This morning, it was announced that the U.S. Food and Drug Administration (FDA) has approved the gene therapy Luxturna (voretigene neparvovec-rzyl) to treat children and adult patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.
In October, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee met to review data presented by Spark Therapeutics and voted 16-0 in favor of the drug. Today’s regulatory action marks the second approval for a gene therapy in the United States. Kymriah was approved for the treatment of acute lymphoblastic leukemia (ALL) in August.
“Today’s approval marks another first in the field of gene therapy — both in how the therapy
