Accelerated Approval For Bluebird’s Skysona Gives Teeth To US FDA Data Questions

The US FDA-approved labeling of bluebird bio’s gene therapy Skysona (elivaldogene autotemcel) stayed consistent with the agency’s position at a June advisory committee meeting, but with one notable twist: the gene therapy received accelerated approval, an option that was not mentioned in the committee briefing documents or meeting transcript.

Neuroscience is not a field that has had much use for accelerated approval, with Biogen, Inc.’s Aduhelm standing as a cautionary outlier