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FDA Grants Priority Review for Gene Therapy

Adeno-associated virus

Electrostatic image of adeno-associated virus (National Institute of General Medical Sciences, NIH)

17 July 2017. A biotechnology company making gene therapies to treat rare diseases says the Food and Drug Administration granted an accelerated review for its treatment of an inherited eye disorder. FDA accepted the biologics license application for voretigene neparvovec, a gene therapy developed by Spark Therapeutics Inc. in Philadelphia, to treat a type of inherited retinal disease, and gave the therapy priority review, which will make it eligible for approval in January 2018.

Voretigene neparvovec, which will have the brand name Luxturna, is designed to treat vision loss due to an inherited retinal disorder resulting from mutations in the RPE65 gene expressed in the retina. The mutations prevent correct encoding of a protein that converts light entering the eye into electrical signals transmitted to the brain, making sight possible. Without this protein functioning properly, visual cycles are disrupted and impaired, leading to blindness.

Spark’s gene therapies are delivered with engineered adeno-associated viruses, benign microbes designed to deliver genetic material into the cells of patients with defective genes. The company says its facilities have the capability to manufacture clinical-grade adeno-associated viruses, used so far with hundreds of patients in several clinical trials.

Voretigene neparvovecis is injected once per eye. The therapy was evaluated in two early-stage and one late-stage clinical trials, where the therapies were assessed against a comparison group. In the late-stage trial, participants were rated on a test where they navigate a maze at various levels of light, with those receiving the gene therapies scoring higher than the comparison group. Gains shown after 30 days also continued for a full year. Spark says that when offered after the trial, all of the comparison group participants elected to receive the therapies.

Long-term safety and efficacy continue to be evaluated for individuals receiving the treatment between 2013 and 2015. In the clinical trials of the treatments, no serious adverse effects or harmful immune responses were reported.

FDA grants priority review to new therapies that represent significant improvements in safety or effectiveness in treating or preventing serious disorders compared to current therapies. Priority review accelerates the time for review of new drug or biologics applications to 6 months, but does not reduce time needed for clinical trials or evidence required for the review.

If FDA keeps to that schedule, Luxturna will be eligible for agency approval by January 2018. The company says Luxturna will not only be the first drug treatment for inherited retinal diseases, but also the first gene therapy approved for a genetic disease in the U.S.

A key question for Spark Therapeutics is pricing of the treatments. The industry web site BioPharma Dive notes that the pharma industry has yet to come up with a satisfactory pricing model for gene therapies. As reported in Science & Enterprise in May 2016, a cancer specialist and venture capital executive proposed in the journal Science a pricing scheme for gene therapies covering several factors to assess their value.

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