New treatment for genetic retinal disease is first gene therapy approved in the USA

Written by RegMedNet

LUXTURNA is the first pharmacologic treatment indicated for inherited retinal disease and the first adeno-associated virus vector gene therapy approved in the USA.

In a USA first, the FDA has approved LUXTURNA for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Developed by Spark Therapeutics (PA, USA), LUXTURNA is the first gene therapy to be approved in the USA. It was approved under Priority Review having previously received orphan drug and breakthrough therapy designations. Spark Therapeutics’ Marketing Authorization Application for LUXTURNA is currently under review with the European Medicines Agency (EMA), which previously granted LUXTURNA orphan product designations.

“Today’s landmark approval of LUXTURNA is a moment decades in the making for the field of gene therapy, the inherited retinal disease (IRD) community, and most importantly, patients with biallelic RPE65 mutation associated retinal dystrophy who now have the option to seek treatment,” commented Jeffrey D. Marrazzo, chief executive officer at Spark Therapeutics.

Jean Bennett, the F.M. Kirby Professor of Ophthalmology in the Perelman School of Medicine at the University of Pennsylvania and Penn’s Scheie Eye Institute (PA, USA), continued: “I believe that the success of the LUXTURNA clinical development program will pave the way for the development of other gene therapies, that may help the millions of patients with genetic diseases who currently have limited or no treatment options.”

In a Phase III clinical trial with a cohort of 31, there was shown to be a statistically significant improvement in the intervention group (n=21) compared with the control group (n=10). Although visual acuity was not significantly improved, there were improvements in the median bilaterial multi-luminance mobility test (MLMT) score and the median first-treated eye MLMT score. The MLMT was designed to assess participants’ ability to navigate a course accurately and at pace under seven levels of illumination, ranging from the equivalent of a brightly lit office (400 lux) and a moonless summer night (1 lux).

LUXTURNA will be manufactured at Spark Therapeutics’ manufacturing center based in West Philadelphia and administered at selected centers in the US after surgeons have received the requisite training on the administration procedure. The price of each treatment has yet to be released but Spark Therapeutics has committed to assisting eligible and enrolled patients navigate the insurance process.