Panel recommends Luxturna FDA approval for rare form of blindness

Luxturna FDA approval news : Luxturna (voretigene neparvovec), a gene therapy from Spark Therapeutics is closing in towards an FDA approval for treating blindness due to an inherited retinal disease (IRD), following the recommendation from an advisory panel of the US drug regulator.

An adeno-associated viral (AAV) vector gene therapy, Luxturna has been recommended to be used in the US, unanimously by the 16-member Cellular, Tissue and Gene Therapies Advisory Committee of the US Food and Drug Administration (FDA).

Luxturna is a gene therapy to be given just once, which is now a step away from being implemented for the treatment of patients who had lost their vision because of confirmed biallelic RPE65-mediated inherited retinal disease.

Luxturna FDA approval has been recommended based on the positive results of a phase 3 gene therapy clinical trial.

During the late stage trial, Spark Therapeutics was successful in demonstrating Luxturna was good enough to maintain the functional vision and boosting visual functions of the enrolled patients, after 30 days of its administration.

Luxturna gene therapy increased the ability of 93% of the participants in safely navigating obstacles in bad light conditions, according to the pharma company in Philadelphia.

Katherine High, the head of the research and development division of Spark Therapeutics commented: “The clinical program for LUXTURNA includes patient data that show efficacy for up to four years on endpoints including bilateral multi-luminance mobility test (MLMT) score change and full-field light sensitivity threshold (FST) testing, with observation ongoing.”

RPE65-mediated inherited retinal disease is an uncommon type of vision loss. As of now, no pharmacologic treatment options are available for patients.

Patients with RPE65-mediated inherited retinal disease frequently suffer from night blindness (nyctalopia). In most of the patients, the RPE65-mediated inherited retinal disease has the potential to make them completely blind.

Luxturna phase 3 Principal Investigator Albert M. Maguire said: “There currently are no pharmacologic treatment options for people living with RPE65-mediated IRD, who in most cases progress to complete blindness.

“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.”

Intended to be given only once, Luxturna is given intravenously or injected into target tissue. Viral vector particles comprising the correct copy of the RP65 gene are delivered to retinal cells through the Luxturna gene therapy. As a result, viral vector particles repair the ability of retinal cells to generate the deficient enzyme.