FDA panel recommends Luxturna gene therapy

Treatment for Leber congenital amaurosis gets green light from US Food and Drug Administration experts


A gene therapy for the treatment of the inherited eye disease, Leber congenital amaurosis (LCA), is a step closer to approval by the US Food and Drug Administration (FDA).

The FDA’s Cellular, Tissue and Gene Therapies Advisory Committee unanimously recommended approval of Luxturna, developed by gene therapy company Spark Therapeutics. The FDA has until 12 January 2018 to approve the treatment.

There are estimated to be 20 different types of LCA, each caused by a different genetic mutation. Luxturna targets one specific mutation in the RPE65 gene.

A Spark Therapeutics spokesperson highlighted the clinical development programme for the treatment included the first randomised, controlled phase three trial that has ever been conducted for a genetic disease.

Luxturna is a one-off treatment that involves injections of healthy RPE65 genes into the retina.

A clinical trial programme involving 41 patients from the ages of four to 44 with mild to advanced LCA showed “statistically significant and clinically meaningful” difference between those who received the treatment and controls.

Measures taken into account included a bilateral multi-luminance mobility test and full-field light sensitivity threshold.

Data from the clinical programme shows that Luxturna can be effective for up to four years.