STAT+: Pharmalittle: FDA approves a gene therapy for a rare blood disorder; pharmacy chains ordered to pay $650 million over opioid crisis
Hello, everyone, and how are you today? We are doing just fine, thank you, courtesy of clear blue skies and cool breezes circling about the relatively quiet Pharmalot campus. We are busy, though, firing up the coffee kettle, since a cup or three of stimulation is in order. Our choice today is maple bourbon. And of course, we are gathering items of interest to help you get started on your journey this morning. On that note, we hope you have a meaningful and productive day. As always, do keep in touch. We relish your insights and tips. …
The U.S. Food and Drug Administration approved a new treatment that delivers a potentially permanent, genetic fix for patients with the inherited blood disorder beta thalassemia — and quite possibly a financial lifeline for its manufacturer, Bluebird Bio, STAT tells us. The therapy, Zynteglo, is just the third gene therapy approved by the FDA, and the first to target a chronic blood disease. It uses an engineered virus to deliver a gene fix into the bone marrow of patients. Zynteglo will cost $2.8 million per patient, making it one of the most expensive medicines ever sold in the U.S. The price does not include the cost of the lengthy hospitalization for patients getting the therapy.
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