Rare Daily Staff

An U.S. Food and Drug Administration Advisory committee unanimously recommended the approval of Spark Therapeutics Luxturna, a one-time gene therapy to treat vision loss from a form of inherited retinal disease.

The FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted 16-0 to recommend the approval of Luxturna for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease. The advisory committee’s vote is non-binding, but agency will take its recommendation into consideration when reviewing the application to market Luxturna and usually follows such recommendations.

If approved, it would be the first gene therapy approved in the United States to treat a genetic disease.

People living with inherited retinal disease due to biallelic RPE65 gene mutations often experience night blindness due to decreased light sensitivity in childhood or early adulthood and involuntary back-and-forth eye movements. As the disease progresses, individuals may experience loss in their peripheral vision, developing tunnel vision, and eventually, they may lose their central vision as well, resulting in total blindness. There are currently no approved pharmacologic treatment options for IRD due to biallelic RPE65 gene mutations.

“There currently are no pharmacologic treatment options for people living with RPE65-mediated IRD, who in most cases progress to complete blindness,” said Principal Investigator Albert Maguire, professor of ophthalmology at the Scheie Eye Institute at the University of Pennsylvania’s Perelman School of Medicine and attending physician in the Division of Pediatric Ophthalmology at Children’s Hospital of Philadelphia. “As a practicing physician who often speaks with patients and families living with IRDs, these conversations have been, up to now, frustrating in that there has been nothing to offer.”

The late-stage clinical trial showed a statistically significant and clinically meaningful difference between intervention and control participants at one year. Participants maintained the functional vision and visual function improvements demonstrated 30 days after Luxturna administration through their last annual follow-up visit, as measured by bilateral multi-luminance mobility test score change and full-field light sensitivity threshold testing. The multi-luminance mobility test tracks participants’ changes in functional vision over time by testing their ability to navigate a course using individual and both eyes.

Investigators observed no serious adverse events associated with Luxturna or deleterious immune responses. Two ocular serious adverse events related to the surgical procedures were reported. It resulted in foveal thinning and a sustained reduction in visual acuity in one participant. The most common adverse reactions related to Luxturna reported in 10 percent or greater of the combined phase 1 and phase 3 trial participants included conjunctival hyperemia, cataract, increased intraocular pressure, and retinal tear.

The FDA is expected to act on Spark’s application to begin marketing Luxturna by January 12, 2018. The agency has previously granted it Orphan Drug, Breakthrough Therapy and Rare Pediatric Disease designations.

October 16, 2017

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