FDA confirms positive impact of Spark Therapeutics gene therapy

FDA confirms positive impact of Spark Therapeutics gene therapy on vision

The U.S. FDA has confirmed short term benefits with Spark Therapeutics’ gene therapy for vision in a rare, inherited form of blindness. As per the preliminary review conducted by FDA team, the short term benefits have been noticed but it is still unclear if the gene therapy offers long term vision benefit as well. The therapy developed by Spark Therapeutics would be the first gene therapy approved in the United States for an inherited condition.

The FDA review has been shared on the official website. Another meeting of advisers will be held before final approval for Spark Therapeutics therapy. However, reports suggest that the approval is highly likely in this case.

As per the FDA report, inherited retinal diseases are a group of rare conditions caused by mutations in one or more than 220 different genes, including one known as RPE65, which tells cells how to produce a crucial enzyme needed for normal vision.

Spark Therapeutics conducted clinical trials and the company reported that 93 percent of participants witnessed improvement in vision. The therapy named Luxturna, or voretigene neparvovec, has been found effective in people suffering from Leber congenital aumaurosis, whose disease was mediated by defects in the RPE65 gene.

Spark Therapeutics stock has jumped by nearly 150 percent over the last one year. The stock was trading strong after FDA posted preliminary review on its website.