FDA Approves New Gene Therapies for Sickle Cell Disease Amid Access Concerns

Bluebird Bio's Lyfgenia and Vertex/CRISPR's Casgevy hit the market with high price tags and a shortage of specialists, raising questions about patient access.

• The FDA has approved two new gene therapies for sickle cell disease: Casgevy by Vertex Pharmaceuticals and CRISPR Therapeutics, and Lyfgenia by Bluebird Bio.
• Casgevy is priced at $2.2 million, while Lyfgenia is priced at $3.1 million. Bluebird Bio's stock fell by over 40% following the approval and pricing announcement of Lyfgenia.
• Bluebird Bio's CEO, Andrew Obenshain, has defended the price of Lyfgenia, stating that the company plans to offer refunds to insurers if the treatment is not as effective as expected.
• Despite the breakthroughs, there is a concern that most patients will not be able to access these treatments due to their high cost and the shortage of adult hematologists.
• The Sickle Cell Disease Comprehensive Care Act, if passed, would establish a demonstration program in up to 10 states to ensure better access to comprehensive, high-quality outpatient care for people living with sickle cell disease.

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