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Voretigene neparvovec injection

Updated 2 Feb 2023 | Voretigene neparvovec


Injection of voretigene neparvovec.

Drugs List

  • LUXTURNA 5tera vector genomes/1ml concentrate for solution for injection
  • voretigene neparvovec 5tera vector genomes/1ml concentrate for solution for injection
  • Therapeutic Indications


    Inherited retinal dystrophy caused by biallelic RPE65 mutations

    Treatment of vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.


    Treatment should be administered by a retinal surgeon experienced in macular surgery.


    1.5x10 to the power of 11 vg voretigene neparvovec given as a subretinal injection in a volume of 0.3ml into each eye. Administration into each eye is performed on separate days, at least 6 days apart.


    1.5x10 to the power of 11 vg voretigene neparvovec given as a subretinal injection in a volume of 0.3ml into each eye. Administration into each eye is performed on separate days, at least 6 days apart.

    Additional Dosage Information

    Prior to administration of voretigene neparvovec in the first eye, it is recommended that an immunomodulatory regimen is initiated, starting three days before.

    Initiation of the immunomodulatory regimen for the second eye should follow the same schedule and supersede that of the first eye.

    3 days prior to administration of voretigene neparvovec: 1mg/kg/day of prednisone (maximum 40mg/day).

    4 days (including the day of administration): 1mg/kg/day of prednisone (maximum 40mg/day).
    Followed by 5 days: 0.5mg/kg/day of prednisone (maximum 20mg/day).
    Followed by 5 days of one dose every other day: 0.5mg/kg/day of prednisone (maximum 20mg/day).


    For subretinal use only.


    Children under 4 years
    Ocular infection
    Ocular inflammation
    Periocular infection

    Precautions and Warnings

    Postpone treatment if there is active or suspected infection
    Advise visual disturbances may affect ability to drive or operate machinery
    Monitor and manage intraocular pressure before and after administration
    Systemic corticosteroids should be given to reduce risk of immunogenicity
    Advise patient and carers to wear gloves when changing dressings
    Aseptic technique should be used throughout
    Record name and batch number of administered product
    Treatment to be administered by or under supervision of specialist
    Vector shedding may occur in patient tears and nasal secretions
    Advise patient to report any symptoms of endophthalmitis immediately
    Advise patient to report any symptoms of retinal detachment immediately
    Advise patient to report any unexpected changes in eye symptoms immediately
    Advise patient to avoid air travelling for 1 week after administration
    Advise patient to avoid donating blood, organs, tissues or cells
    Avoid strenuous exercise or swimming for at least 1-2 weeks after treatment

    Air travel (or other travel at high elevations) should be avoided until the air bubble formed, as a result of administration, has completely dissipated from the eye. An increase in altitude while the air bubble is still present can cause a rise in eye pressure and irreversible vision loss. Dissipation of the air bubble should be verified by ophthalmic examination.

    Transient and low-level vector shedding may occur in patient tears. Patients and caregivers should be advised to wear gloves for dressing changes and waste disposal. It is also advised that waste material generated from dressings, tears and nasal secretion appropriately. These handling precautions should be followed by 14 days after administration.

    Pregnancy and Lactation


    Voretigene neparvovec is contraindicated during pregnancy.

    The manufacturer does not recommend using voretigene neparvovec during pregnancy. At the time of writing there is limited published information regarding the use of voretigene neparvovec during pregnancy. Potential risks are unknown.


    Voretigene neparvovec is contraindicated during breastfeeding.

    The manufacturer advises that the patient either discontinues voretigene neparvovec or discontinues breastfeeding. The presence of voretigene neparvovec in human breast milk is unknown. Effects on exposed infants are unknown.

    Side Effects

    Abdominal pain
    Chorioretinal atrophy
    Conjunctival hyperaemia
    Corneal dellen
    ECG changes
    Eye disorder
    Facial oedema
    Increased intra-ocular pressure
    Lip pain
    Macular degeneration
    Macular hole
    Ocular inflammation
    Ocular irritation
    Ocular oedema
    Ocular pain
    Optic atrophy
    Retinal detachment
    Retinal haemorrhage
    Retinal tear
    Sensation of foreign body in eye
    Visual disturbances
    Vitreous opacities


    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 2021

    Reference Sources

    Summary of Product Characteristics: Luxturna 5 tera vector genomes/ml concentrate and solvent for solution for injection. Novartis Pharmaceuticals UK Ltd. Revised February 2022.

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