Voretigene neparvovec

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. ==Chemistry and production==

Voretigene neparvovec is an AAV2 vector containing human RPE65 cDNA with a modified Kozak sequence. The virus is grown in HEK 293 cells and purified for administration. ==History==

It was developed by Spark Therapeutics and Children's Hospital of Philadelphia. It was granted orphan drug designation for Leber congenital amaurosis and retinitis pigmentosa. A biologics license application was submitted to the US Food and Drug Administration (FDA) in July 2017 with Priority Review. Phase III clinical trial results were published in August 2017. On 12 October 2017, a key advisory panel to the FDA, composed of 16 experts, unanimously recommended approval of the treatment. The FDA approved the drug in December 2017. With the approval, Spark Therapeutics received a pediatric disease priority review voucher. == Society and culture ==

Economics 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. == References ==