A list of terms used on this website and their definitions.


Australian Drug Evaluation Committee


A molecule capable of combining with a biochemical receptor on a cell and initiating the same response as occurs naturally

Airway Responsiveness

The degree to which airways react to a stimulus. Usually used to describe the degree of airway constriction that will be caused by exposure to a stimuli


Relieving pain; a pain-relieving drug


A chemical that acts within the body to reduce the physiological activity of another chemical substancei.e. opposing the action of a drug or a substance occurring naturally in the body by combining with and blocking its receptor


Aridol® is a patented, dry powder formulation of mannitol delivered to the lungs through an inhaler. Aridol™ is applied as a bronchial provocation test to accurately diagnose the presence and severity of bronchial hyper-responsiveness or over-sensitivity, which is characteristic of asthma.


Asthma is a serious condition in which the small airways of the affected person's lungs suddenly constrict when they are exposed to certain triggers. Airflow into and out of the lungs is reduced, and the person has to gasp for breath.


Having the property whereby immune cells respond to tissues in ones' own body.

Beta Interferon

A protein released by connective tissue cells in response to a viral infection. The protein can be synthesised and used in the treatment of multiple sclerosis

Breakdown Products

Products that result from the disintegration or decomposition of a substance in the body

Bronchial hyper-responsiveness or over-sensitivity

When a person's bronchial tubes (tubes that lead to the left and right lung) are abnormally responsive or sensitive to triggers and react by narrowing and becoming inflamed

Bronchial Provocation Test

A lung test that provokes a temporary narrowing of the bronchial tubes in the lungs


A form of chronic obstructive pulmonary disease (COPD) characterised by irreversible dilation and destruction of the bronchial walls

Bronchiectasisis a progressive lung disease, affecting 600,000 people worldwide. It is often mistaken for asthma or pneumonia and misdiagnosis is common. In this disease the airway walls are chronically inflamed, with poor clearing of the increased mucus production. Chronic inflammation of the walls of the airway is common to all types of bronchiectasis. This is often a result of a vicious cycle of bacterial infection, in which damage to the lungs further predisposes the lung to more infections.The body repairs the damaged lung tissue by forming tough, fibrous material, which leads to changes that impair normal lung structure and function. Effects include:

  • Reduced lung capacity;
  • Poor gas-exchange;
  • Changes of the organisation of blood vessels; and
  • Overall increased blood flow through the lungs.

These changes can ultimately lead to heart failure. Recurrent lung infections commonly reduce patients' quality of life; progressive respiratory insufficiency is the most common cause of death.Most cases of bronchiectasis develop during childhood, and can be a result of infections such as pneumonia or the inhalation of noxious substances.

Treatment today is aimed at controlling infections, secretions, airway obstructions and complications. There are no therapeutic products available to effectively clear excess mucus secretions and improve the quality of life of sufferers. Current management of bronchiectasis often involves:

  • Bronchodilators, to dilate the airways to help mucus clearance;
  • Steroids. However, only a minority of mild-moderate bronchiectatic patients respond positively to steroids;
  • Antibiotics to clear infections; and
  • Regular, daily postural drainage to remove bronchial secretions.

Bronchitol® is a patented, dry powder formulation of mannitol delivered to the lungs through an inhaler. Bronchitol™ is designed for the treatment of diseases such as COPD and cystic fibrosis.


A substance that acts to dilate or expand the bronchial airway passages, making it easier for patients to breathe


Potential to cause cancer

Central Nervous System

System of nerves of the brain and spinal cord


A chemical agent that induces movement of cells in the direction of its highest concentration

Chest Percussion

Form of physiotherapy/massage that involves tapping the patient's chest with light, rapid blows to help them expel mucus from their lungs


A disease or condition of long duration or frequent recurrence; in some instances, it may slowly become more serious over time

Chronic Obstructive Pulmonary Disease

A group of lung diseases characterised by limited airflow with variable degrees of air sack enlargement and lung tissue destruction. Emphysema, chronic bronchitis and bronchiectasis are forms of chronic obstructive pulmonary disease. Abbreviated as COPD.

Chronic Obstructive Pulmonary Disease, or COPD, encompasses a number of serious conditions affecting the lungs, including emphysema, chronic bronchitis and bronchiectasis and other chronic and acute pulmonary conditions.According to the World Health Organization, or WHO, 210 million people suffer from COPD and the disease was responsible for 3 million deaths in 2005. The WHO predicts that by 2030, it will be the third largest cause of mortality worldwide.

Since COPD is not diagnosed until it becomes clinically apparent, prevalence and mortality data greatly underestimate the socioeconomic burden of COPD.

According to Datamonitor, there are 16 million people diagnosed with COPD in the U.S., and more than 30 million people are affected with COPD in the seven major pharmaceutical markets. In 2005 there were more than 10million physician office visits and two million hospitalizations per year. The disease was estimated to cost the U.S. healthcare system U.S.$30 billion in 2000. According to a report by Datamonitor,worldwide sales in 2004 of the top seven respiratory therapeutics indicated for COPD were U.S.$4.8 billion.

Management of COPD generally involves bronchodilators and steroids. However, only an estimated 20% of patients respond positively to steroids and it is currently not practical to determine in advance which patients will respond to steroids. We believe that only half of moderate and severe COPD patients achieve an adequate treatment outcome. Therefore, as with asthma, we believe there is room to improve both the diagnosis and management of COPD.


Millions of fine hair-like structures that cover the inside lining of our airways and move continuously to propel secretions up to the throat (also see mucociliary clearance )

Clinical Trials

The development of human therapeutic products is a highly regulated process. Evaluation and testing for safety and efficacy proceed through laboratory (research), animal (pre-clinical) and human (clinical) stages of development.

Pharmaxis conducts its preclinical safety evaluation in accordance with the guidelines provided by the International Committee on Harmonisation, which provides test guidelines applicable to the major pharmaceutical territories of the world. These guidelines cover the manufacture of the drug substance, the manufacture of the dosage form, and the safety testing that must be conducted before evaluation in humans can proceed.

Clinical testing involves a three-phase process.

  • In Phase I, clinical trials are conducted with a small number (typically 10-50) of healthy subjects to determine the early safety profile and pharmacokinetic profile (pattern of drug distribution and metabolism).

  • In Phase II, clinical trials are conducted with groups of patients with a specified disease (typically 100-200) to determine preliminary effectiveness, optimal dosages and expanded evidence of safety. This is intended to show that the drug is effective in different patient populations under a variety of doses.

In Phase III, the company conducts large-scale (typically >1,000), multi-centre, comparative clinical trials with patients with the target disease to provide sufficient data to statistically evaluate the effectiveness and safety of the product. During these clinical studies, the manufacture of the drug will be refined and an optimal formulation will be selected. Additional safety studies will be required, including long-term toxicology studies (possibly of 12 months' duration) and carcinogenicity studies. The company also undertakes a detailed study of the pharmacology of the drug to identify any breakdown products and the routes of excretion from the body.


Refer to chronic obstructive pulmonary disease


Any of the steroid hormones produced by the adrenal cortex or their synthetic equivalents. Corticosteroids are used clinically for hormonal replacement therapy, for suppression of adrenocorticotrophic hormone (ACTH) secretion by the anterior pituitary, as anti-cancer and anti-allergic and anti-inflammatory agents and to suppress the immune response. They may be injected, taken as pills, inhaled via a puffer or rubbed on to the skin.

Cystic Fibrosis (CF)

Cystic fibrosis (CF) is an inherited, life-limiting disease that affects the body's exocrine glands, causing them to secrete fluids that are poorly hydrated and therefore thicker and stickier than fluids in people without CF. This leads to chronic problems in various systems of the body, particularly the lungs.

Dose Responsive Curve

A dose response curve illustrates the relation between the amount of a drug or other chemical administered to a person or an animal and the degree of response it produces.


An endothelial cell layer refers to the layer of cells that lines the blood vessels and airways

Epithelial mast cells

Mast cells are a variety of Leukocytes or white blood cellscontaining granules which store a variety of inflammatory mediators including histamine and serotonin. Mast cells play a central role in inflammatory and immediate allergic reactions. The extracellular release of the mediators is known as degranulation and may be induced by the presence of a specific antigen (allergen). Epithelial mast cells are those found in the epithelium (the membranous tissue composed of one or more layers of cells separated by very little intercellular substance and forming the covering of most internal and external surfaces of the body and its organs. Skin and the lung linings are two examples of epithelium.)

Eucapnic Hyperpnoea

Eucapnic (adjective) is defined as a normal healthy level of carbon dioxide (C02). Hyperpnoea is abnormally fast breathing.

Exercise Challenge Test

A test in which patients undertake a physical activity, such as bike riding, and the body's response to the activity is measured. It can be used to determine if a patient is asthmatic by measuring the degree of bronchial constriction that is induced during a period of exercise.

Exocrine Glands

Glands that produced mucus, saliva, sweat and tears


United States of America's Food and Drug Administration

Flare or Flare-up

Period of worsening symptoms

Head-to-head trial

A clinical trial in which a test compound is evaluated against another compound

Hypertonic Saline

A solution with a higher salt concentration than in normal cells of the body and the blood. A salt solution containing more than 0.9% salt is hypertonic.

In Vitro

In an artificial environment, outside the living body e.g. in a test tube

In Vivo

In the living body of a plant or animal, or in real life

International Committee on Harmonisation (ICH)

An international body that provides test guidelines that cover the manufacture of drug substances, the manufacture of the dosage form, and the safety testing that must be conducted before evaluation in humans can proceed.


Immune cells; white blood cells


A molecule that binds to cell receptors

Lung Function

Ability of a person to move air in and out of their lungs. A measure often used is termed FEV1, which is the volume of air that can be forcibly expelled from the lungs in one second


A type of white blood cell found in the body's lymph, a clear fluid that flows through the body and has an important function in defending the body against disease


Mannitol is a naturally occurring sugar used variously as a food additive, a therapeutic product, and a sweetener.

Marketing Authorisation

The legal authority granted to an individual or company to sell a product


Pooling and examining data from a number of studies

Methacholine Inhalation Test

A test used to diagnose asthma. Aerosolised methacholine is inhaled and causes bronchial constriction in asthmatic patients

Mucociliary clearance

A constant, natural process where the cilialining the lungs move continuously and propel the overlying blanket of salt, water and mucus up to the throat, where secretions are swallowed or expelled as sputum. This helps keep the airways clean, allows the passage of clean, warm air through the lungs, and removes any foreign bodies from the airways, preventing infection.

Mucosal Hydration

The natural process of keeping mucus hydrated to prevent it becoming thick and sticky i.e. maintaining the correct balance of water


Thin, slippery substance secreted by the lungs (and other organs in the body) to defend against germs, dust particles and other foreign bodies

Mutil-Centre Study

Study conducted simultaneously in a number of clinics, hospitals, etc


Primary myelofibrosis is a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar-like material. Over time, this leads to progressive bone marrow failure. Under normal conditions, the bone marrow provides a fine network of fibres on which the stem cells can divide and grow. Specialised cells in the bone marrow known as fibroblasts make these fibres.

In primary myelofibrosis, chemicals released by high numbers of platelets and abnormal megakaryocytes (platelet forming cells) over-stimulate the fibroblasts. This results in the overgrowth of thick coarse fibres in the bone marrow, which gradually replace normal bone marrow tissue. Over time this destroys the normal bone marrow environment, preventing the production of adequate numbers of red cells, white cells and platelets. This results in anaemia, low platelet counts and the production of blood cells in areas outside the bone marrow for example in the spleen and liver, which become enlarged as a result.

Primary myelofibrosis is a rare chronic disorder diagnosed in an estimated 1 per 100,000 population. It can occur at any age but is usually diagnosed later in life, between the ages of 60 and 70 years. The cause of primary myelofibrosis remains largely unknown. It can be classified as either JAK2 mutation positive (having the JAK2 mutation) or negative (not having the JAK2 mutation).

Source: Australian Leukemia Foundation:

Nebulised Medication

Medication delivered to the lungs of patients in fine spray by aerosol or face mask

Non Alcoholic Steatohepatitis (NASH)

The most common cause of chronic liver among children and adults in the western world is non-alcoholic fatty liver disease (NAFLD). NAFLD ranges from simple steatosis to non-alcoholic steatohepatitis (NASH). While NAFL can be quite unobtrusive, NASH is a serious condition, with nearly a quarter of affected patients developing cirrhosis, which, in turn, increases the risk of subsequent progression to hepatocellular carcinoma (HCC). NASH is an often “silent” liver disease. It resembles alcoholic liver disease, but occurs in people who drink little or no alcohol. The major feature in NASH is fat in the liver, along with inflammation and damage. Most people with NASH feel well and are not aware that they have a liver problem. Nevertheless, NASH can be severe and can lead to cirrhosis, in which the liver is permanently damaged and scarred and no longer able to work properly.

NASH is an increasingly common chronic liver disease with worldwide distribution that is closely associated with diabetes and obesity, which have both reached epidemic proportions. It is estimated that there are at least 1.46 billion obese adults worldwide. NASH affects 2 to 5 percent of Americans. An additional 10 to 20 percent of Americans have fat in their liver, but no inflammation or liver damage, a condition called “fatty liver.” Approximately 6 million individuals in the USA are estimated to have progressed to NASH and some 600,000 to NASH-related cirrhosis.

Both NASH and NAFLD are becoming more common, possibly because of the greater number of Americans with obesity. In the past 10 years, the rate of obesity has doubled in adults and tripled in children. Obesity also contributes to diabetes and high blood cholesterol, which can further complicate the health of someone with NASH. Diabetes and high blood cholesterol are also becoming more common among Americans.


NASH is usually first suspected in a person who is found to have elevations in liver tests that are included in routine blood test panels, such as alanine aminotransferase (ALT) or aspartate aminotransferase (AST). When further evaluation shows no apparent reason for liver disease (such as medications, viral hepatitis, or excessive use of alcohol) and when x rays or imaging studies of the liver show fat, NASH is suspected. The only means of proving a diagnosis of NASH and separating it from simple fatty liver is a liver biopsy. Currently, no blood tests or scans can reliably provide this information.


NASH is usually a silent disease with few or no symptoms. Patients generally feel well in the early stages and only begin to have symptoms—such as fatigue, weight loss, and weakness—once the disease is more advanced or cirrhosis develops. The progression of NASH can take years, even decades. The process can stop and, in some cases, reverse on its own without specific therapy. Or NASH can slowly worsen, causing scarring or “fibrosis” to appear and accumulate in the liver. As fibrosis worsens, cirrhosis develops; the liver becomes seriously scarred, hardened, and unable to function normally. Not every person with NASH develops cirrhosis, but once serious scarring or cirrhosis is present, few treatments can halt the progression. A person with cirrhosis experiences fluid retention, muscle wasting, bleeding from the intestines, and liver failure. Liver transplantation is the only treatment for advanced cirrhosis with liver failure, and transplantation is increasingly performed in people with NASH. NASH ranks as one of the major causes of cirrhosis in America, behind hepatitis C and alcoholic liver disease.


Although NASH has become more common, its underlying cause is still not clear. It most often occurs in persons who are middle-aged and overweight or obese. Many patients with NASH have elevated blood lipids, such as cholesterol and triglycerides, and many have diabetes or prediabetes, but not every obese person or every patient with diabetes has NASH. Furthermore, some patients with NASH are not obese, do not have diabetes, and have normal blood cholesterol and lipids. NASH can occur without any apparent risk factor and can even occur in children. Thus, NASH is not simply obesity that affects the liver.

While the underlying reason for the liver injury that causes NASH is not known, factors including insulin resistance, the release of toxic inflammatory proteins by fat cells and oxidative stress (deterioration of cells) inside liver cells are possible candidates:


Currently, no specific therapies for NASH exist. A major attempt should be made to lower body weight into the healthy range. Weight loss can improve liver tests in patients with NASH and may reverse the disease to some extent. People with NASH often have other medical conditions, such as diabetes, high blood pressure, or elevated cholesterol. These conditions should be treated with medication and adequately controlled; having NASH or elevated liver enzymes should not lead people to avoid treating these other conditions.
Experimental approaches under evaluation in patients with NASH include antioxidants, which act by reducing the oxidative stress that appears to increase inside the liver in patients with NASH.

Oral Medication

Medication taken by mouth e.g. tablets, liquids

Osmatic Balance

Osmosis is the passage of water from a region of high water concentration through a semi-permeable membrane to a region of low water concentration. Osmotic balance is when there is no tendency for water to flow across the membrane.


Disease-causing microorganism


Pharmaceutical Benefits Scheme (Australian government program that reduces the cost of some drugs to patients)


Patient Cooperation Treaty 

Pharmaco-economic Evaluation

Evaluation of the potential of a new pharmaceutical product to produce cost savings to a national economy

Pharmacokinetic Profile

How a drug interacts in the body in terms of its absorption, distribution, metabolism, and excretion

Phase II Clinical Trial

Refer to clinical trials above. 

Phase III Registration Study

Refer to clinical trials above.


An inert or innocuous substance used especially in controlled experiments to test and compare the efficacy of another, active, substance

Postural Drainage

A method of draining the lungs in which the patient is placed in an inverted position so that fluids are drawn by gravity


Prior to being administered to volunteers or patients


An enzyme that breaks the internal bonds of a protein

Pulmonary Fibrosis

Pulmonary fibrosis is a serious disease that causes progressive scarring of the lung tissue. The scar formation is preceded by, and associated with inflammation. Some common and some rare diseases can cause pulmonary fibrosis, but in the majority of cases the cause is never found. Pulmonary fibrosis can be a mild or severe disease; it can be so mild as to cause few symptoms, or it can be fatal. If the disease progresses, the lung tissues eventually thicken and become stiff. The work of breathing then becomes difficult, causing breathlessness. It can run a gradual course, remain unchanged or run a rapid course. It can also be fatal. Pulmonary fibrosis most often begins with repeated injury to the tissue within and between the tiny air sacs (alveoli) in your lungs. The damage eventually leads to scarring or fibrosis, which stiffens the lungs and makes breathing difficult. The most common symptoms are shortness of breath and a dry cough.

Current treatments for pulmonary fibrosis include medications and therapy to improve lung function and quality of life. A number of new therapies for pulmonary fibrosis are in clinical trials. In the meantime, a lung transplant may be an option for some people with pulmonary fibrosis.

Pulmonary Function

See lung function

Pulmonary System



A sugar derivative 

R & D

Research and Development 


A recurrence of symptoms of a disease after a period of improvement or remission


Period when the symptoms of the patient's disease are not present

Respiratory Failure

A clinical term used to define the inability of the lungs to function

Respiratory Insufficiency

A clinical term used to define a failure to adequately provide sufficient oxygen to the body, or remove excess carbon dioxide


The study of the flow of materials that behave in an interesting or unusual manner

Safety Profile

Evidence gathered that indicates a substance is safe to be administered to people

Secondary Lung Infections

Infection coming after, or as a result of, an initial or primary infection

Selective Inhibitor

A substance that is used to stop a specific biochemical reaction

Spirometer; Spirometry test

A device used to measure the amount of air a patient can expel from their lungs in one second

Sputum Microbiology

A measure of lung infections

Statistical Significance

A mathematical test that indicates that groups being compared are different


Numerous natural or synthetic compounds that contain a 17-carbon 4-ring system and can modify reactions in the body

Synthesis, Synthetic Compound

A substance that is made by a series of chemical or biochemical reactions


Immune cells that attach themselves to other cells


Australian Therapeutic Goods Administration 


Medicinal, curative

Toxicology Study

Investigation into the adverse effects of a substance in an animal or human

Tumor Necrosis Factor (TNF)

A small molecular-weight protein produced primarily by immune cells. It is a key protein responsible for initiating inflammation


A physical property of fluids that determines the internal resistance to shear forces (the resistance a material has to change in form)