FDA Approves Gene Therapy for an Inherited Disease

[Michael Oberdorfer]

A number of news sources have reported that the Food and Drug Administration has approved the first gene therapy to treat an inherited disease; a rare form of vision loss, Leber Congenital Amaurosis. The therapeutic agent is Luxterna, a genetically modified virus that restores vision by ferrying a normal copy of the RPE65 gene to retinal cells using a AAV viral vector.

The gene therapy was developed by Spark Therapeutics. On the occasion of the approval, FDA Commissioner Scott Gottlieb stated “We’re at a turning point when it comes to this novel form of therapy and at the FDA, we’re focused on establishing the right policy framework to capitalize on this scientific opening.”

Concerns regarding the safety of gene therapy have diminished somewhat, but concerns remain regarding the cost of the treatment, which could amount to $1,000,000 for each patient.