(National Human Genome Research Institute, NIH)
19 December 2017. A therapy using transfers of healthy genes to treat a rare form of inherited vision loss was approved today by the U.S. Food and Drug Administration. The gene therapy is made by Spark Therapeutics in Philadelphia, which FDA says is the first of its kind approved by the agency, in this case for both adults and children.
The gene therapy, known generically as voretigene neparvovec, or by its brand name Luxturna, is approved to treat biallelic RPE65 mutation-associated retinal dystrophy, which results from a mutation in both alternative forms of 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. From 1,000 to 2,000 individuals in the U.S. have this condition.
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, 29 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, and for the longer-term participants, the gains remained stable for up to 3 years. No serious adverse effects, including dangerous immune responses, from the treatments were reported. Most adverse effects in the eyes were mild in severity.
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.
FDA says Luxturna is the first gene therapy approved in the U.S. directly administered to patients and treating a disorder caused by mutations in a specific gene. FDA Commissioner Scott Gottlieb says in a statement that the agency is preparing new policies to take advantage of this initial experience with gene therapies. “Next year,” notes Gottlieb, “we’ll begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products to lay out modern and more efficient parameters, including new clinical measures, for the evaluation and review of gene therapy for different high-priority diseases where the platform is being targeted.”
As reported by Science & Enterprise in July 2017, FDA granted Spark Therapeutics a priority review for Luxturna, with a decision date no later than January 2018 for approval. Priority review accelerates the time for review of new drug or biologics applications to 6 months.
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