Pharmaceutical Technology

SEPTEMBER 30, 2022

Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

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pharmaphorum

APRIL 14, 2022

US cost-effectiveness watchdog ICER has handed bluebird bio some good news ahead of its FDA advisory committee meeting for rare blood disorder gene therapy beti-cel in June, by endorsing its proposed $2.1 million price tag. million if paid through an outcomes-based contract for patients with sustained transfusion independence.”

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pharmaphorum

JUNE 12, 2022

The panel voted 13 to 0 on the question of whether beti-cel’s benefits outweighed the risks of the gene therapy in patients with beta thalassaemia who are dependent on blood transfusions, in a dream result for the biotech which just a few weeks ago was expressing doubts about its ability to continue as an operating concern.

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pharmaphorum

AUGUST 18, 2022

bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. million for the transfusions themselves and $3.7 bluebird has opted to launch it at a cost of $2.8

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Pharmaceutical Technology

APRIL 13, 2023

Although the findings in the report are only preliminary, they shed light at the considerations behind the high price tags of gene therapies. Sickle cell disease is an inherited blood disorder that is caused by mutations in the HBB gene, which codes for the oxygen-carrying protein haemoglobin in red blood cells.

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pharmaphorum

MAY 17, 2021

In addition, 85% of Empaveli-treated patients were transfusion-free over 16 weeks versus 15% of the Soliris-treated group. PNH is an autoimmune disease characterised by uncontrolled activation of the complement system that results in the destruction of red blood cells and low levels of oxygen-carrying haemoglobin. billion and $1.1

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pharmaphorum

FEBRUARY 16, 2021

There is a curative treatment for people who rely on blood transfusions to survive and maintain their levels of red blood cells. In this first draft guidance NICE raised a series of issues with Zynteglo, which bluebird has already agreed to supply at a confidential discount from its hefty price tag, which is around €1.57

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