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Amylyx ALS drug draws criticism over $158,000 price tag

pharmaphorum

Amylyx Pharmaceuticals’ Relyvrio – a new treatment for amyotrophic lateral sclerosis (ALS) approved by the FDA on Thursday, after review of the data from its phase 2 trial – was the next day set at a list price of $158,000 per year in the US, sparking outcry. ALS affects over 30,000 people in the US.

Drugs 52
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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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Clover biopharmaceuticals raises $230 M; BrainStorm after ALS; BARDA grants $5.65 M LightDeck for SARS-CoV-2 antigen test; Libtayo for first-line advanced NSCLC subset

Delveinsight

The company plans to use the capital to expand its pipeline of protein-based vaccines and biologic cancer therapies utilizing its innovative and proprietary Trimer-Tag technology platform. BrainStorm Receives Feedback on BLA Submission for ALS. The post Clover biopharmaceuticals raises $230 M; BrainStorm after ALS; BARDA grants $5.65

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Biogen’s Aduhelm rollout goes from bad to worse

pharmaphorum

Vounatsos also insisted that Aduhelm’s price tag has not been a factor discouraging treatment with the drug. Biogen stumped up $1.525 billion upfront for rights to the drug and another Sage candidate for neurological diseases last year, when the expectation was that Aduhelm was unlikely to get FDA approval.

Sales 122
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Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Pharmaceutical Technology

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

Drugs 246
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Perfusion MRI and Other Perfusion Imaging Techniques for Clinical Trials

XTalks

An example of an anti-angiogenic compound working to decrease tumor perfusion is shown by representative images collected by DCE-MRI for a Phase I study ( Figure 1 ); here, the red and yellow areas of the baseline image are areas with very high blood flow, and Liu et al., Figure from Liu et al., Figure from Ricart et al.,

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Sponsored content: Why choose Bethyl antibodies for your R&D

Drug Discovery World

The principles that have guided our rigorous validation practices for decades preceded a publication (Uhlen et al., Pillar 5: Protein OE/Epitope Tags. Bethyl antibodies are highly regarded for passing strict validation testing before arriving in customer hands. A proposal for validation of antibodies.