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Aegerion Pharmaceuticals, Inc. (AEGR) Message Board

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

    Part III - AAT orphan drug

    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 undetected AAT deficiency.
    2. Growth in Speciality Plasma Products such as ATT
    Over the past twenty years, the plasma industry has extracted new proteins and
    commercialised them, many as orphan drugs. Plasma proteins offer safe and
    efficacious treatments. As the commercial viability of new proteins is hampered by
    the small number of patients, the orphan drug regulations have facilitated their
    development and market introduction.
    The worldwide plasma fractions market has grown from around $1.9bn in 1986 to
    nearly $14bn in 2011. The market share of Intravenous Immune Globulin
    (IVIG/SCIG) has grown the fastest, followed by speciality products which include
    ATT, antithrombin III, C1 Esterase Inhibitor, etc..
    3 Limitations of Legacy Plasma Manufacturing Technology
    Despite the recent successes, plasma proteins remain the last resort drugs for
    many rare diseases. This is to some extent because the vast majority of players in
    the plasma industry still rely on the traditional Cohn process, which was developed
    during World War II, to primarily recover Albumin from plasma for use in
    hypovolemic shock. In order to be economically sound, a plasma fractionation plant
    must produce three major products: IVIG/SCIG, Albumin and Factor VIII.
    While the Cohn process yields around 82% Albumin, the yield of other valuable
    plasma proteins e.g. Factor VIII (for Haemophilia A), IVIG (for Primary Immune
    Deficiencies) or ATT remain relatively poor at around 35%, 70% and 23%,
    Consequently, the Cohn process is not able to meet worldwide demand. The
    complex steps and regulations required to collect donated plasma and complete the
    highly technical, innovative manufacturing processes that ultimately result in a final
    therapy result in long intervals between donation and final product release —

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