About 20,000 people in the US are thought to have a severe enough form of the disease to potentially qualify for a treatment like this. ![]() “We are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today.”Ĭasgevy will cost $2.2 million for the one-time treatment, Vertex said in a regulatory filing, while Lyfgenia will cost $3.1 million, bluebird said in a news release.Ī drug pricing analysis group, the Institute for Clinical and Economic Review, had suggested that a price of $1.35 million to $2.05 million for each therapy would have made them cost-effective and encouraged the companies to consider pricing the medicines at the lower end of that range to enable broader access.įor many in the sickle cell community, the approvals have been a long time coming. The disease afflicts about 100,000 people in the US, including an estimated 1 of every 365 Black babies born, according to the US Centers for Disease Control and Prevention, and has long been considered neglected by the pharmaceutical industry. Nicole Verdun, director of the FDA’s Office of Therapeutic Products within its Center for Biologics Evaluation and Research. “Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need,” said Dr. Both were cleared for people 12 and older with histories of vaso-occlusive crises, painful events caused by the disease. The medicines, called Casgevy and Lyfgenia, are potential cures for people with sickle cell, a debilitating and life-shortening inherited red blood cell disorder that disproportionately affects African Americans.Ĭasgevy is the CRISPR-based treatment, made by Vertex Pharmaceuticals and Crispr Therapeutics Lyfgenia, made by bluebird bio, uses an older gene therapy approach. (Tammy Ljungblad/The Kansas City Star/Tribune News Service via Getty Images) Tammy Ljungblad/The Kansas City Star/Tribune News Service via Getty ImagesįDA considers first CRISPR gene editing treatment that may cure sickle cell A worker from the Community Blood Center hangs a bag of blood during a transfusion for Kevin Wake at the Sickle Cell Center at University Health on March 7, 2023.
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