Thalassemia Patients and Friends

Discussion Forums => Working Towards a Cure => Topic started by: Bostonian_04 on August 18, 2022, 02:14:15 PM

Title: US FDA Approves ZYNTEGLO® (betibeglogene autotemcel)
Post by: Bostonian_04 on August 18, 2022, 02:14:15 PM

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

Title: Re: US FDA Approves ZYNTEGLO® (betibeglogene autotemcel)
Post by: Andy Battaglia on September 03, 2022, 08:10:39 PM

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

Title: Re: US FDA Approves ZYNTEGLO® (betibeglogene autotemcel)
Post by: VB on September 25, 2022, 10:11:50 PM

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.

Title: Re: US FDA Approves ZYNTEGLO® (betibeglogene autotemcel)
Post by: Andy Battaglia on January 07, 2023, 07:54:06 PM

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.

Title: Re: US FDA Approves ZYNTEGLO® (betibeglogene autotemcel)
Post by: wario on September 15, 2023, 06:04:34 PM

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.