english.prescrire.org > Spotlight > Archives : 2020 > In the December issue of Prescrire International: Betibeglogene autotemcel (Zynteglo°) in certain forms of severe beta thalassaemia

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In the December issue of Prescrire International: Betibeglogene autotemcel (Zynteglo°) in certain forms of severe beta thalassaemia

FREE DOWNLOAD Despite the uncertainties, an option for achieving transfusion independence in the absence of a compatible donor.
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  • Beta thalassaemia is a genetic disorder that affects the production of beta globin chains, one of the two protein components of haemoglobin. Betibeglogene autotemcel (Zynteglo°, Bluebird Bio) is a gene therapy product which allows some patients to stop transfusion therapy without developing severe anaemia.
     
  •  OFFERS AN ADVANTAGE  Betibeglogene autotemcel gene therapy has not been shown to be more effective than allogeneic stem cell transplantation in patients with beta thalassaemia who require regular blood transfusions and produce low levels of beta globin. The limited preliminary evaluation data available on betibeglogene autotemcel show that it enables about 80% to 90% of patients to achieve sustained transfusion independence. The adverse effect profile of this gene therapy product is uncertain due to the paucity of data and short follow-­up. The treatments preceding the infusion of betibeglogene autotemcel can provoke serious adverse effects. In practice, as of 2020, betibeglogene autotemcel is an option to offer patients who are eligible for transplantation but have no compatible donor, informing them that this treatment has undergone very limited evaluation.

  •  EDITORS' OPINION  Betibeglogene autotemcel is one of the first drugs to have obtained European marketing authorisation through the European Priority Medicines (“Prime”) scheme. Early marketing authorisation often means that the drug’s evaluation was based on very limited and typically non-comparative clinical data. And early cooperation between drug companies, the EMA and national health technology assessment bodies strengthens the pharmaceutical industry’s influence over these agencies’ decisions and compromises the assessment bodies' role as a “watchdog”.


©Prescrire 1 December 2020

Source: "Betibeglogene autotemcel (Zynteglo°) in certain forms of severe beta thalassaemia" Prescrire International 2020; 29 (221): 290-291. Free.

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