$3.5-Million Hemophilia Gene Therapy Is World’s Most Expensive Drug

Image caption: Haemophilia is a genetic condition that affects the formation of blood clots (pictured). Image credit: Micro Discovery / Getty Images. Article by Miryam Naddaf. Scientific American – December 9, 2022. Originally published in Nature – December 6, 2022.

On 22 November, the US Food and Drug Administration (FDA) approved the first gene therapy for the genetic blood-clotting disorder haemophilia B—a one-time treatment that costs US$3.5 million. Hemgenix—developed by the pharmaceutical company CSL Behring, based in King of Prussia, Pennsylvania—uses a modified virus to deliver a gene to the recipient’s liver cells. […]

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