bluebird bio announced this week that the FDA will not be scheduling an advisory committee meeting for its sickle cell disease drug, lovo-cel.
Sickle cell disease has seen limited treatment options, with only four approved therapies that do not address the genetic cause. Lovo-cel is designed as a one-time treatment and introduces a functional gene copy to produce adult hemoglobin.
When the agency accepted bluebird's BLA, it granted it priority review status, Orphan Drug, Fast Track, Regenerative Medicine Advanced Therapy, and Rare Pediatric Disease indications, and set a PDUFA goal date of December 20, 2023.
Throughout the journey of the lovo-cel BLA submission, bluebird bio faced challenges. Initially aiming for a Q1 2023 submission, the company had to navigate manufacturing refinements and provide requested comparability data. While awaiting feedback from the FDA on CMC, bluebird ended up postponing its submission until late April.
The drug’s efficacy and safety have been highlighted by the HGB-206 study Group C cohort, which included 36 patients. Safety data from the entire lovo-cel program provided the longest follow-up among all gene therapy programs for SCD.
According to bluebird CEO Andrew Obenshain, the company anticipates an FDA decision by the end of this year.