"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 have been severely disrupted by the disease," said Nicole Verdun, director of the Office of Therapeutic Products in the FDA's Center for Biologics Evaluation and Research.
The Institute for Clinical and Economic Review, which advises payers, estimated an appropriate price for Casgevy would be between $1.35 million and $2.05 million per patient.
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