Remove tag thalassaemia
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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

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. After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals.

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ICER says bluebird bio’s $2.1m gene therapy is cost-effective

pharmaphorum

million price tag. The FDA is currently reviewing beti-cel – also known as Zynteglo (betibeglogene autotemcel) – as a treatment for patients with beta thalassaemia who require regular blood transfusions. The gene therapy has already been approved for marketing in the EU and UK, where it was launched at a cost of around $1.7

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Second unanimous FDA adcomm vote boosts bluebird bio

pharmaphorum

Thursday saw the Cellular, Tissue and Gene Therapies advisory committee back elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD) – see our report here – and the following day it was the turn of betibeglogene autotemcel (beti-cel) for beta thalassaemia.

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bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval

pharmaphorum

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. bluebird has opted to launch it at a cost of $2.8 million, which compares to the $1.7 million, spit between $1.6

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Zynteglo halt re-ignites viral vector safety concerns; analysts

pharmaphorum

Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. . million in Europe.

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bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy

pharmaphorum

It’s back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance. The post bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy appeared first on. million in Europe.

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Advanced therapies and the high-profile pricing dilemma

pharmaphorum

Only a few years later, bluebird bio’s gene therapy, Zynteglo (betibeglogene autotemcel), was approved in the European market for the treatment of the rare blood disorder, beta thalassaemia. However, with a price tag of €1.58 At more than $2.1 The post Advanced therapies and the high-profile pricing dilemma appeared first on.

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