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voretigene neparvovec-rzyl · 2 trials · 1 indication
The primary outcome measures are safety and tolerability. Secondary outcome measure(s) include changes in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests.
| Arm | Type | Description |
|---|---|---|
| voretigene neparvovec-rzyl (AAV2-hRPE65v2) | EXPERIMENTAL | Administration of study agent (AAV2-hRPE65v2) to the previously, uninjected contralateral eye: |
| dose cohort 1 | EXPERIMENTAL | 1.5E10 vector genomes voretigene neparvovec-rzyl in 150 microliters administered subretinally |
| dose cohort 2 | EXPERIMENTAL | 4.8E10 vector genomes voretigene neparvovec-rzyl in 150 microliters administered subretinally |
| dose cohort 3 | EXPERIMENTAL | 1.5E11 vector genomes voretigene neparvovec-rzyl in 300 microliters administered subretinally |
| Name | Type | Description |
|---|---|---|
| voretigene neparvovec-rzyl | BIOLOGICAL | One time, subretinal administration of 1.5E11 vg AAV2-hRPE65v2 vector in 300 microliters to the contralateral, previously uninjected eye. |
Inclusion Criteria: * Prior participation in Phase 1 study with unilateral, subretinal administration of AAV2-hRPE65v2. * Visual acuity equal to or greater than light perception. * Sufficient viable retinal cells in contralateral, previously uninjected eye, as determined by non-invasive means, such...
Voretigene neparvovec-rzyl is used for Leber Congenital Amaurosis, a rare inherited retinal disease. It is an investigational gene therapy being studied in clinical trials. The drug is not approved and is still in clinical development.
Voretigene neparvovec-rzyl is a gene therapy that targets the RPE65 gene, which is mutated in Leber Congenital Amaurosis. It delivers a normal copy of the gene to retinal cells to restore function. The target class is gene therapy.
Voretigene neparvovec-rzyl is developed by Roche Holding AG, traded as RHHBY on the OTC market. Roche is the developer of this investigational gene therapy for Leber Congenital Amaurosis.
Voretigene neparvovec-rzyl is in Phase 1 clinical development. It is an investigational drug, not FDA approved. There are two Phase 1 trials, one completed and one active but not recruiting.
Voretigene neparvovec-rzyl has two Phase 1 trials. NCT00516477 is a completed safety study in subjects with Leber Congenital Amaurosis. NCT01208389 is an active, not recruiting follow-on study of the vector in LCA2. Both enrolled 12 participants.
Voretigene neparvovec-rzyl is associated with the vector AAV2-hRPE65v2, as named in the clinical trial titles. The trials study this vector in subjects with Leber Congenital Amaurosis. The drug is also known by this vector name.