FDA Panel Recommends Approval of Experimental Gene TherapyTreatment

Experimental Gene Therapy Treatment for Leber's Congenital Amaurosis


The CBS Evening News (10/12, story 10, 1:50, Mason) reported, “An FDA advisory panel today recommended approval of an experimental treatment for” Leber’s congenital amaurosis (LCA), “a rare form of blindness.”

In a segment on the NBC Nightly News (10/12, story 9,. 1:50, Holt) , NBC News medical correspondent Dr. John Torres explained that physicians “inject directly into the eye a normal gene to fix the defective gene.” Torres said scientists “believe one day, this approach could treat other conditions caused by genetic defects” such as some “cancers, heart disease and Parkinson’s.”

The Washington Post (10/12, McGinley) reports the committee recommended the therapy, Luxturna, be made the first gene therapy to be approved in the US for an inherited disorder. Spark Therapeutics produces the therapy, which “does not give patients perfect vision but does produce substantial improvements, researchers said.” The treatment “represents what is thought of as true gene therapy, in which a functional gene does the job of a defective one.”

Bloomberg News (10/12, Chen, Tracer) reports the treatment’s approval “would be a major scientific milestone – and also open the door for record-breaking drug prices and novel ways to pay for them.” The article says Luxturna may face pricing impediments to comply with Medicaid, noting CMS said it “is committed to further exploring the development of innovative payment arrangements for new and potentially life-saving treatments.”

Reuters (10/12, Clarke) reports the FDA panel “voted unanimously in favor of the treatment.”

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