4). Voretigene neparvovec-rzyl will be a successful pioneer for gene therapies
Voretigene neparvovec-rzyl (âvoretigene,â Luxturna, Spark Therapeutics) is the first gene therapy for a genetic disease, first and only pharmacologic treatment for an inherited retinal disease (IRD), and first adeno-associated virus (AAV) vector gene therapy approved in the United States. With all these first-in-class titles, there will be new challenges ahead for this innovative advance in medicine. Drug cost reimbursement, insurance coverage, distribution, and marketing/education will be the critical topics that will require creative solutions.
However, I believe voretigene is more than ready for the battle and it will be very successful in pioneering this new frontier. The company has already put into motion an organized structural model to address these pivotal issues and they have the skills to deliver this exciting new treatment to patients across our country. They will be making history and writing the blueprint for which the entire class of gene therapies will utilize in the future.