Politico December 8, 2023
The FDA approved the landmark treatment on Friday. It’s expected to cost more than $1 million.
The Food and Drug Administration’s approval Friday of a first-of-its-kind gene-editing therapy offers hope to patients with sickle cell disease — a rare, but debilitating, genetic disorder that disproportionately affects Black people.
About 100,000 Americans, and millions worldwide, suffer from the disease, in which a defective gene alters blood cells and causes extreme pain. The FDA approved the treatment, which requires a lengthy hospitalization and destroys patients’ fertility, for patients as young as 12 with no upper age limit. The agency expects doctors to prescribe it for people with severe forms of the disease.
The price tag for the one-time treatment will likely be...
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