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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Biogen’s Aduhelm rollout goes from bad to worse

pharmaphorum

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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Fidelis Specialty Pharmacy Selects Citus Health to Enable Real-Time Virtual Patient Communications and Faster Collection of Key Patient Data

Citus Health Specialty Pharmacy

Innovative specialty pharmacy provider for hemophiliac and bleeding disorder patients to implement Citus Health’s HIPAA-compliant digital health solution suite to improve patient and staff communications, therapy adherence rates and patient outcomes.