A treatment that can stop patients from going blind is poised to be the first gene therapy for an inherited condition approved in the U.S., in what would be a major scientific milestone — and also open the door for record-breaking drug prices and novel ways to pay for them.
Spark Therapeutics Inc.’s Luxturna therapy crossed a key hurdle Thursday when it won backing from a group of advisers to the U.S. Food and Drug Administration. After discussing the trial design, treatment procedure and safety profile, the panel of 16 experts voted unanimously that the drug’s benefits outweighed its risks.
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