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FDA approves Pfizer's first gene therapy for rare inherited bleeding disorder (www.nbcnews.com)
submitted 4 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 4 months ago

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 4 months ago

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 4 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.

this post was submitted on 28 Apr 2024
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