Source: CSL Behring

Source: CSL Behring

Over 20 therapeutic plasma proteins are commercialized today. The industry seeks new clinical uses for existing products and purifies and evaluates new proteins for therapeutic use. Most of the diseases treated with plasma products are considered “rare” – affecting relatively few patients. Consequently, many of these products are classified as orphan drugs. There are four main categories of plasma-based products:

  1. Immune globulins (IgG): they are produced by B-cells in the body to identify and aid in the destruction of foreign molecules including bacteria and viruses. They are the main function of the body’s humoral immune system. Replacement therapy with immune globulins has been used extensively since their commercial introduction in the 1950’s. Two categories of immunoglobulin preparations exist today:
    1. Intravenous and subcutaneous immunoglobulins (IVIG/SCIG)a.     are the most common preparations, and were first prescribed as a replacement therapy for congenital antibody deficiencies. Over the years, IVIG/SCIG has been found to be increasingly efficacious in treating neurological conditions, as well as diseases in other areas, including hematology, infectious diseases, etc. Over 200 diseases have been identified for IVIG/SCIG treatment, many of them extremely rare.
       
      The indications approved by the FDA and many other regulatory agencies include: Primary Immunodeficiency (PID), Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Idiopathic Thrombocytopenic Purpura (ITP), Multifocal Motor Neuropathy (MMN), Kawasaki Disease, Chronic Lymphocytic Leukemia (CLL) and post-Bone Marrow Transplant (BMT).
    2. Hyperimmune immunoglobulins are preparations in which the level of some specific antibodies has been enriched to target a particular antigen, as opposed to the naturally diverse set of antibodies present in IVIG and SCIG. Specific hyperimmune preparations are made for specific conditions, including; Anti-D (anti-RhoD), Cytomegalovirus (CMV), Hepatitis A and B, Rabies, Tetanus, and Varicella, with more in development.
  2. Hemophilia: Congenital hemophilia is the result of a missing or dysfunctional protein in the protein-clotting cascade of an individual. Thus, a person with hemophilia is often unable to control a bleeding situation without “replacement therapy” for the missing/damaged clotting protein. The missing protein is often one of the factor proteins, such as factor VIII, correcting the most common factor deficiency.
    1. Hemophilia A: Factor VIII deficiency causes hemophilia A, and can be mild, moderate or severe. Many moderate and severe patients need factor VIII replacement therapy either on a regular basis (prophylaxis) or in the event of a bleeding episode (on-demand).
    2. Hemophilia B: Factor IX deficiency causes hemophilia B, and like hemophilia A, it may be mild, moderate or severe, although it is not as prevalent as hemophilia A worldwide. It also requires factor IX replacement therapy. Hemophilia A and B males at much higher rates because it is an X-linked chromosome disease.
    3. Von Willebrand Disease (vWD): Von Willebrand Disease is caused by the lack or dysfunction of the von Willebrand Factor (vWF), which binds with factor VIII to promote blood clotting. This disease is more prevalent than hemophilia, but a greater percentage of patients are mild and need little treatment. Severe patients often require the use of a product containing vWF, which generally also contains factor VIII.
    4. Other Factor Deficiencies: There are several other factor deficiencies, some of which are acquired during surgery or blood loss situations, while others are congenital. These include deficiencies of factor II, V, VII, X, XI, XIII and fibrinogen.
  3. Albumin

    Albumin

    Albumin has been used since the 1940’s as a volume replacement for blood or fluid loss, either in surgery or in trauma situations. It is also used in sepsis, septic shock, therapeutic plasma exchange, burn therapy and renal dialysis, among others. In some of these conditions, albumin competes with non-protein based volume replacement solutions such as starches, Ringer’s Lactate or saline.

  4. Alpha-1 antitrypsin (AAT) or Alpha-1 Protease Inhibitor (A1PI) 1. is used as a replacement therapy for A1PI deficiency, which is a rare genetic disorder characterized by reduced serum levels of the A1PI enzyme. It causes a variety of conditions including emphysema, chronic obstructive pulmonary disease (COPD), bronchiectasis, chronic bronchitis, cirrhosis, neonatal hepatitis and jaundice. Alpha-1 antitrypsin deficiency is found mainly among people of northern European descent, and it is less common in other populations.
  5. Antithrombin III (AT-III) is used as a replacement therapy for AT-III deficiency, which results in the inability to adequately control the clotting process, leading to excessive clotting and risk of thrombosis. The congenital deficiency is rare, but the acquired form can develop from liver dysfunction, sepsis, or major surgery. AT-III can be used before, during or after surgery as an anticoagulant for patients suffering from low AT-III protein levels.
  6. C1 Esterase Inhibitor (C1-INH) is used as a replacement therapy for Hereditary Angioedema (HAE), which results in the swelling of the face, neck, extremities and upper airways. If left unchecked, this rare disease causes swelling of the larynx, which can lead to suffocation. C1-INH therapy replaces the missing protein in this congenital disease.