Mon. 22 Nov 2021, 6:31am ETBenzinga
In: News, FDA

If approved, beti-cel will be the first one-time treatment option to address the underlying genetic cause of disease

Current standard of care relies on regular red blood cell transfusions and iron management that carry the risk of progressive multi-organ damage and increased risk of morbidity and mortality

FDA set PDUFA date of May 20, 2022