Remove tag patient-outcomes
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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. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases.

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BD partners with Biocorp on connected self-injection devices

pharmaphorum

Medtech giant Beckton Dickinson (BD) has signed a deal with France’s Biocorp to use the latter’s near-field communication (NFC) tags in injectable devices. The Injay tag can confirm a complete injection and transfer that information via an NFC reader to a smartphone or tablet for review by a healthcare professional.

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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. million if paid through an outcomes-based contract for patients with sustained transfusion independence.”

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FDA gives Daiichi Sankyo’s HER3 drug a breakthrough tag

pharmaphorum

The designation – which is awarded to therapies that could represent a significant improvement over standard of care in either efficacy, safety, or both – has been awarded on the strength of phase 1 results in patients with metastatic or locally advanced EGFR-mutated non-small cell lung cancer (NSCLC).

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Revolutionizing Medicine and Public Health: The Emergence of Big Data in Healthcare

Roots Analysis

Velocity: Big Data needs to be handled promptly as businesses generate data at an unprecedented speed, driven by the growing adoption of Internet of Things (IoT), and technologies, such as RFID tags, sensors, and smart meters are used to deal with the high velocity of data in real-time. It is loosely arranged into categories using meta tags.

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Biogen’s Aduhelm rollout goes from bad to worse

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As it turned out, Aduhelm (aducanumab) made just $300,000 in the three-month period from July to September, against analysts’ estimates of $12 million, suggesting that few patients are being treated with the $56,000 a year therapy. Biogen reported $1.6 million in sales for the drug in the second quarter.

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Second unanimous FDA adcomm vote boosts bluebird bio

pharmaphorum

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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