Children whose vision improved following gene therapy, plus their parents, doctors, and scientists, will speak in front of a Food and Drug Administration panel on Thursday, as the committee decides whether it will recommend approving the therapy.
The FDA has until Jan. 18 to decide if it wants to approve Luxturna, which would be the first gene therapy available in the United States for an inherited disease and the first where a corrective gene is directly given to a patient, The Associated Press reports. Luxturna has been tested on people with Leber congenital amaurosis, who are unable to make a protein needed for the retina due to flaws in the RPE65 gene. They typically are only able to see blurred shapes and bright lights, until they lose their sight all together.
A study, funded by Luxturna's manufacturer, Spark Therapeutics, found that while it does not give patients 20/20 vision, it did improve the vision of nearly everyone who participated in the trials. The company hopes patients would only need one treatment, which involves injecting a modified virus with the corrective gene into the retina. It's not known yet how long the benefits last, but it usually only takes about a month for sight to start to improve. Thanks to the therapy, children have been moved from Braille classrooms to sighted classrooms, and adults who have never held jobs before due to limited sight can now work, Dr. Katherine High, president of Sparks Therapeutics, told AP.