Voretigene neparvovec
Gene therapy medication
From Wikipedia, the free encyclopedia
Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis.[4]
| Gene therapy | |
|---|---|
| Target gene | RPE65 |
| Vector | Adeno-associated virus serotype 2 |
| Nucleic acid type | DNA |
| Clinical data | |
| Trade names | Luxturna |
| Other names | voretigene neparvovec-rzyl |
| AHFS/Drugs.com | Professional Drug Facts |
| License data | |
| Pregnancy category |
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| Routes of administration | Subretinal injection |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
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| KEGG | |
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 was the first in vivo gene therapy approved by the US Food and Drug Administration (FDA).[13]
Medical uses
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.[14]
History
It was developed by Spark Therapeutics and Children's Hospital of Philadelphia.[15][16][17] It was granted orphan drug designation for Leber congenital amaurosis and retinitis pigmentosa.[18][19] A biologics license application was submitted to the US Food and Drug Administration (FDA) in July 2017 with Priority Review.[7] Phase III clinical trial results were published in August 2017.[20] On 12 October 2017, a key advisory panel to the FDA, composed of 16 experts, unanimously recommended approval of the treatment.[21] The FDA approved the drug in December 2017.[9][5] With the approval, Spark Therapeutics received a pediatric disease priority review voucher.[22]
