Nearly all patients treated so far have been relieved of the blood-clogging crises of the disease.
Sickle-cell disease has afflicted people of African descent for generations, causing agony and death among those who inherit its deformed red blood cells. The end of that unhappy inheritance inches closer on Tuesday—as the Food and Drug Administration asks a panel of experts to weigh in on what’s likely to be the country’s first-approved genetic treatment for the disorder.
FDA documents posted online in preparation for the meeting don’t reveal great concerns at the agency over exa-cel. After reading them last week, William Blair analyst Sami Corwin wrote that they set up “a near-best-case scenario” for the meeting—and for exa-cel’s approval as a treatment for sickle cell, and another red blood cell disorder called beta thalassemia whose sufferers require lifelong transfusions.
“Given the strongly positive results,” says the agency’s review document, “FDA does not believe that the study design limitations call the efficacy of exa-cel into question.”
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