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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 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).
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.
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