Philadelphia-based Spark Therapeutics recently received unanimous backing from an FDA advisory panel for Luxturna™, a one-shot gene therapy treatment for biallelic RPE65-mediated inherited retinal dystrophy (IRD). The condition affects around 3,500 individuals in the U.S. and Europe and is currently untreatable. The FDA is now expected to decide whether to greenlight the therapy by January 12, 2018. This marks a pivotal moment not only for Spark Therapeutics, but for all gene therapies currently in development.
The FDA panel based its decision on data from the primary phase 3 trial of Luxturna, in which 93% of participants showed some improvement in their functional vision as measured by the bilateral multiluminance mobility test. Luxturna was subsequently granted a rare priority review status, indicating that the treatment is likely to be approved in the next few months. The market reacted positively to the news, with Spark Therapeutics shares climbing as the company moves a step closer to becoming America’s first FDA-approved gene therapy. It should be noted that there is a small chance that the FDA goes against the advisory panel and rejects the treatment, as agency reviewers have questioned the clinical endpoints used in the trial and long-term effects of the treatment remain unknown.