AGM Annual General Meeting of Shareholders.
AMI Acute Myocardial Infarction, commonly known as a heart attack, results from the interruption of
blood supply to a part of the heart causing heart cells to die. Heart attacks are the leading cause of death
for both men and women worldwide.
AMR Antibody-mediated rejection occurs when a transplant because of suboptimal histo-compatibility,
is perceived by the recipient as a foreign body. The immune system is activated and the foreign body is
attacked, which can lead to organ failure and immunological rejection of the organ.
BLA In the US, pharmaceuticals are approved for marketing under the provisions of the Public Health
Service (PHS) Act. The Act requires a firm which manufactures a pharmaceutical for sale in interstate
commerce to hold a license for the product. To commercialise a new biological product in the US, the FDA
needs to approve a Biologics License Application (BLA). A BLA is a submission that contains specific
information on the manufacturing processes, chemistry, pharmacology, clinical pharmacology and the
medical affects of the biologic product. If the information provided meets FDA requirements, the
application is approved and a license is issued allowing the company to market the pharmaceutical.
Biological products include amongst others monoclonal antibodies, growth factors, blood products and
proteins intended for therapeutic use. The concerning FDA centre is the Center for Biologics Evaluation
and Research (CBER).
BOM The Board of Management of Pharming Group N.V.
C1INH C1 esterase inhibitor or C1INH is a serine protease inhibitor protein present in human blood
serum. C1INH is involved in the regulation of the first protein in the complement system (C1), which is
part of the immune system. Insufficient C1 inhibitor action or amounts can cause inflammation and HAE
attacks.
CHMP The Committee for Medicinal Products for Human Use (CHMP) plays a vital role in the marketing
procedures for medicines in the European Union. Amongst others, the CHMP is responsible for preparing
the EMA’s opinions on all questions concerning medicinal products for human use, in accordance with
Regulation (EC) No 726/2004.
Clinical trial/studies
Clinical trials are tests on human individuals, ranging from healthy people to patients, to evaluate safety
and efficacy of new pharmaceutical products before they can be approved. Clinical trials typically range
from Phase I to Phase IV and even V.
CMO A Contract Manufacturing Organisation (CMO) is an organisation that provides clients from the
pharmaceutical industry with comprehensive services from drug development through manufacture.
DGF A DGF or delayed graft function is a common complication affecting all solid organs in the posttransplant period. DGF results in significant morbidity and mortality from early graft dysfunction and
from decreased long-term graft survival. The condition also prolongs hospitalisation and requires
substitute therapies for these patients, such as dialysis or ventilation support. DGF remains a critical
unmet medical need despite improvements in immunosuppression, organ preservation, and surgical
technique. C1 inhibitor has been shown in numerous models of organ transplantation to improve early
graft function. In the USA alone, over 25,000 solid organs were transplanted in 2005, including kidney,
liver, lung and heart transplants.
EGM Extraordinary General Meeting of Shareholders.
EMA The European Medicines Agency (EMA) is the regulatory office for pharmaceuticals in the
European Union and is responsible for approving new drugs prior to marketing of the product ensuring
their safety and efficacy.
FDA The FDA or Food and Drug Administration is the regulatory office responsible for drug approval in
the United States.
GMP GMP status or Good Manufacturing Practice is a term that is recognised worldwide for the control
and management of manufacturing and quality control testing of foods and pharmaceutical products.
HAE HAE or Hereditary Angioedema is a human genetic disorder caused by insufficient activity of the C1
inhibitor protein. HAE patients suffer from recurrent unpredictable acute attacks of painful and in some
cases fatal swelling of soft tissues (edema), including regions of the skin, abdomen and the mouth and
throat. Attacks can last up to five days when untreated. In the Western world, approximately 1 in 30,000
individuals suffers from Hereditary Angioedema, having an average of seven acute attacks per year.
HAEI Hereditary Angioedema International (patient organisation).
hLF Human lactoferrin is a natural protein that helps to fight and prevent infections. The protein is
present in substantial quantities in mother’s milk and plays an important role in the defense system of
infants. The protein is also present in various body fluids and continues to play an important role against
a wide range of bacterial, fungal and viral pathogens in adults. Pharming produces a recombinant version
of the natural lactoferrin protein.
IFRS, IAS and IASB
International Financial Reporting Standards (IFRS) along with International Accounting Standards (IAS)
are a set of accounting standards issued by the International Accounting Standards Board (IASB).
IND An IND (investigational new drug application) is the vehicle through which a sponsor advances to
the next stage of drug development known as clinical trials (human trials).
IRI Ischaemia Reperfusion Injury (IRI) is a complication arising from lack of oxygen due to an
interruption of the blood supply (ischaemia) resulting in tissue damage. This can occur in a transplanted
organ, in the brain in case of stroke, and in the heart in case of myocardial infarction ('heart attack').
LTIP Pharming’s Long Term Incentive Plan.
MAA A Marketing Authorisation Application is a request for market approval in the European Union.
Orphan Drug A drug being developed to treat a rare disease (affecting less than 200,000 individuals in
the USA) can receive Orphan Drug designation from the FDA. This status is granted under the US Orphan
Drug Act of 1983, which was established to encourage, support and protect the development of treatment
for rare, but serious diseases. Orphan Drug status provides several advantages including market
exclusivity for seven years, various financial incentives and a well-defined regulatory approval path. The
EMA can grant a similar status to products being developed to treat rare diseases (affecting not more than
five in ten thousand persons in Europe), namely Orphan Medicinal Product. This status is granted under
European Parliament and Council Regulation (EC) No 141/2000 of 16 December, 1999, on Orphan
Medicinal Products, which introduces incentives for Orphan Medicinal Products research, development
and marketing, in particular by granting exclusive marketing rights for a ten-year period.
POC A Proof of Concept (POC) is a study to verify that a concept or theory has the potential of being used.
Protein Proteins are large organic molecules, like C1 inhibitor, fibrinogen and collagen, and form the
basis to all living organisms. They are composed of one or more chains of amino acids joined together by
peptide bonds. The sequence of these amino acids is defined by genes, which are present in the DNA.
Recombinant Recombinant refers to the combination of genetic material (DNA) from different
biological sources. Pharming, like all biotechnology firms, uses recombinant technology to produce
proteins such as recombinant human C1 inhibitor.
rhC1INH Recombinant human C1 esterase inhibitor or rhC1INH is the active component of
Ruconest®/Rhucin®. Natural C1 inhibitor DNA from a human source is used in Pharming’s protein
production technology to ensure expression of the C1 inhibitor protein. This product might be useful for
certain indications, such as the prevention of complications that sometimes arise after organ
transplantation.
rhFVIII Recombinant human Factor VIII is a natural human blood clotting factor and is in early-stage
development for treatment of Haemophilia A. Haemophilia A is a hereditary disorder caused by defects in
the Factor VIII gene. Lack of functional Factor VIII diminishes the body’s clotting ability, which in turn
can lead to damaging- or fatal bleeding episodes. On this project, Pharming has a service agreement with
Renova Life.
Rhucin® Rhucin® is the global registered trade mark for Pharming’s recombinant human C1 inhibitor
and has been renamed Ruconest®
RLI Renova Life, Inc.
Ruconest® Ruconest® is the global registered trade mark for Pharming’s recombinant human C1
inhibitor. Human C1 inhibitor is a protein involved in the regulation of the first protein in the complement
system (C1), which is part of the immune system. Insufficient C1 inhibitor action or amounts can cause
inflammation and HAE attacks.
Sobi Swedish Orphan Biovitrum International AB.
SPA A Special Protocol Assessment (SPA) is a declaration from the FDA that an uncompleted Phase III
trial's design, clinical endpoints, and statistical analyses are acceptable for FDA approval.
Transgenic An organism is called transgenic when its cells carry genetic material from another species
in addition to its own genetic material. Pharming produces specific human products in the milk of
transgenic rabbits and cows carrying the human recombinant gene responsible for expressing that
product.
VWAP Volume Weighted Average Price of shares.