LEIDEN, the Netherlands and
BURTON-ON-TRENT, England,
July 21, 2015 /PRNewswire/ --
Unique patient
organization-driven
program is
enabling
people with HAE who
cannot access effective treatments
to receive medication for the
first time
Pharming Group N.V. ("Pharming or the Company"), Euronext:
PHARM) and Clinigen Group plc ('Clinigen' or the 'Group', AIM:
CLIN) today announce that their global collaboration to provide
access to Pharming's RUCONEST® (conestat alfa) is now live. The
unique access program was initiated by HAEi, the International
Patient Organization for C1- Inhibitor Deficiencies.
RUCONEST is a recombinant human C1- inhibitor, approved by the
European Medicines Agency (EMA) and US Food and Drug Administration
(FDA) for the treatment of acute attacks of hereditary angioedema
(HAE). The "HAEi GAP" program enables patients in all countries
where RUCONEST is not commercially available to gain access to the
drug through an ethical and regulatory compliant "Named Patient
Program" mechanism.
Physicians wishing to request RUCONEST through the HAEi GAP
program should contact the Clinigen customer services team at
customer.services@clinigengroup.com / +44 1283 494340.
"HAEi is committed to securing access to HAE medications for
patients across the globe," said Anthony J.
Castaldo, President of HAEi. "We are extremely proud to have
established HAEi GAP with our current partners and to be able to
announce that, from today, physicians are able to request RUCONEST
to meet the needs of their patients."
Sijmen de Vries, CEO, Pharming said: "As the first
pharmaceutical company to partner with HAEi and provide access to
RUCONEST through HAEi GAP, we are leading the charge to improve the
lives of those HAE patients that otherwise would continue suffering
from this debilitating and unpredictable disease."
Simon Estcourt, Managing
Director, Managed Access, Clinigen Group said: "We are very pleased
to work with Pharming to help HAEi realize their mission to ensure
that sufferers of hereditary angioedema worldwide can access this
effective and potentially life-saving treatment. The
ground-breaking program provides RUCONEST to patients in an ethical
and compliant way, removing the need and the risk for patients to
resort to other less reliable or even illegal sources of the
drug."
About HAE
Hereditary Angioedema (HAE) is a rare genetic disorder. It 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 edema 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® (C1 Esterase Inhibitor [Recombinant]) 50 IU/kg is an
injectable medicine that is used to treat acute angioedema attacks
in adult and adolescent patients with hereditary angioedema (HAE).
HAE is caused by a deficiency of the C1 esterase inhibitor protein,
which is present in blood and helps control inflammation (swelling)
and parts of the immune system. A shortage of C1 esterase inhibitor
can lead to repeated attacks of swelling, pain in the abdomen,
difficulty breathing and other symptoms. RUCONEST® contains C1
esterase inhibitor at 50 IU/kg.
When administered at the onset of HAE attack symptoms at the
recommended dose, RUCONEST works to return a patient's C1-INH
levels to normal range and quickly begins to relieve the symptoms
of an HAE attack with a low recurrence of symptoms. RUCONEST is the
first and only plasma-free, recombinant C1-INH approval from the
U.S. Food and Drug Administration (FDA) and was approved in
July 2014 and by the European
Medicines Agency (EMA) in October
2010.
About Global Access Programs
Global Access Programs provide biopharmaceutical companies with
a way to allow ethical access to their pre-license/unlicensed
medicines to help patients with unmet medical needs. Access is
provided in response to physician requests, in a fully compliant
manner, where no alternative treatment options are available.
About HAEi
HAEI - International Patient Organization for C1 inhibitor
Deficiencies - is a global organization dedicated to raising
awareness of C1 inhibitor deficiencies around the world. It is a
non-profit international network of national HAE patient
Associations. HAEI is established to promote co-operation,
co-ordination and information sharing between HAE Specialists and
National HAE Patient Associations in order to help facilitate the
availability of effective diagnosis and management of C1 inhibitor
deficiencies throughout the world. Our purpose is to join the
efforts and experience of the global HAE community to achieve
optimal standards of care and treatment for all those patients
affected by C1 inhibitor deficiencies.
For more information, please visit: http://www.haei.org
About Pharming Group NV
Pharming Group NV is developing innovative products for the
treatment of unmet medical needs. RUCONEST® (conestat alfa) is a
recombinant human C1 esterase inhibitor approved for the treatment
of angioedema attacks in patients with HAE in the USA, Israel
all 28 EU countries plus Norway,
Iceland, and Liechtenstein.
RUCONEST is commercialised by Pharming in Austria, Germany and The
Netherlands. 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 partnered with Salix Pharmaceuticals, Ltd. ("Salix")
in North America. Salix is part of
Valeant Pharmaceuticals International, Inc. (NYSE: VRX/TSX:
VRX)
RUCONEST is also being investigated in a randomised Phase II
clinical trial for prophylaxis of HAE, 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 has a unique GMP compliant, validated platform for the
production of recombinant human proteins that has proven capable of
producing industrial volumes of high quality recombinant human
protein in a more economical way compared to current cell-based
technologies. Leads for Enzyme Replacement Therapy (ERT) in Pompe,
Fabry's and Gaucher's diseases are under early evaluation. The
platform is partnered with Shanghai Institute of Pharmaceutical
Industry (SIPI), a Sinopharm Company, for joint global development
of new products. Pre-clinical development and manufacturing will
take place at SIPI and are funded by SIPI. Pharming and SIPI
initially plan to utilise this platform for the development of
recombinant human Factor VIII for the treatment of Haemophilia A.
For more information, please visit http://www.pharming.com
About Clinigen Group
Clinigen Group is a global pharmaceutical and services company
with a unique business model dedicated to delivering the right drug
to the right patient at the right time. The Group consists of four
complementary businesses that provide medicines to patients with
unmet need through clinical trials, licensed and unlicensed supply;
Clinigen Specialty Pharmaceuticals (SP), Clinigen Clinical Trial
Services (CTS), Idis Managed Access (MA) and Idis Global Access
(GA).
We are global leaders in ethically sourcing and supplying
unlicensed medicines to hospital pharmacists and doctors for
patients with a high unmet need. Our Managed Access business
provides early access to unlicensed investigational medicines on
behalf of pharmaceutical companies.
For more information, please visit http://www.clinigengroup.com
Contact Details
Clinigen Group
Peter George, Group Chief Executive Officer
Shaun Chilton, Group Chief Operating Officer
Tel: +44(0)1283-495-010
Instinctif Partners (Clinigen media relations)
Melanie Toyne-Sewell/Jen Lewis/Emma Barlow
Tel: +44(0)20-7457-2020
Email: clinigen@instinctif.com
Pharming Group
Sijmen de Vries, Chief Executive Officer
Tel: +31-71-5247400
FTI Consulting (Pharming media relations)
Julia Phillips/Victoria Foster Mitchell
Tel: +44(0)20-3727-1136
PRN NLD