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voretigene neparvovec · 1 trial · 1 indication
Full-field light sensitivity threshold (FST) is evaluated using white light, as averaged over both eyes.
| Arm | Type | Description |
|---|---|---|
| Voretigene neparvovec | EXPERIMENTAL | 1.5 E11 vg (0.3 mL subretinal injection in each eye, 6-18 days apart) |
| Name | Type | Description |
|---|---|---|
| voretigene neparvovec | GENETIC | Voretigene neparvovec is an adeno-associated viral type 2 (AAV2) gene therapy vector driving expression of normal human retinal pigment epithelium 65 kDa protein (hRPE65) gene. |
Inclusion Criteria: * Japanese participants with biallelic RPE65 mutation-associated retinal dystrophy; molecular diagnosis of RPE65 mutation must be confirmed by a Novartis designated laboratory in Japan. * Age four years or older. * Visual acuity worse than 20/60 (both eyes) and/or visual field l...
Voretigene neparovec is a gene therapy used for biallelic RPE65 mutation-associated retinal dystrophy, a rare inherited eye disease that causes progressive vision loss. It is administered to patients with this specific genetic mutation to address the underlying cause of the condition.
Voretigene neparovec is developed by Novartis AG, a global healthcare company. Novartis is responsible for the clinical development and commercialization of this gene therapy for the treatment of biallelic RPE65 mutation-associated retinal dystrophy.
Voretigene neparovec is in Phase 3 clinical development. A Phase 3 trial has been completed in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy, and the drug remains an investigational therapy in clinical development.
Voretigene neparovec has been studied in a Phase 3 clinical trial with the identifier NCT04516369. This trial, titled 'Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy,' enrolled 4 participants and has been completed.
Voretigene neparovec is also known as Luxturna, a brand name for this gene therapy. It is used to treat biallelic RPE65 mutation-associated retinal dystrophy, and the drug is developed by Novartis AG.