Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses his company’s first approved product, Luxturna — a one-time gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.
The U.S. Food and Drug Administration (FDA) approved Luxturna in December, 2017. It is the first FDA-approved gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease (IRD) and the first adeno-associated virus (AAV) vector gene therapy approved in the United States.
A genetic test is the only way to verify the gene mutation(s) that is the underlying cause of an IRD, including those associated with biallelic RPE65 mutations.
