Leiden, the Netherlands and LUXEMBOURG – 26 Sept 2017 – Pharming Group N.V in association with HAEi, the international umbrella organization for the world’s Hereditary Angioedema (HAE) patient groups, announce the appointment of Inceptua Medicines Access as their new distribution partner for the “HAEi Global Access Program” (HAEi GAP) enabling patients in all countries where Pharming’s product RUCONEST^® is not commercially available to gain access to the drug through an ethical and regulatory compliant mechanism.  It is the only known program of this type which has been initiated through a patient group.

The program is the only Global Access Program in hereditary angioedema (HAE), a very rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 (1) to 1 in 50,000 people (2) HAE symptoms include attacks of swelling in various body parts, including the hands, feet, face, intestinal walls and airway. Swelling in the airway is particularly dangerous and can lead to death by asphyxiation. (2) There is currently no cure for HAE, but certain treatments exist to prevent and ease attacks. These treatments are not licensed or available in all countries worldwide, leaving patients unable to prepare for and treat attacks.

RUCONEST® is the first treatment to be made available through the HAEi GAP program in countries where it is not commercially available. 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) (3,4)

Physicians wishing to request RUCONEST® for their patients through the HAEi GAP program should contact  HAEiGAP@inceptua.com or alternatively ring +44 20 3910 7670. Please note that direct patient inquiries cannot be handled.

“HAEi exists to support patients with HAE gain access to HAE therapies to improve their quality of life and potentially be life-saving in the case of a laryngeal attack,” commented Henrik Balle Boysen, HAEi Executive Director. “Through this partnership with Pharming Group N.V. and Inceptua Medicines Access, physicians, who may otherwise have no access to modern HAE therapies, have the opportunity to request RUCONEST for their patients, effective immediately.”

“We are delighted to announce, in association with HAEi, that Inceptua Medicines Access are our new trusted distribution partner, offering a very tailored solution, providing navigation support to physicians in an ethical and regulatory compliant way to gain access to this urgently-required treatment for their patients,” said Paul Janssen, Pharming Group N.V., Vice-President Commercial Operations Europe and ROW. 

Mark Corbett, Executive Vice-President, Inceptua Medicines Access commented:

“We are providing a transformational approach to medicines access, we will strive to provide unrivalled service in navigating access to RUCONEST® for the treatment of patients in need. We are delighted to work with both HAEi and Pharming in delivering what is the only known Medicines Access Program instigated by a patient organization. We will navigate access to this medicine with agility, quality and regulatory compliance, ultimately to help the treatment of patients in need.”

Contacts:

Mark Corbett
Executive Vice President, Inceptua Medicines Access
Medicinesaccess@inceptua.com
+44 207 907 9200

Pharming Group N.V.
Sijmen de Vries, CEO, Tel: +31 71 524 7400
Robin Wright, CFO, Tel: +31 71 524 7400

References:

1. Agostoni A, Aygoren-Pursun E, Binkley KE, et al. Hereditary and acquired angioedema: problems and progress: proceedings of the third C1 esterase inhibitor deficiency workshop and beyond. J Allergy Clin Immunol. 2004 Sep;114(3 Suppl):S51-131

2. Bowen T, Cicardi M, Bork K, et al. Hereditary angioedema: a current state-of-the-art review, VII: Canadian Hungarian 2007 International Consensus Algorithm for the Diagnosis, Therapy, and Management of Hereditary Angioedema. Ann Allergy Asthma Immunol. 2008 Jan;100(1 Suppl 2):S30-40

3. EMA. RUCONEST SmPC.
http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-Product_Information/human/001223/WC500098542.pdf Last accessed June 2017

4. FDA. SPC. https://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM405634.pdf Last accessed June 2017

5. Zuraw BL. Clinical Practice. Hereditary angioedema. N Engl J Med. 2008 Sep 4;359(10):1027-36

6. Lumry WR, Castaldo AJ, Vernon MK, et al. The Humanistic Burden of Hereditary Angioedema: impact on health-related quality of life, productivity and depression. Allergy Asthma Proc. 2010 Sep-Oct;31(5):407-14

7. Bork K, Stauback P, Eckardt AJ, et al. Symptoms, course and complications of abdominal attacks in hereditary angioedema due to C1 inhibitor deficiency. Am J Gastroenterol. 2006 Mar;101(3):619-27