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.

Dr. Gottlieb further commented, “Today’s approval marks another first in the field of gene therapy—both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss….The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses.”

Next year, the FDA aims to start rolling out a series of what it calls disease-specific guidance documents on the development of specific gene therapy products. These will “lay out modern and more efficient parameters—including new clinical measures—for the evaluation and review of gene therapy for high-priority disease where the platform is being targeted,” Dr. Gottlieb stated. “We’re at a turning point when it comes to this novel form of therapy, and, at the FDA, we’re focused on establishing the right policy framework to capitalize on this scientific opening.” (ontinueReading