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takyon [soylentnews.org] writes:

The Food and Drug Administration [fda.gov] (FDA) has approved a gene therapy [scientificamerican.com] to treat RPE65 [wikipedia.org]-mutation associated retinal dystrophy. But it will be expensive:

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 [statnews.com] 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.

Also at NPR [npr.org].

Previously: FDA Approves a Gene Therapy for the First Time [soylentnews.org]
FDA Committee Endorses Gene Therapy for a Form of Childhood Blindness [soylentnews.org]

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