FDA Committee Unanimously Recommends Approval for Spark’s RPE65 Gene Therapy – Final Decision Due in January 2018
Spark Therapeutics has taken a major step closer to gaining marketing approval for its vision-restoring gene therapy for people with RPE65 mutations causing Leber congenital amaurosis (LCA) and retinitis pigmentosa. At the conclusion of a public hearing on October 12, 2017, an advisory committee comprised of FDA-selected experts voted unanimously – 16 to 0 – to recommend approval. The FDA is due to make a final decision on marketing approval for the treatment, known as voretigene neparvovec, by January 12, 2018.
The event held at FDA headquarters included the presentation of trial results from Spark representatives, as well as compelling testimony from patients, family members, and industry stakeholders.
Twenty-four-year-old Katelyn Corey told hearing attendees that before receiving the treatment, her constant adaptation to dwindling vision didn’t leave time for much else in her life. But her circumstances changed dramatically in December 2013, after she received the RPE65 gene therapy in Spark’s Phase III clinical trial.
“Within days, I could see vibrant colors. I could even see the Philadelphia City Hall clock tower at night,” she said. “Also, I can go to a restaurant and see everything by candlelight, and I can see stars in the night sky.” Katelyn recently earned a master’s degree in epidemiology and works as a research analyst for the U.S. Department of Veterans Affairs.
Eleven-year-old Cole Carper, the youngest speaker at the hearing, said he loves playing with Legos now that he has better vision thanks to the gene therapy. His 13-year-old sister, Caroline, who was also in Spark’s Phase III study, enjoys reading print books instead of Braille. She’s also preparing for a role in the play “Shrek”- something her mom, Ashley, said would have been very difficult before treatment.