First Gene Tx for Inherited Retinal Disease Gets Thumbs Up from FDA Committee

Luxturna, an investigational, potential one-time gene therapy
Luxturna, an investigational, potential one-time gene therapy

The Food and Drug Administration (FDA) Cellular, Tissue and Gene Therapies Advisory Committee unanimously (16–0) recommended the approval of Luxturna (voretigene neparvovec; Spark Therapeutics), an adeno-associated viral vector gene therapy, for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD).

“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 M. Maguire, MD, 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. Today's advisory committee vote is an important step closer to the day that discussion can include potentially treating the blindness caused by their IRD.”

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The recommendation was based on data from the Luxturna clinical development program which included a Phase 3 trial in patients 4–44 years of age. The primary endpoint of the study was mean bilateral multi-luminance mobility testing (MLMT) score change. A statistically significant and clinically meaningful difference was seen in patients administered Luxturna (n=21) versus the control group (n=10) at 1 year (difference of 1.6; 95% CI, 0.72, 2.41; P=0.001).

In addition, patients who received Luxturna showed a marked difference compared to control across 2 secondary endpoints: full-field light sensitivity threshold (FST) testing averaged over both eyes (P=0.001) and the mobility test score change for the first injected eye (P=0.001). 

A third secondary endpoint, the change in visual acuity averaged over both eyes, was not statistically significant between the Luxturna group and control group (P=0.17). 

With regard to safety, no serious adverse events or deleterious immune responses were observed in patients administered Luxturna.

While not bound by the Advisory Committee's recommendation, the FDA takes its guidance into consideration. Luxturna is under Priority Review, with a Prescription Drug User Fee Act date of January 12, 2018.

For more information visit Sparktx.com.