comscore FDA advisers endorse gene therapy for blindness | Honolulu Star-Advertiser

FDA advisers endorse gene therapy for blindness


    Dr. Barry Byrne listens to testimony concerning the approval of a potentially breakthrough drug for a form of blindness during a meeting of the The Cellular, Tissue and Gene Therapies advisory committee today at the FDA in Silver Spring, Md.

SILVER SPRING, Md. >> U.S. health advisers have endorsed an experimental approach to treating inherited blindness, setting the stage for the likely approval of an innovative new genetic medicine.

A panel of experts to the Food and Drug Administration voted unanimously in favor of Spark Therapeutics’ injectable therapy, which aims to improve vision in patients with a rare mutation that gradually destroys normal vision. The vote amounts to a recommendation to approve the therapy.

The FDA has until mid-January to make its decision. The agency does not have to follow the panel’s recommendation, though it often does.

If approved, the treatment called Luxturna would be the first gene therapy in the U.S. for an inherited disease, offering hope to patients with a variety of genetic disorders.

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