bluebird’s Skysona to test market’s appetite for $3M rare disease therapy

FDA’s accelerated approval of bluebird’s gene therapy Skysona will test the market’s appetite for a premium-priced gene therapy that offers curative potential for a rare disease, but comes with a known oncogenic risk.

The agency’s approval late Friday of lentiviral gene therapy Skysona elivaldogene autotemcel (“eli-cel”) to treat cerebral adrenoleukodystrophy (CALD) wasn’t a surprise, as an FDA advisory committee had voted unanimously in June that the treatment’s benefit-risk profile would be acceptable, given the disease’s severity and the lack of other approved therapies...