Pharmaceutical Executive December 8, 2023
FDA approval of bluebird bio’s Lyfgenia and Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy marks significant milestone in the treatment of sickle cell disease.
The FDA has approved a pair of landmark treatments for sickle cell disease (SCD) in patients 12 years of age and older, marking the first cell-based gene therapies for SCD. As part of today’s regulatory action, both bluebird bio’s Lyfgenia (lovotibeglogene autotemcel [lovo-cel]) and Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel [exa-cel]) gained approval for the inherited blood disorders, which affect approximately 100,000 people in the United States.1
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives...
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