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Posts tagged with "Regulatory Affairs"

  • Covid-19

    Trouble at the FDA and CDC

    That title is the only way that I can describe the events of the last few days. Like so many other things this year, what we’re seeing now at the top of the drug regulatory structure is unprecedented, and not in a good way at all. Let’s recap. A few days ago, HHS Secretary Alex… Read More
  • Covid-19

    Vaccine Transparency

    I’ve been emphasizing for some time that our efforts to find and deploy a coronavirus vaccine have to be as transparent as possible to increase the chances for success. Recent events make that more clear than ever – and not in a good way. Update: Moderna has now published their complete clinical trial protocol, and… Read More
  • Covid-19

    Convalescent Plasma: The Science and the Politics

    I suppose that I’m going to have to say something about yesterday’s convalescent plasma announcement. First, the medical aspects: in my view, for what that’s worth, convalescent plasma is likely to be at least somewhat helpful to hospitalized coronavirus patients. I think that its safety profile is likely to be good as well, and I… Read More
  • Regulatory Affairs

    Not Approved Until It’s Approved

    For some years now, Biomarin has been working on a gene therapy for hemophilia A. That’s the form of the disease caused by a deficiency in Factor VIII, which is a necessary part of the blood-clotting cascade. Like many such conditions, it comes in a wide range of phenotypes. One group of patients (around 10%) Read More
  • Current Events

    The Politics of Hydroxychloroquine

    I had not been planning to return to the topic of hydroxychloroquine so soon, but here we are. This will not be a calm, measured blog post – fair warning. Yesterday, Dr. Rick Bright was pushed out of his post at HHS, where he was deputy assistant secretary for preparedness and response and director of BARDA… Read More
  • Alzheimer's Disease

    A Prospective Alzheimer’s Trial Reports

    For the past several years, a clinical trial from Washington University (St. Louis) has been underway in people with genetic mutations that lead to early-onset Alzheimer’s. The Dominantly Inherited Alzheimer’s Network (Trials Unit), DIAN-TU, has been dosing 194 such patients with one of two anti-amyloid antibodies, either Lilly’s… Read More
  • Clinical Trials

    Opening the Lid on Sarepta’s Drug Approvals

    Let’s talk Sarepta. And FDA approval, because you can’t bring up that company without immediately starting a regulatory affairs argument. I was not happy when their initial exon-skipping therapy (Exondys, eteplirsen) for Duchenne muscular dystrophy was approved in 2016, because I thought that the efficacy data were simply not strong eno… Read More
  • Business and Markets

    EQRx’s Challenge, And My Challenge to Them

    It’s time to talk about a new venture called EQRx. This has made quite a splash in the last few days at the JP Morgan investor conference, and it’s been launched by Alexis Borisy (involved with founding and/or helping run CombinatoRx, Foundation, Blueprint, WarpDrive Bio, Editas, Relay and others) with the aim of generating cheaper… Read More
  • Academia (vs. Industry)

    Getting Around to Reporting Clinical Data, Real Soon Now

    Science has an interesting report on the publication of clinical trial results. Some readers will recall similar efforts from 2015 and 2017/2018 in the US and Europe; this is actually a follow-up by one of the same US authors. It should actually be a dull report, because the requirements for such disclosure are clear. The rules… Read More
  • Alzheimer's Disease

    They Don’t Know

    Well, Biogen has released more data on its Alzheimer’s antibody, aducanumab. The people (like me) who were doubtful (or worse) that they had enough to make a case for FDA approval remain doubtful. And the people (there are some) who think that it’s approvable haven’t changed their minds, either, from what I can see. Frankly… Read More