voretigene neparvovec: clinical use

Prescribing considerations

• Confirm pathogenic biallelic RPE65 mutations and sufficient viable retinal cells before treatment.
• Administration requires an experienced retinal surgeon, specialist surgical facilities and aseptic subretinal technique.
• Check for active infection before associated immunomodulation and surgery; postpone treatment until recovery where appropriate.
• Use perioperative systemic corticosteroid immunomodulation according to the authorised product protocol.
• Record the product name and batch number for biological-product traceability.
• Advise that treated patients must not donate blood, organs, tissues or cells for transplantation.

Contraindications and cautions

• Hypersensitivity to voretigene neparvovec or an excipient
• Ocular or periocular infection
• Active intraocular inflammation

Monitoring

• Assess retinal viability and macular anatomy before treatment.
• Measure intraocular pressure before and after administration.
• Monitor for endophthalmitis, intraocular inflammation, retinal tears or detachment, macular complications and haemorrhage.
• Continue retinal imaging and functional vision assessment, including surveillance for progressive chorioretinal atrophy.
• Confirm resolution of the intraocular air bubble before permitting air travel or high-altitude travel.

Clinical pharmacology

A recombinant AAV2 vector delivers human RPE65 complementary DNA to retinal pigment epithelial cells. Expression of functional RPE65 supports production of 11-cis-retinal and restoration of the retinoid visual cycle.

Formulation and product differences

• Supplied as a frozen sterile concentrate with separate solvent; it requires thawing and aseptic dilution before use.
• The diluted solution is for subretinal injection only and must not be administered intravitreally.
• Each vial is single-use for administration to one eye; unused material requires disposal under local biosafety procedures.