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Aegerion Pharmaceuticals, Inc. (AEGR) Message Board

  • hankmanoo hankmanoo Aug 17, 2013 10:07 PM Flag

    Part II - AAT orphan drug

    PLI’s strategy has attracted considerable investment from
    various biopharmaceutical companies, particularly those based
    in Asia, where the high cost of legacy technology has limited
    demand. As the management of PLI pointed out at yesterday’s
    Q2 results presentation, Asia Pacific uses less than 15% of the
    IVIG supply today despite representing 60% of the world’s
     Potential value of AAT sales
    AAT has been approved by the FDA for various orphan
    indications; however the market has grown by a CAGR of just
    16% since 2000 to reach $750m in 2012. At a price point of
    €70,000 in the EU and $120,000 in the US, only a minority of
    sufferers are being treated. With a much improved recovery
    yield, PLI can create a much larger market for AAT not just in
    the US/EU, but worldwide.
    1. What is AAT?
    Alpha-1 antitrypsin is a protein made by cells in the liver. It passes out from the
    liver into the bloodstream and can travel to the lungs. Its main function is to
    protect the lungs from damage caused by other types of proteins called enzymes.
    Enzymes are essential for the normal working and development of the body. In the
    lungs, certain enzymes called proteases help to fight infection, by removing
    bacteria and may also be released to try to protect the lungs from tobacco smoke.
    However, the activity of these protease enzymes needs to be balanced. If the
    balance tips and there is too much activity, then the tissue of the lungs can start to
    become damaged by the enzymes. AAT helps to balance the protease enzymes in
    the lungs and stop lung damage.
    AAT deficiency is an inherited genetic condition. It can lead to lung and, in some
    people, liver damage at any age. According to PLI, there is an estimated 100,000
    people affected by AAT deficiency in the USA alone with less than 10% treated.
    According to the Alpha-1 Foundation, there may be as many as 3% of the 20m
    patients suffering from Chronic Obstructive Pulmonary Disease (COPD) that may
    also have an undetect

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