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Michael Murphy, CFA
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Michael Murphy grew up in Newark, Delaware, which a sign at the city limits proclaimed: “The Educatoinal Center of the State.” After graduating from the public schools he earned an A.B. cum laude from Harvard College and began working as a COBOL mainframe programmer on an IBM 1401 in the... More
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  • What Katrina Was to FEMA, Swine Flu Deaths Are to FDA - Buy BioCryst 4 comments
    Sep 21, 2009 12:14 AM | about stocks: BCRX
    In contrast to their good job shepherding swine flu vaccines through the approval process, the FDA has failed for months to provide Intensive Care Unit doctors with an an effective intravenous antiviral to treat the sickest victims of H1N1. FDA began a Pre-Emergency Use Authorization process for BioCryst's peramivir almost six months ago. The development of peramivir has been sponsored by HHS, and it has completed Phase I and II trials in the U.S. and Phase I, II and III trials in Japan. It is known to be safe and effective, yet Americans are dying daily in the ICU without timely access to the drug.
     
    Why is an intravenous antiviral the FDA knows to be safe and effective languishing in their bureaucratic process while Intensive Care Unit doctors plead for it to be released before more victims die? The FDA approached BioCryst Pharmaceuticals of Birmingham, AL almost six months ago to begin the Pre-Emergency Authorization Use process for peramivir. The Phase I and II U.S. trials were sponsored by the Department of Health & Human Services under a $102.4 million contract. FDA had already reviewed Phase I safety data and approved the drug for Phase II studies. They then reviewed the Phase II safety and efficacy data and approved the drug for Phase III studies that may not be completed until 2011. In June, they gave the drug Emergency IND status, so a single doctor with an identified patient can fill out forms and ask the FDA to permit BioCryst to ship a dose of the drug. This shows again that the FDA knows the drug is safe and effective.
     
    The Emergency Use Authorization (EUA) process is designed to allow drugs in development to be sold or stockpiled to treat an imminent health threat, especially when no other effective treatment is available. There is no intravenous antiviral for the H1N1 flu approved anywhere in the world, including the U.S. Peramivir is the closest to achieving approval, in both Japan and Korea. I expected the FDA's EUA approval process to take no more than 60 days. It has now taken six months, with no end in sight, and many critically ill U.S. swine flu victims have died unnecessarily as a direct result of the delay.

    Most people who contract the swine flu simply suffer through the typical symptoms of flu. But 10% to 20% of the victims, who are often otherwise-healthy children, young adults or pregnant women become sick enough to be hospitalized. Most of those can be treated with Tamiflu or Relenza, which are already in national stockpiles around the world. But a significant portion — as high as 15 per cent, according to the World Health Organization — end up in ICUs for many days or even weeks, hovering between life and death.
     
    Dr. Paul Hebert, editor of the Canadian Medical Association Journal and an intensive care physician in Ottawa who has treated many of these sickest patients, said: “I've never seen this. As an ICU doctor, it's very, very, very rare I can't deliver enough oxygen to someone to keep him alive. They die of other things, right? They die because their organs fail. In this case, we can barely oxygenate them.”
     
    Peramivir is a very effective intravenous antiviral that can deliver a large load of drug very quickly, as opposed to the five-day treatment regimen with Tamiflu. Because the H1N1 virus often goes deep into the lungs and replicates much faster than the seasonal flu, many critically ill patients cannot use inhaled Relenza.
    If an ICU patient does not respond to or cannot take Tamiflu or Relenza, the ICU doctors have nowhere else to turn. Although peramivir is available on a case-by-case basis under the awkward, slow Emergency IND process, in most cases the drug cannot be transported to doctors in time to save a patient's life.
     
    In one recent case, desperate ICU doctors in Great Britain broke open a Relenza inhaler and mixed the contents into an IV bag, saving the life of a 22-year old woman. ICU doctors need an intravenous antiviral immediately, and peramivir is the only one that has completed Phase III clinical trials. Shionogi announced the positive results of their Phase III trials this summer, again showing that intravenous peramivir is a safe and effective drug.
     
    As a result of the FDA's intolerable delay in issuing the EUA and an order to stockpile the drug, hundreds of Americans have died unnecessarily, including 144 children in the flu season so far. Yet the logic for approval is simple:
     
    1. Is the drug safe and effective? Yes, as shown by the FDA's review of Phase I and Phase II results, the Japanese Ministry of Health review of Phase I and Phase II results, Shionogi's Phase III results reported recently at the ICAAC Conference in San Francisco, and the FDA's own granting of the Emergency IND status.
     
    2. Is there a need for the drug? Yes, there is no other intravenous antiviral available for ICU doctors to use when Tamiflu and/or Relenza have failed or cannot be given to the patient.
     
    3. Is the Emergency IND process sufficient to fill the need in ICUs? No, the cumbersome, slow process has resulted in less than 10 patients having access to peramivir since June, while hundreds have died.
     
    I am waiting for a crusading journalist, columnist or politician to hold the FDA's feet to the fire on this issue, and demand the Emergency Use Authorization and a substantial stockpiling order immediately. BioCryst has already signed sales reps in Mexico, Brazil, Israel and China, and I expect India and Europe to be next. These countries can place orders for peramivir with less bureaucracy than the FDA, and the drug might be saving lives overseas while Americans are still dying without it.

    To quote one well-known biotech columnist: “
    Murphy...seems to be the only person I hear slamming the FDA about its response to the flu, blaming regulators for dying kids. As if Margaret Hamburg has blood on her hands.”
    My sentiments exactly.

    I expect BCRX to book a large order for the U.S. stockpile, equally large orders from the BRIC countries, Mexico, Israel, Europe and the World Health Organization, and ongoing orders for many years to replenish the peramivir that is used in ICUs or emergency rooms. Thus, my disclosure.
     
    Disclosure: Long BCRX, obviously

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Comments (4)
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  • jq1234
    , contributor
    Comments (68) | Send Message
     
    Most ridiculous article on SA.
    21 Sep 2009, 01:08 AM Reply Like
  • shadowhunter
    , contributor
    Comment (1) | Send Message
     
    jq1234, if you opine about the article negatively, maybe you provide your evidence if you are bing truthful.
    21 Sep 2009, 09:11 AM Reply Like
  • lrrplrrpalpha
    , contributor
    Comment (1) | Send Message
     
    What is the FDA waiting for? Increasing numbers of Americans are getting terribly sick from the swine flu, and there is NO readily available drug to save their lives. However, due to American ingenuity and tenacity, Biocryst Pharmaceutical HAS developed and proven the safety/efficacy of an H1N1 antiviral drug, specifically for the sickest of the sick patients.

     

    FDA, do you view your job as protecting the major pharma companies, or to protect individual citizens first???? Please do make Peramavir available through stockpiles, etc, so our citizens can be saved!!

     

    Louis
    21 Sep 2009, 01:37 PM Reply Like
  • clawmann
    , contributor
    Comments (52) | Send Message
     
    And, today, after this was published FDA gave Biocryst $77.2 million to fund the Phase III US trials for peramivir. They also took a big step toward the EUA Mr. Murphy is arguing for. They sent an RFP (request for proposal) to Biocryst soliciting a proposal (essentially an offer) on a proposed stockpile order of 1,000 - 40,000 courses of peramivir. This will likely result in a contract very soon, and then the FDA will almost certainly have to issue the EUA as there is no point in having the drug in the stockpile if it cannot be used. In fact the RFP specifically referenced an EUA. Once the EUA is issued the ICU demand for the drug will become evident and it may well be that 40,000 courses will soon prove to be insufficient. Not a big problem as HHS can, if necessary, raise the ceiling on the contract, and this possibility was also indicated in the RFP.
    Furthermore, once HHS issues the EUA, this may well trigger the issuance of similar authorizations by foreign regulators, many of which simply issue approvals for a drug if it has been approved in the EU or the US.

     

    Put simply: I can see peramivir being readily available in a number of countries in the near future to help critically ill patients survive this and other viral infections.
    21 Sep 2009, 06:49 PM Reply Like
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