FDA approved Spark Therapeutics’ gene therapy Luxturna™ (voretigene neparvovec-rzyl) for treating children and adults with the rare inherited blindness disorder biallelic RPE65 mutation-associated retinal dystrophy. Approval of the one-time adeno-associated virus vector (AAV)-delivered gene therapy, which FDA Commissioner Scott Gottlieb, M.D., described in a statement as “a milestone that reinforces the potential of this breakthrough approach in treating a wide-range of challenging disease,” marks the first gene therapy approval for a genetic disease. Spark has not confirmed the cost of treatment, although some commentators are anticipating a $1 million per patient price tag. Luxturna is currently under review by the European Medicines Agency.
FDA clearance of Luxturna marks the third approval by the agency for a gene therapy, but the first for an AAV-based treatment. In August this year, the U.S. regulator cleared Novartis’ cell-based chimeric antigen receptor T-cell (CAR-T) gene therapy Kymriah™ (tisagenlecleucel) for treating refractory or relapsed B-cell acute lymphoblastic leukemia. In October, Kite Pharma’s CAR-T therapy Yescarta™ (axicabtagene ciloleucel) was given the FDA nod for treating refractory or relapsed diffuse large B-cell lymphoma.
