Spark Therapeutics has gained US Food and Drug Administration (FDA) approval for its one-time gene therapy product, Luxturna (voretigene neparvovec-rzyl), to treat children and adults with biallelic RPE65 mutation-associated retinal dystrophy.
Luxturna is said to be the first directly administered adeno-associated virus (AAV) vector gene therapy approved in the country to target a condition caused by mutations in a specific gene.
The mutation associated retinal dystrophy is an inherited form of vision loss that could result in complete blindness.
Luxturna directly delivers a normal copy of the RPE65 gene to retinal cells, allowing production of a normal protein that works in the retina to restore vision loss.
Set to be manufactured at the firm’s facility in West Philadelphia, US, the gene therapy will be administered at select treatment centres in the country in the first quarter of next year.
The firm has received priority review, breakthrough therapy and orphan drug designation for Luxturna, along with a rare paediatric disease priority review voucher that can be used during future marketing application for a different product.
FDA commissioner Scott Gottlieb said: “This approval marks another first in the field of gene therapy, both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss. And this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases.
“Next year, we’ll begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products to lay out modern and more efficient parameters, including new clinical measures, for the evaluation and review of gene therapy for different high-priority diseases where the platform is being targeted.”