Panel backs gene therapy to treat form of blindness
SILVER SPRING, Md. — A potentially groundbreaking treatment for a rare form of blindness moved one step closer to U.S. approval Thursday, as federal health advisers endorsed the experimental gene therapy for patients with an inherited condition that gradually destroys eyesight.
The panel experts that report to the Food and Drug Administration voted unanimously in favor of Spark Therapeutics’ injectable therapy, which aims to improve vision by replacing a defective gene needed to process light.
The vote amounts to a recommendation to approve the therapy. The FDA has until mid-January to make its decision and does not have to follow the panel’s recommendation, though it often does.
If approved, Luxturna would be the first gene therapy in the U.S. for an inherited disease and the first in which a corrective gene is given directly to patients. While the therapy from Spark Therapeutics targets a small group of patients — about 2,000 in the U.S. — experts say it could pave the way for other genetic treatments for a variety of inherited conditions.