LEIDEN, The Netherlands,
November 14, 2016 /PRNewswire/ --
Pharming Group N.V. (the "Company") (EURONEXT: PHARM) today
announced that the results of its "Randomized, Double-Blind,
Placebo-Controlled Trial of Recombinant Human C1 Inhibitor for
Prophylaxis of Hereditary Angioedema Attacks", were presented by
Marco Cicardi, Professor of Internal
Medicine University of Milan,
Hospital L. Sacco Milan and
co-prinicipal investigator for the study. The presentation was held
during the American College of Allergy, Asthma and Immunology 2016
Scientific Meeting ("ACAAI") meeting in San Francisco yesterday afternoon. The
presentation can be found on our website, here.
About HAE
Hereditary Angioedema (HAE) is a rare genetic disorder. The
condition is caused by a deficiency of the C1 esterase inhibitor
protein, which is normally present in blood and helps control
inflammation (swelling) and parts of the immune system. Because
defective C1-Inhibitor does not adequately perform its regulatory
function, a biochemical imbalance can occur and produce unwanted
peptides that induce the capillaries to release fluids into
surrounding tissue, thereby causing swelling or edema.
HAE is characterized by spontaneous and recurrent episodes of
swelling (edema attacks) of the skin in different parts of the
body, as well as in the airways and internal organs. Edema of the
skin usually affects the extremities, the face, and the genitals.
Patients suffering from this kind of edema often withdraw from
their social lives because of the disfiguration, discomfort and
pain these symptoms may cause. Almost all HAE patients suffer from
bouts of severe abdominal pain, nausea, vomiting and diarrhea
caused by swelling of the intestinal wall.
Edema of the throat, nose or tongue is particularly dangerous
and potentially life-threatening and can lead to obstruction of the
airway passages. Although there is currently no known cure for HAE,
it is possible to treat the symptoms associated with angioedema
attacks. HAE affects about 1 in 10,000 to 1 in 50,000 people
worldwide. Experts believe that a lot of patients are still seeking
the right diagnosis: although HAE is (in principle) easy to
diagnose, it is frequently identified very late or not discovered
at all. The reason HAE is often misdiagnosed is because the
symptoms are similar to those of many other common conditions such
as allergies or appendicitis. By the time it is diagnosed
correctly, the patient has often been through a long-lasting
ordeal.
About RUCONEST®
RUCONEST® (recombinant C1 esterase inhibitor) is indicated for
the treatment of acute attacks in adult and adolescent patients
with hereditary angioedema (HAE). Effectiveness in clinical studies
was not established in HAE patients with laryngeal attacks.
RUCONEST contains C1 esterase inhibitor at 50 IU/kg. When
administered at the onset of HAE attack symptoms at the recommended
dose, RUCONEST may help to return a patient's C1 esterase inhibitor
levels to normal range and relieve the symptoms of an HAE attack
with a low recurrence of symptoms within 24 hours.
RUCONEST is the only recombinant C1 esterase inhibitor approved
by the U.S. Food and Drug Administration (FDA) and was approved in
July 2014.
Recently RUCONEST® demonstrated very positive data for
prophylaxis (prevention) of acute attacks of HAE in patients with
HAE. If approved for this indication, RUCONEST® will have
access to this additional market. RUCONEST® therefore, has the
potential to be the only recombinant C1 esterase inhibitor product
approved to target both the acute market (worth approximately
$845 million) and the HAE prophylaxis
market (separately worth approximately $700
million).
Please see Prescribing Information for RUCONEST® as
applicable for various jurisdictions
EMA:
http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/001223/WC500098542.pdf
FDA:
http://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM405634.pdf
About Pharming Group N.V.
Pharming is a specialty pharmaceutical company developing
innovative products for the safe, effective treatment of rare
diseases and unmet medical needs. Pharming's lead product,
RUCONEST® (conestat alfa) is a recombinant human C1 esterase
inhibitor approved for the treatment of acute Hereditary Angioedema
("HAE") attacks in patients in Europe, the US and rest of the world. The
product is available on a named-patient basis in other territories
where it has not yet obtained marketing authorization.
RUCONEST® is commercialized by Pharming in Algeria, Andorra, Austria, Bahrain, Belgium, France, Germany, Ireland, Jordan, Kuwait, Lebanon, Luxembourg, Morocco, Netherlands, Oman, Portugal, Qatar, Syria,
Spain, Switzerland, Tunisia, United Arab
Emirates, United Kingdom
and Yemen.
RUCONEST® is distributed by Swedish Orphan Biovitrum AB (publ)
(SS: SOBI) in the other EU countries, and in Azerbaijan, Belarus, Georgia, Iceland, Kazakhstan, Liechtenstein, Norway, Russia, Serbia, and Ukraine.
RUCONEST® is distributed in the United
States by a subsidiary of Valeant Pharmaceuticals
International, Inc. (NYSE: VRX/TSX: VRX), following Valeant's
acquisition of Salix Pharmaceuticals, Ltd.
RUCONEST® is distributed in Argentina, Colombia, Costa
Rica, the Dominican
Republic, Panama and
Venezuela by Cytobioteck, in
South Korea by HyupJin Corporation
and in Israel by Megapharm.
RUCONEST® is also being investigated in a Phase II clinical
trial for the treatment of HAE in young children (2-13 years of
age) and evaluated for various additional follow-on
indications.
Pharming's technology platform includes a unique, GMP-compliant,
validated process for the production of pure recombinant human
proteins that has proven capable of producing industrial quantities
of high quality recombinant human proteins in a more economical and
less immunogenetic way compared with current cell-line based
methods. Leads for enzyme replacement therapy ("ERT") for Pompé and
Fabry's diseases are being optimized at present, with additional
programs not involving ERT also being explored at an early stage at
present.
Pharming has a long term partnership with the China State
Institute of Pharmaceutical Industry ("CSIPI"), a Sinopharm
company, for joint global development of new products, starting
with recombinant human Factor VIII for the treatment of Haemophilia
A. Pre-clinical development and manufacturing will take place to
global standards at CSIPI and are funded by CSIPI. Clinical
development will be shared between the partners with each partner
taking the costs for their territories under the partnership.
Pharming has declared that the
Netherlands is its "Home Member State" pursuant to the
amended article 5:25a paragraph 2 of the Dutch Financial
Supervision Act.
Additional information is available on the Pharming website:
http://www.pharming.com
Forward-looking Statements
This press release of Pharming Group N.V. and its
subsidiaries ("Pharming", the
"Company" or the
"Group") may contain forward-looking
statements including without limitation those regarding
Pharming's financial projections, market
expectations, developments, partnerships, plans, strategies and
capital expenditures.
The Company cautions that such forward-looking statements may
involve certain risks and uncertainties, and actual results may
differ. Risks and uncertainties include without limitation the
effect of competitive, political and economic factors, legal
claims, the Company's ability to protect intellectual
property, fluctuations in exchange and interest rates, changes in
taxation laws or rates, changes in legislation or accountancy
practices and the Company's ability to identify,
develop and successfully commercialize new products, markets or
technologies.
As a result, the Company's actual performance,
position and financial results and statements may differ materially
from the plans, goals and expectations set forth in such
forward-looking statements. The Company assumes no obligation to
update any forward-looking statements or information, which should
be taken as of their respective dates of issue, unless required by
laws or regulations.
Contacts:
Pharming Group N.V.
Sijmen de Vries, CEO, Tel: +31-71-524-7400
Bruno Giannetti, COO, Tel:
+31-71-524-7400
FTI Consulting:
Julia Phillips/ Victoria Foster Mitchell, Tel:
+44(0)203-727-1136
Lifespring Life Sciences Communication
Leon Melens, Tel:
+31-20-705-95-90
PRN NLD