FDA approves $3.5 million treatment for hemophilia, now the most expensive drug in the world

[USALiberal]

FDA approved Hemgenix based on safety and effectiveness evaluated in two studies of about 60 adult men.
Sarah Silbiger/Getty Images
CNN
—
The US Food and Drug Administration on Tuesday approved Hemgenix, a new drug to treat hemophilia. Manufacturer CSL Behring set the price at $3.5 million per treatment, making it the most expensive drug in the world.

Hemgenix is a gene therapy to treat adults with hemophilia B, a genetic bleeding disorder in which people do not produce a protein needed to create blood clots. About 1 in 40,000 people have the disease, most of whom are men.

Scientist preparing to view a blood sample under a microscope in laboratory for medical testing. Hemophilia B is a bleeding disorder that slows the blood clotting process.
Gene therapy reduced risk of excessive bleeding for people with hemophilia B, early trial data suggests
Typical treatment requires routine intravenous infusions to maintain sufficient levels of the missing or deficient clotting factor, but the new gene therapy is meant to be a one-time IV infusion.

“Gene therapy for hemophilia has been on the horizon for more than two decades. Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life,” Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “Today’s approval provides a new treatment option for patients with Hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia.”

More at: https://www.cnn.com/2022/11/23/health/hemophilia-drug-hemgenix

[Gaugamela]

1. So if they sell it to the 2000 people on the planet who have the condition and who

Last edited Fri Nov 25, 2022, 01:43 PM - Edit history (1)

can afford it, they’ll gross 7 billion.

Sounds like a business plan.

[ck4829]

2. What's going to be done to make sure poor and middle class hemophiliacs can get this drug?