Voretigene neparvovec

USDailyMed:Voretigene neparvovec AU:B2 S01XA27(WHO) AU:S4(Prescription only)CA: Rx-only / Schedule DUS:℞-onlyEU:Rx-onlyCH: Rx-only 1646819-03-5 DB13932 2SPI046IKD D11008 Voretigene neparvovec, sold under the brand nameLuxturna, is agene therapymedication for the treatment ofLeber congenital amaurosis. Leber's congenital amaurosis, or biallelicRPE65-mediated inheritedretinaldisease, 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 was the firstin vivogene therapy approved by the USFood and Drug Administration(FDA).