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US FDA Approves ZYNTEGLO® (betibeglogene autotemcel)

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Bostonian_04:
FINALLY!

Bluebird bio announced that the U.S. Food and Drug Administration (FDA) has approved ZYNTEGLO® (betibeglogene autotemcel), a one-time gene therapy to treat patients with beta-thalassemia who require regular red blood cell (RBC) transfusions.

You can find a full press release and more information at https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-fda-approval-zynteglor-first-gene-therapy

Andy Battaglia:
The price tag is enormous. Do you know anything about if insurance companies will help cover the cost?

VB:
Is ZYNTEGLO really able to help Thal Majors keep their Hb in normal or near-normal ranges ? Is there anyone on this group who has tried this drug ? Yes, the cost is too expensive. About 2-3Million dollars.

Andy Battaglia:
I would also like to hear from anyone who has experience, either personal or with a child who has undergone gene therapy and what the results are. With the cost of this therapy, one must be certain a cure will be had by the introduction of gene therapy.

wario:
I am also interested in talking with someone who has had this treatment or is currently in the process. I have been told by my doctors that I am on the list for getting this but I have a few questions. Please let me know if you have some time to talk.

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