Landmark global program opens access to HAE medicine to rare disease sufferers for the first time
Lausanne, Switzerland – 16 May 2015 – Thousands of patients suffering from a rare and potentially fatal genetic disease will have access to medicines for the first time, with the launch of the HAEi Global Access Program (HAEi GAP), a “first-of-its-kind” medication access program.
Driven by HAEi, the International Patient Organization for C1 inhibitor Deficiencies, the new program is the first Global Access Program in hereditary angioedema (HAE), a rare inherited blood disease which leads to attacks of swelling. HAE can be life threatening due to risk of suffocation from swelling attacks in the throat. There is no cure but effective treatments exist to prevent and ease attacks, but these treatments are not licensed or available in all countries worldwide.
“The HAEi Global Access Program brings the opportunity of access to HAE medications in countries where innovative effective therapies are otherwise unavailable,” said Henrik Balle Boysen, Executive Director HAEi. “This is the first program of its kind in HAE, and we believe the first time this type of program has been initiated by a patient organization.”
To realize the program, HAEi has partnered with Clinigen Group plc’s Global Access Programs business/division to manage the distribution and logistics for medications in the HAEi GAP. The first HAE therapy available through the program is the human recombinant C1-inhibitor Ruconest® (conestat alfa) manufactured by Pharming Group N.V. HAEi GAP expects to go live in a matter of weeks.
“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 will continue to work with other manufacturers with the hope of expanding the program in the future.”
HAEi GAP follows ethical and regulatory compliant mechanisms and enables patients to gain access to Ruconest through a Named Patient Program (where Ruconest is currently not commercially available). Individuals with a confirmed diagnosis of HAE may be eligible for HAEi GAP. Their doctors can apply to HAEi GAP via Clinigen customer services (firstname.lastname@example.org).
“It is a desperate situation for patients in countries without access to licensed HAE therapies. We hear of patients resorting to the black market or international pharmacy websites to try and access medicine”, advised Henrik Balle Boysen. “HAEi GAP offers legitimate access to effective medicine for eligible patients with the security of an established logistics and supply chain to ensure the right medicine reaches the right patient whenever, and wherever they need it”.
Henrik Balle Boysen, Executive Director, HAEi
Email: email@example.com – Phone: +45 31 591 591
Anthony J. Castaldo, President, HAEi
Email: firstname.lastname@example.org – Phone: +1 202 247 8619
Contact for physicians:
Clinigen customer services:
Phone: +44 1283 494 340
Notes to Editors:
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 hae day :-)
On 16 May every year the hae day :-) aims to raise awareness of HAE among the general public and medical community in order to ensure better care, earlier and more accurate diagnosis, and knowledge that HAE patients can lead a healthy life. National member organizations and national HAE patient groups around the world support the event. For more information, please visit http://www.haeday.org
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: 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 27 EU countries plus Norway, Iceland, and Liechtenstein. RUCONEST is commercialized 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. Valeant Pharmaceuticals International, Inc. (NYSE: VRX/TSX: VRX) completed its acquisition of Salix Pharmaceuticals, Ltd. on April 1, 2015.
RUCONEST is also being investigated in a randomized 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 utilize this platform for the development of recombinant human Factor VIII for the treatment of Hemophilia A.
For more information, please visit http://www.pharming.com
About Clinigen Group plc
The Clinigen Group is a specialty global pharmaceutical company headquartered in the UK, with offices in the US and Japan. The Group is dedicated to delivering ‘the right drug, to the right patient at the right time’. In April 2015, Clinigen acquired Idis, the market leader in the global supply of unlicensed medicines. The Group now operates as four synergistic businesses; Clinigen Specialty Pharmaceuticals, Clinigen Clinical Trials Supply, Idis Managed Access Programs (this now includes the previously branded Clinigen Global Access Programs business) and Idis General Access.
The Managed Access Programs business develops and implements exclusive access programs for biotechnology and pharmaceutical companies and has provided physicians with an ethical solution to access unlicensed medicines for thousands of patients with an unmet medical need.
For more information, please visit www.clinigengroup.com