Atidarsagene Autotemcel
Atidarsagene autotemcel (LENMELDY) is an ex vivo gene therapy approved for the treatment of children with metachromatic leukodystrophy (MLD), a rare, progressive, often fatal neurodegenerative disease caused by deficiency of the arylsulfatase A (ARSA) enzyme. The therapy uses a lentiviral vector to insert functional copies of the ARSA gene into the patient's own hematopoietic stem cells, which are then reinfused to produce the deficient enzyme after engraftment in the bone marrow.
LENMELDY is a gene therapy for children with metachromatic leukodystrophy that modifies the patient's own stem cells to produce the missing ARSA enzyme.
Key facts at a glance
- ATC
- A16AB21
- RxNorm
- 2677916
- Generic
- Atidarsagene Autotemcel
- Class
- atc-a16
- Brands
- Lenmeldy
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 500mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 250mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 400mg daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
11 groupspregnancy
first trimestercontraindicated - avoid use
avoid use during pregnancy due to requirement for busulfan conditioning which is teratogenic
not applicable - avoid pregnancy before and after treatment
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
second trimestercontraindicated - avoid use
avoid use during pregnancy due to requirement for busulfan conditioning which is teratogenic
not applicable - avoid pregnancy before and after treatment
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
third trimestercontraindicated - avoid use
avoid use during pregnancy due to requirement for busulfan conditioning which is teratogenic
not applicable - avoid pregnancy before and after treatment
Use of this medication in this population requires careful consideration of risks and benefits.
lactation
breastfeedingcontraindicated
avoid breastfeeding during treatment and for period following conditioning chemotherapy
not applicable
Use of this medication in this population requires careful consideration of risks and benefits.
pediatric_age
infantsno adjustment - weight-based dosing
indicated for presymptomatic late infantile MLD patients under 30 months and early juvenile MLD patients under 12 years
close hematologic monitoring required post-infusion due to myelosuppression from conditioning
pediatric_age
childrenno adjustment - weight-based dosing
indicated for early juvenile MLD patients under 12 years of age
close hematologic monitoring and engraftment assessment required
geriatric
elderlyuse with caution
limited safety and efficacy data in patients over 65 years; benefit-risk assessment required
enhanced monitoring of cardiac and hepatic function given age-related comorbidities
Use with caution and monitor for adverse effects.
renal_impairment
mildno initial dose adjustment for gene therapy; busulfan may require adjustment
busulfan conditioning dose adjustment may be needed based on renal function
monitor renal function and adjust conditioning regimen accordingly
Use with caution and monitor for adverse effects.
renal_impairment
moderate to severedose adjustment required for conditioning regimen
busulfan conditioning dose reduction recommended; consider alternative conditioning if severe impairment
frequent monitoring of renal function and drug levels during conditioning
Use with caution and monitor for adverse effects.
hepatic_impairment
mildno specific adjustment for gene therapy
caution advised; evaluate hepatic function before conditioning
monitor hepatic function parameters
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate to severeuse with caution
not studied in moderate to severe hepatic impairment; careful risk-benefit assessment required
enhanced hepatic function monitoring
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA Approval Date
FDA approved Atidarsagene Autotemcel (Lenmeldy) for metachromatic leukodystrophy
Therapeutic Class
Classification as autologous gene therapy
Indication
Approved indication for early-onset MLD
Mechanism of Action
Gene therapy using lentiviral vector to deliver functional ARSA gene
Atidarsagene autotemcel is indicated for the treatment of children with metachromatic leukodystrophy (MLD), a rare lysosomal storage disorder caused by ARSA deficiency that leads to progressive neurological deterioration.
The therapy involves collecting the patient's CD34+ hematopoietic stem cells and modifying them ex vivo using a lentiviral vector to insert functional copies of the ARSA gene. After the transduced cells are reinfused, they engraft in the bone marrow and repopulate, enabling long-term production of functional ARSA enzyme.
This is an autologous therapy using the patient's own cells, so there is no risk of graft-versus-host disease or immune rejection. However, as a one-time gene therapy, it requires specialized administration at qualified treatment centers. Safety monitoring for potential vector-related effects and hematologic considerations apply.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy