This section contains copies of the official designation letters from the FDA Office of Orphan Products Development.
1. Designation #08-2593 (June 24, 2008): For treatment of Leber congenital amaurosis due to RPE65 mutation.
2. Designation #15-4927 (March 18, 2015): For treatment of retinitis pigmentosa due to autosomal recessive RPE65 gene mutations.
3. Designation #16-5744 (November 29, 2016): For the use of 'adeno-associated viral vector type 2 expressing human recombinant retinal pigment epithelial 65KDa protein gene for the treatment of inherited retinal dystrophy due to biallelic RPE65 mutations.'