FAIRFAX, Va. - May 15, 2019 - s4story -- A 'one-of-a-kind' medication access program from HAE International (HAEi) is now successfully delivering potentially life-saving medication to patients in South Africa, who suffer from the rare genetic condition hereditary angioedema (HAE).

HAE causes severe and unpredictable swelling (edema) in different areas of the body including the hands, feet, and face. Patients experience bouts of excruciating abdominal pain, nausea, and vomiting caused by gastrointestinal swelling. Laryngeal edema is particularly dangerous and can lead to death by asphyxiation.  The historical mortality for untreated patients can be as high as 30 percent. There is no cure for HAE but effective treatments exist to control symptoms.

No modern medications are commercially available in South Africa therefore until now HAE patients were unable to access them. "The HAEi Global Access Program (HAEi GAP) offers a regulatory compliant process that allows healthcare professionals to offer treatment for their HAE patients," says Henrik Balle Boysen, Executive Vice President & Chief Operating Officer of HAE International.  "We are delighted to have initiated HAEi GAP in South Africa where patients will now have the option to access an effective therapy to treat painful, debilitating, and potentially life threatening HAE swellings."

The first shipment of medication to South Africa was requested by Associate Professor Jonny Peter from Groote Schuur Hospital and the University of Cape Town Lung institute Allergy and Immunology unit, Cape Town. "It is wonderful to finally be able to access a rapid, targeted treatment for our HAE patients. The HAEi GAP provides an access route to much-needed targeted treatments at reasonable costing that is fundamental to HAE patients having the freedom to live their own life and to be in control of HAE" commented Dr Peter.

The Netherlands based Pharming is the HAEi GAP pharmaceutical company partner. Mischa Boeijen, Sr. Product Manager Ruconest® of Pharming said "Pharming is extremely privileged to be part of the Global Access Program, a successful collaboration project initiated with HAEi. We are delighted to be able to provide Ruconest®, a potentially life-saving therapy to those patients in South Africa who would otherwise not be able to access it. Pharming looks forward to extending our reach to patients across the globe in the future."

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Inceptua is supporting HAEi and Pharming as a specialist service provider. Mark Corbett, Executive Vice-President of Inceptua Medicines Access said "Helping to ensure access to treatments for patients in need – where suitable products are not commercially available – is at the very core of what we do. At Inceptua we are pleased to be supporting HAEi on their critical mission to facilitate access to HAE therapies through the HAEi Global Access Program".

For HAE patients in South Africa who would like to find out more about HAE and support in South Africa, please visit the HAE South Africa website - https://haei.org/southafrica/

̶̶  Ends –

Contacts:
Henrik Balle Boysen
EVP & COO, HAE International
E:  h.boysen@haei.org
P: +45 31 591 591

Deborah Corcoran
Chief Specialist Projects & Research, HAE International
E:  d.corcoran@haei.org
P: +44 7780 608 797

Notes to Editors:

About HAE
Hereditary Angioedema (HAE) is a rare and potentially life-threatening genetic condition that affects from around 1 in 50,000 people. HAE symptoms include episodes of swelling (edema) in various body parts including the hands, feet, face and airway. Edema of the skin usually affects the extremities, the face, and the genitals.  Almost all patients experience bouts of excruciating abdominal pain, nausea and vomiting that is caused by swelling in the intestinal wall. Edema of the throat is particularly dangerous and can lead to death by asphyxiation. Although there is currently no known cure for HAE, it is possible to treat the symptoms associated with edema attacks. Although HAE is (in principle) easy to diagnose, it is frequently identified very late in life or misdiagnosed because symptoms are similar to those of many other common conditions such as allergies or appendicitis.

About The HAEi Global Access Program (GAP)
The HAEi GAP is a named patient program which means a doctor is required to request medication for a specific patient. Legal organization and compliant distribution of requested medication is handled by a medicines access solutions company who respond directly to the requests. Once the request has been checked and verified, the shipment is addressed and sent to the requesting healthcare professional, who is then able to prescribe the medication to their patients. The cost of the medication is then reimbursed by the government, hospital or the patient's medical insurance. 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.

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About HAE International (HAEi)
HAEi is the international umbrella organization for the world's Hereditary Angioedema (HAE) patient groups. Our organization is a global non-profit network of patient associations and we are dedicated to raising awareness of C1-inhibitor deficiencies around the world. We strive to improve time to diagnosis and facilitate access to and reimbursement of life saving HAE therapies, which will enable lifelong health for all patients – no matter where they live.  HAEi currently has member organizations in 74 countries. For more information, please visit: www.haei.org

About Pharming
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, Israel and South Korea. The product is available on a named-patient basis in other territories where it has not yet obtained marketing authorization.