FDA panel support gene therapy for rare type of blindess

The FDA panel voted unanimously to approve Luxturna, developed by Spark Therapeutics, to treat inherited retinal diseases caused by probls in a gene called RPE65.The gene is responsible for the production of an enzyme associated with normal vision and retinal disease caused by defects to RPE65 affects between 1,000–2,000 people in the US.{{quote-A:R-W:450-Q: Results of clinical trials have found 93% of the participants reported improvent in functional vision. }}Roughly half of those affected become legally blind by the age of 16 and all are legally blind by the age of 34, while most progress to complete blindness.Results of clinical trials have found 93% of the participants reported improvent in functional vision, which was measured by their ability to navigate through various controlled levels of light.The FDA panel decision followed scientific presentations from Spark, along with testimonies from participants regarding the impacts of the trial treatments.Spark CEO Mr Jeffrey Marrazzo refused to disclose what the company would charge for the treatment, but told Reuters a fair comparison would be similar drugs that have been developed for ultra-rare diseases such as Hunter Syndrome, Pope Disease and Paroxysmal Nocturnal Hoglobinuria.The cost of those drugs ranges from US$300,000–600,000 (AU$392,000–783,000) a year.If approved, analysts expect Luxturna could generate annual sales of around $400 million (AU$522 m) by 2021.The company’s shares increased by around 160% over the past 12 months in anticipation of the FDA’s approval for Luxturna, reaching a high of US$91 (AU$118.80) at the end of Septber.The FDA is not bound to follow the recommendations of its advisers, but it usually does. A decision is expected to be announced on January 12 next year.