An advisory board at the Food and Drug Administration today endorsed the first gene therapy for an inherited disorder — a rare condition that causes a progressive form of blindness that usually starts in childhood.
The recommendation came in a unanimous 16-0 vote after a day full of hearings that included emotional testimonials by doctors, parents of children blinded by the disease and from children and young adults helped by the treatment.
The treatment will now progress to a final decision from the FDA and, if approved, will be the first gene therapy legally available in the United State for an inherited disorder. The FDA is under no obligation to follow the advisory board’s recommendation but usually does.
The treatment, which will be marketed as Luxturna, fixes a mutation in the RPE65 gene. It involves a single treatment to each eye, which introduces genetically engineered virus particles carrying a corrected version of the mutated gene. Spark Therapeutics, the treatment’s developer, estimates that 6,000 people around the world could benefit from this treatment. More than 90 percent of the patients treated in the study showed some improvement in eyesight within just a few days of treatment.
This is a huge step forward for the field of gene therapeutics. “[O]n multiple fronts, it’s a first and ushers in a new era of gene therapy,” assistant professor of ophthalmology at the Oregon Health and Science University, Paul Yang, told NPR.
Alone, this treatment could also be applied to other formally incurable genetic eye diseases. “There are a lot of retinal diseases like this, and if you added them together it’s a big thing because they are all incurable,” says lead researcher Albert Maguire in an interview with NPR before the hearing.