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.
U.S. health advisers are reviewing a novel treatment for a rare form of inherited blindness, potentially setting the stage for the launch of a groundbreaking new genetic medicine.
The Food and Drug Administration panel will vote Thursday on whether to recommend approval of Philadelphia-based Spark Therapeutics’ treatment, which aims to improve vision in some patients with hereditary blindness by replacing a defective gene that affects vision.
The FDA is not required to follow the group’s recommendation, but such deliberations are generally the final step in the agency’s decision-making process. The FDA has until mid-January to decide. If approved, it would be the first gene therapy in the U.S. for an inherited disease, offering hope to patients with a variety of related conditions.
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