this post was submitted on 28 Apr 2024
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FDA approves Pfizer's first gene therapy for rare inherited bleeding disorder (www.nbcnews.com)
submitted 7 months ago by MicroWave@lemmy.world to c/health@lemmy.world
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The agency greenlit the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B who meet certain requirements.

The Food and Drug Administration on Friday approved Pfizer’s treatment for a rare genetic bleeding disorder, making it the company’s first-ever gene therapy to win clearance in the U.S. 

The agency greenlit the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B who meet certain requirements.

The treatment will be available by prescription to eligible patients this quarter, a Pfizer spokesperson told CNBC. It has a hefty $3.5 million price tag, before insurance and other rebates, the spokesperson added, making it by far one of the most expensive drugs in the U.S.

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[–] paysrenttobirds@sh.itjust.works 3 points 7 months ago (1 children)

BEQVEZ is a one-time treatment that is designed to enable people living with hemophilia B to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that are often administered multiple times a week or multiple times a month.1,2

A 2021 study found that the cost for an adult’s lifetime treatmentTrusted Source of moderately severe to severe hemophilia B averages $22,987,483 million for preventive FIX treatment and $20,971,826 for on-demand FIX treatment. --https://www.healthline.com/health/hemophilia-treatment#costs

[–] IamtheMorgz@lemmy.world 4 points 7 months ago (1 children)

Ooh, I think I remember reading somewhere that these patients have to be moved around insurance pools in the US just to keep the insurance industry working, lol. Because they're so expensive. I guess 3.5 is less than 20.

[–] paysrenttobirds@sh.itjust.works 1 points 7 months ago

But, it's actually even so much better because it's supposed to work as a one time cure. So, in addition to smaller price tag all of the ongoing stress and coordination for the patients and the system just evaporates! So wonderful if it works.