Plasma Protein Therapies

What is a plasma protein therapy?

These unique therapies treat well-defined medical conditions, replacing missing or deficient proteins found in plasma, to allow their recipients to lead healthier and more productive lives. The patient populations that rely upon plasma protein therapies generally require regular infusions or injections for the duration of their lives.

Plasma-derived therapies and their recombinant analogs, collectively referred to as plasma protein therapies, are unique, biologic medicines, versus synthetic or chemical medicines, because production begins with plasma, a biologic starting material. Each therapy has its own unique biochemical profile, due to differences in production and processing methods, which results in different clinical responses and efficacy among patients.

Plasma protein therapies are not interchangeable and have been defined by regulators as sole-source biologic products because no generics or substitutions exist. In addition, their biological nature demands storage and handling requirements by specialty distributors that ensure their safety.

Therapies

Therapy	Conditions Treated	Treatment Outcomes
Coagulation factors: Essential for blood clotting, used to treat genetic bleeding disorders and surgical bleeding.	Bleeding from trauma Over dosage of anticoagulants Liver disease Bleeding Disorders Hemophilia A and B - Inherited disorders caused by mutations in Factor VIII and Factor IX leading to abnormalities in blood clotting. Von Willebrand disease - The most common inherited bleeding disorder due to mutations in Von Willebrand Factor. *Other rare coagulation disorders*	Improved quality of life Increased life expectancy
Immunoglobulins: Proteins used to neutralize foreign objects, such as bacteria and viruses, and regulate the immune system. Used as a therapy in primary and secondary immunodeficiencies and autoimmune disorders.	Immunology-Immunodeficiencies Primary - Life threatening genetic or idiopathic defects that compromise immune system. Secondary - Caused by outside factors such as viruses, chemotherapy, and immunosuppressing therapies. Neurology-Immune-mediated diseases Chronic Inflammatory Demyelinating Polyneuropathy, Guillain-Barré syndrome, Multifocal Motor Neuropathy Haematology Idiopathic Thrombocytopenic Purpura Dermatology Dermatomyositis	Infection prevention Regulation of overreacting immune system Improved quality of life Increased life expectancy
Hyperimmune Globulins: Prevention and treatment of specific infections and certain conditions.	Rabies, tetanus and hepatitis Rh negative pregnancy Transplant therapy	Prevention Treatment Protection of babies in utero
Alpha-1 Proteinase Inhibitors: Protects tissues from enzymes of inflammatory cells.	Alpha-1 Antitrypsin Deficiency - Genetic deficiency which may result in life-threatening lung disease in adults and/or liver disease in people of any age.	Improved quality of life Halts disease progression
Albumin: The major plasma protein, regulating blood volume and providing many essential functions.	Cardiac surgery Liver disease Severe infections Emergency and Surgical Medicine - Used to treat shock, severe burns and during surgery.	Lifesaving in severe situations Decreased morbidity and mortality
C1-esterase inhibitor (C1-INH): A protein found in the fluid part of your blood. It controls a protein called C1, which is part of the complement system. This system is a group of proteins that move freely through your bloodstream.	*Hereditary angioedema* - Rare but potentially life-threatening condition characterized by acute attacks of usually non-itching edema (swelling) of the face, larynx (airway), abdomen and extremities.	Improved quality of life Increased life expectancy

Who needs plasma protein therapies?

Rare Diseases and Conditions

These therapies replace missing or deficient proteins that allow individuals to lead healthy and more productive lives. The patients that rely on these therapies generally require regular infusions or injections throughout their lives. The diseases and conditions treated by plasma protein therapies are considered rare diseases because they affect a relatively small percent of the population. Most are genetic, chronic conditions.

Alpha-1 Proteinase Inhibitor therapies are used to treat:

Alpha-1 Antitrypsin Deficiency

Alpha-1 Antitrypsin Deficiency is one of the most common serious hereditary disorders in the world and can result in life-threatening liver disease in children and adults and lung disease in adults. It is often referred to as genetic emphysema.

C1 Esterase Inhibitor therapies are used to treat:

Hereditary Angioedema

Hereditary Angioedema is caused by a missing C1 esterase inhibitor protein (C1-INH), which helps regulate inflammation. Patients can experience edema (severe swelling) which can be fatal if the airway becomes obstructed.

Coagulation Factors are used to treat:

Hemophilia A

Hemophilia A is a heredity bleeding disorder that is caused by a lack of clotting factor VIII. As a result, individuals with this condition suffer from bleeding into joints and other complications. For the most part, men have hemophilia A as the defective gene is found on the X chromosome. A woman who has the defective gene is considered a carrier and any male offspring have a 50% chance of having hemophilia A and female offspring a 50% chance of being a carrier. It affects one in 10,000 people. With treatment, individuals are able to lead relatively normal lives.

Hemophilia B

Hemophilia B is a blood clotting disorder caused by a mutation of the Factor IX gene. It is rarer than hemophilia A and affected royal families in both Europe and Russia. It affects one in 25,000 men. Replacement of Factor IX through recombinant therapy allows individuals to lead relatively normal lives.

Von Willebrand Disease

The most common bleeding disorder, Von Willebrand affects about 1.25 million men and women worldwide, although it is estimated that as many as 3 million are undiagnosed. Common symptons include excessive menstrual bleeding and nosebleeds. There are several kinds of VWD and symptom severity varies. With proper treatment, individuals may live relatively normal lives.

Antithrombin III Deficiency

Antithrombin III is a protein that prevents blood clots from forming. It is caused by an abnormal gene that may lead to blood clots. Data on incidence is uncertain.

Immune Globulins are used to treat:

Primary Immunodeficiency Disease (PID)

PID is a genetic condition that prevents an individual's immune system from functioning properly. The World Health Organization (WHO) estimates that there may be as many as 150 PIDs. PID results a high susceptibility to infection and an inability to fight them with traditional antiobiotics.

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

CIDP is a rare autoimmune disorder that affects the peripheral nervous system of both children and adults. Nerves in the arms and legs may become weakened and lead to paralysis.

Idiopathic Thrombocytpenic Purpura (ITP)

ITP is an autoimmune blood disorder that results in reduced blood platelet levels, essential for blood clotting.

Kawasaki Disease

A condition that primarily affects children under the age of five and is the leading cause of acquired heart disease in children. If it is not detected, it can result in heart damage and death.