OREANDA-NEWS. Clinigen Group plc ("Clinigen" or the "Group", AIM: CLIN) and Pharming Group N.V. ("Pharming or the Company", Euronext: PHARM) have entered into an international global access collaboration for HAEi, the International Patient Organization for C1- Inhibitor Deficiencies. The "HAEi GAP" will provide access to RUCONEST® (conestat alfa) to eligible patients with hereditary angioedema (HAE), who currently do not have access to effective medication to treat acute attacks of the disease.

HAEi GAP is the first global access program for HAE and the first to be driven by a patient organization. The program enables patients in countries where RUCONEST is not commercially available to gain access to the drug through an ethical and regulatory compliant "Named Patient Program" mechanism.

"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."

Simon Estcourt, Managing Director, Managed Access Programs, Clinigen Group said: "HAEi's ground-breaking program will ensure that HAE sufferers worldwide can gain access to effective and potentially life-saving treatment. Our secure supply chain and regulatory expertise will enable us to work closely with Pharming to supply patients with RUCONEST ethically and legitimately, removing the need and the risk for patients to resort to other less reliable or even illegal sources of the drug. We look forward to working with both HAEi and Pharming on this unique project."

Sijmen de Vries, CEO, Pharming said: "We are delighted to be the first partners of HAEi and to initiate this program by providing access to RUCONEST, a recombinant human C1- inhibitor, approved by EMA and FDA for the treatment of acute attacks of HAE in patients. In this way we can help to improve the lives of those HAE patients that otherwise would continue suffering from this debilitating and unpredictable disease."

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/ conestat alfa) 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.