FDA approves first gene therapy targeting rare form of inherited blindness

A first-of-its-kind gene therapy received approval from the Food and Drug Administration on Tuesday to treat a rare, inherited form of childhood blindness.

The FDA marketing clearance of Spark Therapeutics’s Luxturna is historic for scientific and financial reasons. Luxturna is the first gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.

A Spark spokesman said the company will not disclose the Luxturna price tag until early January. Wall Street analysts expect the gene therapy to command a $1 million price tag — another first, but not necessarily a welcome one. At a time when drug prices are coming under intense scrutiny, Spark will need to convince insurers, politicians, and pharma critics that the benefit to patients offered by Luxturna justifies its high cost.

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