Voretigene neparvovec
| Gene therapy | |
|---|---|
| Target gene | RPE65 |
| Vector | Adeno-associated virus serotype 2 |
| Nucleic acid type | DNA |
| Clinical data | |
| Trade names | Luxturna |
| Other names | voretigene neparvovec-rzyl |
| AHFS/Drugs.com | Professional Drug Facts |
| License data | |
| Pregnancy category |
|
| Routes of administration | Subretinal injection |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis.
Leber's congenital amaurosis, or biallelic RPE65-mediated inherited retinal disease, is an inherited disorder causing progressive blindness. Voretigene is the first treatment available for this condition. The gene therapy is not a cure for the condition, but substantially improves vision in those treated. It is given as a subretinal injection.
Voretigene neparvovec was approved for medical use in the United States in December 2017, in Australia in August 2020, in Canada in October 2020, and in Switzerland in February 2020. It is the first in vivo gene therapy approved by the US Food and Drug Administration (FDA).