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 —