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FDA approves Pfizer’s first gene therapy for rare inherited bleeding disorder


The agency greenlighted the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B.

Notably, some health experts have said that high costs and logistical issues, among other factors, have limited the uptake of Hemgenix and another approved gene therapy for the more common hemophilia A. Here are Thursday's biggest analyst calls: Nvidia, Meta, Tesla, IBM, UPS, Five Below, Amazon, TJX Companies & more Here's where to invest $1 million right now, according to the pros Forget Nvidia: Morgan Stanley says Intel's much-hyped AI chip will boost 3 global stocks These 5 stocks will power the AI revolution as data centers spread and electricity demand doubles, says Bank of America Earnings playbook: Your guide to trading a huge week of reports, including Meta Platforms The agency greenlighted the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B. Pfizer said the treatment will be available by prescription to eligible patients this quarter and has a hefty $3.5 million price tag before insurance and other rebates.

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