Drugs

FDA Approves Luxturna for Rare Vision Loss

FDA has approved Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl), a new gene therapy to treat children and adults with an inherited form of vision loss that may result in blindness. An agency news release says that Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.

Luxturna is approved for treating patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients.

According to FDA, Luxturna works by delivering a normal copy of the RPE65 gene directly to retinal cells. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore patients’ vision loss. Luxturna is said to use a naturally occurring adeno-associated virus, which has been modified using recombinant DNA techniques, as a vehicle to deliver the normal human RPE65 gene to the retinal cells to restore vision.

Luxturna’s safety and efficacy were established in a clinical development program with a total of 41 patients between the ages of four and 44 years. The primary evidence of efficacy was based on a Phase 3 study with 31 participants by measuring the change from baseline to one year in a subject’s ability to navigate an obstacle course at various light levels. The most common adverse reactions from treatment with Luxturna included eye redness, cataract, increased intraocular pressure, and retinal tear.

Luxturna received priority review, a breakthrough therapy designation, and an orphan drug designation. Spark is being given a rare pediatric disease priority review voucher it can redeem in the future for a different product.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome