FDA approved a gene therapy for an inherited form of blindness


Hereditary retinal dystrophies are a broad group of genetic retinal disorders that are associated with progressive visual dysfunction and are caused by mutations in any one of more than 220 different genes. The FDA’s decision to approve Luxturna follows a unanimous recommendation in its favor by an advisory committee in October.In tests on patients, the treatment often produced dramatic results, restoring the ability of patients to see things they could never see before.


The treatment also enabled patients to read, play sports, ride bicycles and go outside at night by themselves. “Today’s approval marks another first in the field of gene therapy,” said FDA Commission Scott Gottlieb in a statement. In August, the FDA approved Kymriah, the first gene-therapy product to treat a form of leukemia. A second gene-based treatment, called Yescarta, was approved for a form of lymphoma in October. Spark Therapeutics, which makes Luxturna, hasn’t said how much it will charge. But there is a widespread expectation that it could cost at least $1 million to treat each patient.


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