Positive CHMP opinion to change terms of marketing authorisation for Ruconest®

Friday, February 26, 2016

Pharming Group N.V. announced that following evaluation of a dossier submitted by the Company last year, the CHMP has issued a positive opinion to the European Commission on Pharming’s request to include the treatment of HAE attacks in adolescents with HAE and to remove the requirements for rabbit IgE testing that forms part of the EU label for RUCONEST®.

Following adoption of the CHMP opinion by the European Commission, this will mean that adolescents with HAE now also have access to (non- blood derived) recombinant C1- inhibitor therapy for the treatment of their angioedema attacks. In addition, the requirement to test HAE patients for pre-existing antibodies against rabbit dander, prior to treatment with RUCONEST and following each tenth treatment with RUCONEST, will be removed from the label. The requirement for IgE testing was a specific EU request based on a single adverse drug reaction in a study subject, who did not disclose the pre-existing rabbit allergy prior to rhC1Inh treatment. The need for testing was not required in the US as more safety data were available at the time of the Biologics License Application (BLA) and subsequent FDA-approved label in 2014. Prof. Bruno Giannetti, MD, PhD, Pharming’s COO commented: “This EU label change will now also give adolescent in the EU the long awaited access to treat their HAE attacks with a non- blood derived C1- inhibitor and in addition, the positive CHMP opinion confirms the well-established safety profile of RUCONEST® , based on a database of a dozen controlled clinical trials as well as more than 12,000 post-marketing doses of RUCONEST® provided to HAE patients. After adoption of the CHMP opinion by the European Commission, this will remove the burden on patients and doctors to perform testing prior to and after treatments with RUCONEST® and enable emergency treatment with Ruconest® for HAE attacks in previously untreated HAE patients. HAE patients previously dependent on plasma derived C1INH therapies, including adolescents, will be able to receive RUCONEST® with the benefit of eliminating risks of exposure to known blood borne pathogens, such as Hepatitis A, B, C, E, HIV, and CJD, as well as continuously (re)-emerging pathogens, such as the recent ZIKA virus.” Please see Prescribing Information for RUCONEST as applicable for various jurisdictions. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR__Product_Information/human/001223/WC500098542.pdfEMA http://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM405634.pdf

About HAE

Hereditary Angioedema (HAE) is a rare genetic disorder. It is characterised 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 Pharming Group N.V.

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, South Korea, all 28 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 distributed in Argentina, Colombia, Costa Rica, the Dominican Republic, Panama and Venezuela, by Cytobioteck. RUCONEST is distributed in South Korea by HyupJin Corporation and in Israel by Megapharm. 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 and Fabry 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

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