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.[7] The gene therapy is not a cure for the condition, but substantially improves vision in those treated.[8] It is given as a subretinal injection.
Voretigene neparvovec was approved for medical use in the United States in December 2017,[9] in Australia in August 2020,[10] in Canada in October 2020,[11] and in Switzerland in February 2020.[12]. It is the first in vivo gene therapy approved by the US Food and Drug Administration (FDA).[13]
Medical uses
Voretigene neparvovec is indicated for the treatment of people with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.[6]
The first commercial sale of voretigene neparvovec, which was also the first sale of any gene therapy product in the United States, occurred in March 2018.[23][13] The price of the treatment at the time was announced as being $425,000 per eye.[24]
Russell S, Bennett J, Maguire AM, High KA (2018). "Voretigene neparvovec-rzyl for the treatment of biallelic RPE65 mutation–associated retinal dystrophy". Expert Opinion on Orphan Drugs. 6 (8): 457–464. doi:10.1080/21678707.2018.1508340. S2CID81437112.