FDA approves gene therapy to cure form of inherited blindess

21 December 2017

In a decision that promises to further unlock the potential of gene therapy, the Food and Drug Administration yesterday approved a DNA-altering medication that can reverse an inherited form of progressive blindness.

The FDA's nod makes Luxturna, a treatment for retinal dystrophy caused by a specific genetic mutation, the first gene therapy approved for use in the US to treat a genetic disease other than cancer.

The announcement yesterday, comes as the third time in five months that the drug safety agency has allowed a gene therapy, a form of treatment with a long and inconsistent safety history in the US market. Kymriah, which treats a form of leukemia, was the first therapy to be approved in August.

The agency followed it up in October, clearing a second gene-based treatment called Yescarta to treat a form of lymphoma.

''Gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,'' Dr Scott Gottlieb, the FDA's commissioner, said yesterday Los Angeles Times reported. ''We're at a turning point when it comes to this novel form of therapy.''

The agency would release guidelines in the coming year to speed the evaluation and approval of additional gene therapy treatments for a range of diseases so that the country may ''capitalize on this scientific opening,'' he added.

The newest gene therapy, known to scientists as voretigene neparvovec-rzyl, will be used to treat children and adults suffering from an inherited form of vision loss. It usually starts in childhood or adolescence and progresses to complete blindness in adulthood.

The disease is caused when individuals inherit two faulty copies of the RPE65 gene, one from each parent. The US has between 1,00 and 2,00 such people. Luxturna replaces the faulty genes with normal versions, thus eliminating the mutations' harmful effects.

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