FDA Approves Novel Gene Therapy for Rare Form of Vision Loss

FDA Approves Novel Gene Therapy for Rare Form of Vision Loss

The US Food and Drug Administration (FDA) today approved a new gene therapy to treat a rare form of inherited vision loss that can lead to blindness.

Voretigene neparvovec-rzyl (Luxturna, Spark Therapeutics) is targeted for adults and children with confirmed biallelic RPE65 mutation–associated retinal dystrophy, a disease that affects between 1000 and 2000 people each year in the United States.

The treatment, which is delivered surgically via subretinal injection, is the first gene therapy approved in the United States that targets a disease caused by mutations in a specific gene.

It comes on the heels of two other gene therapy approvals this year for patients with rare and serious diseases.

An advisory committee to the FDA unanimously recommended this therapy in October.

The RPE65 gene normally provides instructions for making an enzyme that is essential for normal vision. When there are mutations, that process is blocked, resulting in impaired vision. Luxturna works by sending a normal copy of the RPE65 gene directly to retinal cells, which can then produce the normal protein to restore vision.

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