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Atidarsagene autotemcel parenteral

Updated 2 Feb 2023 | Metachromatic leukodystrophy


Infusions of atidarsagene autotemcel.

Drugs List

  • atidarsagene autotemcel dispersion for infusion bag
  • LIBMELDY dispersion for infusion bag
  • Therapeutic Indications


    Metachromatic leukodystrophy

    Treatment of metachromatic leukodystrophy (MLD) in children with late infantile or early juvenile forms, without clinical manifestations of the disease and 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.


    Pre-medicate with 0.25mg/kg (maximum dose 10mg) intravenous chlorpheniramine or an equivalent 15 to 30 minutes before the atidarsagene automcel before the infusion.

    The dose of atidarsagene autotemcel is based on the patient's weight at the time of infusion. The minimum recommended dose is 3 x 10 to the power of 6 CD34+ cells/kg.

    The maximum volume of atidarsagene autotemcel to be administered should be less that 20% of the patient's estimated plasma volume. Only one bag should be infused per hour.


    Atidarsagene autotemcel should be prepared according to the manufacturer's instructions and using appropriate precautions. It should then be administered by intravenous infusion centrally.

    Atidarsagene autotemcel is strictly for autologous use only.


    History of haematopoietic stem cell gene therapy
    Within 6 weeks of live vaccines

    Precautions and Warnings

    Children aged 7 to 18 years
    Restricted sodium intake
    Hepatic impairment
    Renal impairment

    Avoid live vaccines 6 weeks before, during and until immune recovery
    Sodium content of formulation may be significant
    Adhere to standard protective measures to avoid infection
    Consider use of anti-infective prophylaxis during neutropenic phase
    Correct acid-base disorders prior to treatment
    Give pre-treatment counselling and consideration of oocyte cryopreservation
    Give pre-treatment counselling and consideration of sperm cryopreservation
    Premedicate with intravenous antihistamines
    Treatment to be initiated and supervised by a specialist
    Contains dimethyl sulfoxide (DMSO)
    For autologous use only
    Record name and batch number of administered product
    Monitor thyroid function prior to and periodically during treatment
    Screen all patients for HIV, HTLV, HBV, HCV and mycoplasma
    Monitor for cytopenia for at least 6 weeks after infusion
    Monitor for signs and symptoms of infection after treatment
    Monitor for signs and symptoms of metabolic acidosis
    Monitor for signs and symptoms of thrombosis
    Monitor for signs of leukaemia or lymphoma
    Monitor full blood count regularly
    Monitor patient before, throughout infusion & for at least 3 hrs afterwards
    Monitor patient for catheter-related adverse events
    Monitor patient for signs of serious infection
    Antibodies to ingredient may develop
    May lead to false positive HIV test result in non-infected individuals
    Advise patient to avoid donating blood, organs, tissues or cells
    Remind patient of importance of carrying Alert Card with them at all times

    Gloves and glasses should be worn to avoid the transmission of infectious blood-borne diseases.

    As atidarsagene autotemcel is strictly for autologous use only, it must be verified before infusion that the patient's identity matches the identifiers on the infusion bag label and lot information sheet.

    Patients should avoid anti-retroviral products from at least one month prior to mobilisation and/or bone marrow harvest until at least 7 days after atidarsagene autotemcel infusion. If anti-retrovirals are required following exposure to HIV/HTLV then initiation of atidarsagene autotemcel should be delayed until an HIV/HTLV western blot ad viral load assay have been performed at 6 months post-exposure.

    If cytopenia persists beyond 6 to 7 weeks despite granulocyte mobilising medicines, then the non-transduced back up stem cells should be infused. If cytopenia then still persists, alternative treatments should be sought.

    Monitor for anti-ARSA antibodies prior to treatment, between 1 and 2 months after gene therapy and then at 6 months, 1 year, 3 years, 5 years, 7 years, 9 years 12 years and 15 years post treatment. Additional monitoring is recommended in the case of disease onset or significant disease progression.

    Immunoglobulin G should be maintained above 5g/l to prevent potential late infections occurring later than 100 days post therapy, associated with severe hypogammaglobinaemia.

    If by day 60 after the infusion neutrophil engraftment failure exists, then the non-transduced back-up stem cells should be infused according to local standards.

    Mobilisation and conditioning medicines may cause dizziness and fatigue.

    Pregnancy and Lactation


    Not indicated in patients of child bearing age, relevant data is not available.


    Not indicated in patients of child bearing age, relevant data is not available.

    Side Effects

    Alanine aminotransferase increased
    Antibody formation
    Aspartate aminotransferase increased
    Back pain
    Bone pain
    Cytomegalovirus infection
    Fluid overload
    Gastro-intestinal haemorrhage
    Increase of liver transaminases
    Metabolic acidosis
    Oropharyngeal pain
    Ovarian failure
    Positive Aspergillus test
    Skin exfoliation
    Staphylococcal infection
    Urinary tract infections
    Veno-occlusive disease
    Viral infection


    It is strongly recommended that the UK National Poisons Information Service be consulted on cases of suspected or actual overdose where there is doubt over the degree of risk or about appropriate management.

    The following number will direct the caller to the relevant local centre (0844) 892 0111

    Information may be obtained if you have access to ToxBase the primary clinical toxicology database of the National Poisons Information Service. This is available via password on the internet ( ) or if this is unavailable at the backup site ( ).

    Further Information

    Last Full Review Date: March 2022

    Reference Sources

    Summary of Product Characteristics: Libmeldy dispersion for infusion. Orchard Therapeutics (Europe) Limited. Revised April 2021.

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