Key takeaways:
- FDA approved two CRISPR-based gene therapies, Casgevy and Lyfgenia, for sickle cell disease treatment.
- Both therapies have shown promising results in eliminating sickle cell's characteristic pain crises.
- High costs and accessibility issues may pose challenges for patients, particularly those covered by Medicaid.
FDA Approves Groundbreaking Gene Therapies for Sickle Cell Disease
The U.S. Food and Drug Administration (FDA) approved two cell-based gene therapies, Casgevy and Lyfgenia, for sickle cell disease treatment, marking a significant advancement in the field[1][3]. Casgevy, the first CRISPR therapy approved by the FDA, will cost .2 million, while Lyfgenia is priced at .1 million[2].
Promising Results in Clinical Trials
Both Casgevy and Lyfgenia have shown promising results in clinical trials, eliminating sickle cell's characteristic pain crises, which can cause serious health problems, including death[2]. The therapies have the potential to treat the inherited blood condition for many years, if not a lifetime, offering hope to patients who have had few treatment options.
High Costs and Accessibility Issues
The high costs of these therapies raise questions about access, particularly for a patient population that has historically faced discrimination and barriers to care[2]. While similar to the cost of gene therapies for other rare diseases, Casgevy and Lyfgenia are now among the most expensive medicines on the market.
Accessibility issues may also pose challenges, particularly for Medicaid patients, who make up a significant portion of the eligible population[2][3]. The preparatory chemotherapy regimen can also cause infertility, and while Vertex plans to offer fertility support to commercially insured patients, they are unable to do the same for people in Medicaid[2].
Moving Forward
Gene therapy will likely be available in early 2024, and both therapies are approved for people ages 12 and up with sickle cell disease SS and S-beta-zero-thalassemia[3]. SCDAA encourages gene therapy centers to partner with sickle cell centers to ensure expertise in monitoring for sickle cell organ damage[3].
Health officials are working to ensure equitable access to these breakthrough treatments, and gene therapy treatments are expected to cost around .2 million for Casgevy and an undisclosed amount for Lyfgenia[3][4].
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