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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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Can gene therapies for haemophilia defend their high price tags?

Pharmaceutical Technology

Reimbursement has been a major obstacle in the EU for expensive gene therapies, including bluebird bio’s beta-thalassemia therapy Zynteglo. The European Commission withdrew the marketing authorisation of Zynteglo, at the request of bluebird bio, in March 2022. After its approval, bluebird had set Zynteglo’s price at roughly EUR1.6

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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. An FDA advisory committee recommended approval of Zynteglo on the strength of that data in June.

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FDA Approval of First Rapid-Acting Oral Depression Drug + Approval of $2.8 Million Gene Therapy for Rare Blood Disorder – Xtalks Life Science Podcast Ep. 75

XTalks

Bluebird bio was awarded the approval for its gene therapy Zynteglo (beti-cel), which is a one-time treatment for the rare blood disorder. Hear more about the therapy, some of the setbacks bluebird has had to face on the road to its approval and why the treatment has a steep price tag. Bluebird’s $2.8M

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STAT+: Pharmalittle: Vertex exec says prices for gene therapies are ‘in the ballpark’; insulin access in poor countries remains spotty

STAT News

million tag as cost effective for a Bluebird Bio drug called Zynteglo for the blood disorder beta thalassemia, Arbuckle said in an interview.  Even a drug-price watchdog, the Institute for Clinical and Economic Review, deemed the $2.8

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

In 2022, bluebird won approvals for two gene therapies — Skysona for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD) and Zynteglo for beta-thalassemia. Morgan analysts were also hopeful about bluebird’s clearer commercialization plan for Lyfgenia and market readiness from Zynteglo.

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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. . The post Zynteglo halt re-ignites viral vector safety concerns; analysts appeared first on.