route of administration, and marketing period.Access drug product information from over 10 global regions.- Brand Name Prescription Products
Name Dosage Strength Route Labeller Marketing Start Marketing End Region Image Lenmeldy Suspension 11800000 1/1 Intravenous Orchard Therapeutics (Europe) Ltd 2024-03-21 Not applicable US 
Libmeldy 6000000 cells/mL Intravenous Orchard Therapeutics (Netherlands) Bv 2021-02-11 Not applicable EU 
Categories
- ATC Codes
- A16AB21 — Atidarsagene autotemcel
- Drug Categories
- Classification
- Not classified
- Affected organisms
- Humans and other mammals
Chemical Identifiers
- UNII
- EPP8G99QG4
- CAS number
- Not Available
References
- General References
- 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]
- 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]
- EMA Summary of Product Characteristics: Libmeldy (atidarsagene autotemcel) dispersion for intravenous infusion [Link]
- EMA EPAR: Libmeldy (atidarsagene autotemcel) [Link]
- FDA Approved Drug Products: Lenmeldy (atidarsagene autotemcel) suspension for intravenous infusion [Link]
- FDA Press Announcement: FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy [Link]
- External Links
- 2677916
- Wikipedia
- Atidarsagene_autotemcel
Clinical Trials
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Not Available
- Dosage Forms
Form Route Strength Suspension Intravenous 11800000 1/1 - Prices
- Not Available
- Patents
- Not Available
Properties
- State
- Liquid
- Experimental Properties
- Not Available
Targets
References
- EMA Summary of Product Characteristics: Libmeldy (atidarsagene autotemcel) dispersion for intravenous infusion [Link]
Drug created at February 15, 2023 18:57 / Updated at March 27, 2024 05:12