Voretigene Neparvovec-rzyl Application Scope

Voretigene neparvovec-rzyl is a gene therapy used to treat retinal dystrophy caused by biallelic RPE65 mutations, including:

Leber Congenital Amaurosis (LCA) type 2 (66% of cases)
Early-onset severe retinal dystrophy (EOSRD)
Retinitis pigmentosa (RP) phenotype (16% of cases).

It is administered via subretinal injection to deliver a functional RPE65 gene to retinal cells, restoring the visual cycle in patients with confirmed mutations.

Voretigene Neparvovec-rzyl Characteristics

Ingredients: Recombinant adeno-associated virus serotype 2 (AAV2) vector containing human RPE65 cDNA (AAV2-hRPE65v2).
Properties:
- Single-dose gene therapy.
- Targets retinal pigment epithelium (RPE) cells to restore chromophore production.
Specification:
- Dose: 1.5 × 10¹¹ vector genomes (vg) in 0.3 mL subretinal injection per eye.
Packaging Specification: Not detailed in search results.
Storage: Likely requires cold chain (exact conditions not specified).
Expiry Date: Not specified.
Executive Standard: FDA-approved (2017), EMA-approved (2019).
Approval Number: Not provided.
Date of Revision: Last FDA label update: May 2024.
Manufacturer: Novartis (brand name: Luxturna).

Guidelines For The Use Of Voretigene Neparvovec-rzyl

Dosage and Administration:

Administered as a single subretinal injection per eye, with procedures separated by at least 6 days.
Requires pars plana vitrectomy for delivery.
Pre-treatment: Oral steroids (e.g., prednisone) may be given to reduce inflammation.
Post-treatment: Patients should avoid air travel, high elevations, or scuba diving for ≥1 week due to risk of intraocular air bubbles.

Adverse Reactions:

Common:
- Conjunctival hyperemia.
- Cataracts, increased intraocular pressure (IOP).
- Retinal tears, macular holes, subretinal deposits.
Serious:
- Endophthalmitis (eye infection).
- Vision changes (floaters, light flashes, blindness).

Contraindications:

Hypersensitivity to voretigene or its components.
Active ocular infections.

Precautions:

Monitor for retinal detachment, inflammation, or IOP spikes.
Avoid rubbing eyes; viral shedding in tears may persist for 7 days post-treatment.

Voretigene Neparvovec-rzyl Interactions

Drug Interactions:

No specific interactions noted, but systemic corticosteroids are often used concurrently to manage inflammation.
Avoid other retinal therapies that may increase procedural risks (e.g., anticoagulants).

Note:

If there is a new packaging for the drug, the new packaging shall prevail. The above information is sourced from DengYue Medicine.
It is only for internal discussion among medical staff and does not serve as a basis for medication. For specific medication guidelines, please consult the attending physician.