This will help many people.
The US Food and Drug Administration (FDA) granted approval for two groundbreaking gene-based treatments for sickle cell disease, marking a monumental stride in medical advancements. This development introduces a new era in treating genetic conditions, notably leveraging CRISPR, a gene-editing technique, for the first time in therapy. The medications, Casgevy and Lyfgenia, hold promise as potential cures for sickle cell, a hereditary red blood cell disorder that disproportionately affects African Americans, causing debilitating symptoms and shortening life expectancy.
Casgevy, developed by Vertex Pharmaceuticals and Crispr Therapeutics, utilizes CRISPR technology, while Lyfgenia, created by bluebird bio, employs an older gene therapy method. Both treatments received clearance for individuals aged 12 and above with histories of vaso-occlusive crises, the painful events triggered by sickle cell disease.
Dr. Nicole Verdun, Director of the FDA’s Office of Therapeutic Products, expressed enthusiasm about advancing treatments in a condition marked by significant unmet medical needs. She emphasized the approval of two cell-based gene therapies as a crucial step forward, particularly for individuals whose lives have been severely affected by this disease.
However, the cost of these groundbreaking treatments has sparked discussions. Casgevy is priced at $2.2 million for a single treatment, according to Vertex, while Lyfgenia comes with a $3.1 million price tag, as per bluebird’s release. A drug pricing analysis group recommended a lower pricing range between $1.35 million and $2.05 million per therapy to ensure cost-effectiveness and broader accessibility.
The approvals carry immense significance for the sickle cell community, which has long awaited advancements in treating this disease. Approximately 100,000 individuals in the US, including 1 in every 365 Black babies born, are affected by sickle cell disease, as reported by the Centers for Disease Control and Prevention (CDC). This approval signals a shift in focus towards a condition historically neglected by the pharmaceutical industry, potentially benefiting about 20,000 individuals with severe forms of the disease.
Mayo Clinic bioethicist Megan Allyse acknowledges the significance of this newfound attention to sickle cell therapy development but raises concerns about the accessibility of a treatment priced at over $2 million. While the spotlight on sickle cell disease is welcomed, the accessibility of such cutting-edge treatments remains a pertinent issue.
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