The Food and Drug Administration has granted full approval to a new gene therapy treatment for sickle cell disease, marking a historic milestone in the fight against the genetic blood disorder. The therapy, developed by Vertex Pharmaceuticals, works by editing a patient's own stem cells to produce functional hemoglobin.
Clinical trial data showed that 94% of patients treated with the therapy were free of vaso-occlusive crises for at least two years following treatment. The one-time procedure is expected to cost approximately $1.8 million per patient.
Patient advocacy groups are urging insurers and government programs to ensure equitable access, noting that sickle cell disease disproportionately affects African American communities.
