US FDA Approves ZYNTEGLO® (betibeglogene autotemcel)

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

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

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.

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.

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.