FDA approves first AAV vector gene therapy – LUXTURNA

Fri, 2017 / 12 / 22

LUXTURNA is the first adeno-associated virus vector-based gene therapy, which has been approved by the FDA in the U.S. this week as a promising and as the only pharmacologic option available for the treatment of patients with vision loss due to IRD caused by mutations in the RPE65 gene. This summer, Spark Therapeutics also submitted the Market Authorization Application for LUXTURNA to the European Medicines Agency (EMA) which is currently under evaluation.

RPE65-mediated inherited retinal dystrophy (IRD) is a rare disease, which leads to a decreased ability to perceive light and central vision impairment as well as to a declining visual acuity. During disease progression, the symptoms can extremely limit activities of daily living, especially for the mostly young patients and in worst cases it can result in total blindness. So far, there is no pharmacologic treatment option available or approved.

LUXTURNA as a one-time gene therapy for the treatment of RPE65-mediated IRD is indicated for children (≥12months) and adults. During the clinical development program, the administration of LUXTURNA was done by a subretinal injection and was completed within one day. The safety and efficacy of LUXTURNA is shown in two phase 1 studies and one phase 3 study (see Russel et al. 2017 for further information).

The approval of LUXTURNA in the U.S. is considered a major step in the ongoing development of gene therapies for various conditions. Gene therapies will be one of the next BIG THINGs in the pharmaceutical industry in the next decade. Therefore, new ways of benefit assessments for these kind of therapies as well as new pricing and reimbursement strategies can and should be developed (please view the blog Gene therapy in Germany – Innovation vs. Reimbursement).

BY Prof. Matthias P. Schönermark, M.D., Ph.D., Founder and Managing Director, SKC Beratungsgesellschaft mbH

Sources:
Spark - FDA Approves Spark Therapeutics’ LUXTURNA
FDA - FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss
Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet (London, England). 2017;390(10097):849-60.