FDA Committee Backs Gene Therapy Candidate

A US Food and Drugs Administration (FDA) advisory committee has given its unanimous support to what should become the first gene therapy for genetic disease.

Voretigene neparvovec (Luxturna), a proposed gene therapy treatment for biallelic RPE65-mediated inherited retinal disease (IRD), received a 16-0 favorable vote from an FDA committee, 3 months prior to the administration’s expected ruling date.

Luxturna was given Priority Review status by the FDA in June, accelerating its Biologics License Application (BLA) process by 4 months. The designation also indicated its significant potential for treatment progression — IRD is a rare ophthalmologic disease with limited treatment options.

Jeffrey D. Marrazzo, chief executive officer of Spark Therapeutics, noted in June that Luxturna’s progression at the time was an “important development for people living with RPE65-mediated IRD.”

“As we work closely with the FDA in the months ahead, we will remain steadfast in our commitment to bring this important investigational therapy to people living with RPE65-mediated IRD who currently have no pharmacologic treatment options,” Marrazzo said.

Luxturna proved its efficacy and safety in a phase 3 trial involving 41 patients with vision loss. The patients, aged from 4 to 44 years old, had clinically-confirmed bialleic RPE65 mutations and sufficient viable retinal cells.

The trial population to receive the therapy reported statistically significant and clinically meaningful improvements versus control in the mean bilateral multi-luminance mobility testing (MLMT) score, at 1 year.

Patients to receive the therapy also reported improvements in full-field light sensitivity testing, and mobility test change score for the first injected eye.

Luxturna’s FDA BLA ruling date is scheduled for January 12, 2018.