CHMP Adopts Positive Opinion to Include Self-administration for RUCONEST®

LEIDEN, The Netherlands, November 11, 2016 /PRNewswire/ --




- Home-administration recommended to be included in label for use of RUCONEST(R),
with a new custom-designed administration kit 
- Final decision from European Commission anticipated in January 2017 
- Administration kits expected to be available in EU markets soon thereafter  
- EU approval of self-administration is further to US approval received in 2014 


Pharming Group N.V. (the "Company") today announced that following evaluation of a dossier submitted by the Company earlier this year, the Committee for Medicinal Products for Human Use (CHMP), an advisory committee of the European Medicine Agency (EMA), has adopted a positive opinion recommending an extension to the terms of the marketing authorisation for RUCONEST(R) to the European Commission. This recommendation will allow self-administration of RUCONEST(R) for acute hereditary angioedema (HAE) attacks by adolescents and adults with a new custom-designed administration kit. Following normal timelines after the adoption of the positive opinion by CHMP, the final decision from the European Committee is expected in January 2017. It is expected that the kits will become available for use in the various EU markets soon thereafter.
HAE is a rare disease caused by a deficiency of the C1 esterase inhibitor protein and 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 throat, nose or tongue is particularly dangerous and potentially life-threatening and can lead to obstruction of the airway passages.
If the CHMP opinion is adopted by the European Commission and the approval of the associated educational materials is granted by national authorities, the new RUCONEST(R) self-administration kit will become available for patients, making dealing with an attack simpler. These custom-designed self-administration kits have already been favorably tested by volunteers. These kits are intended to enable patients or their caregivers to treat their HAE attacks in the comfort and privacy of their own homes or at any other place they choose, without the necessity of a healthcare professional (HCP) being present.
Prof. Bruno Giannetti, MD, PhD, Pharming's COO, commented:  
"This EU label change proposal is yet another testament to RUCONEST(R)'s well-established and favorable safety profile.  Over 25,000 post-approval vials of RUCONEST(R) to treat HAE attacks have now been prescribed, making it a convenient, safe and effective way to stop these attacks. Following approval of this administration kit, it will be as convenient to use RUCONEST(R) in the EU as it already is in the US." 
Self-administration (at home or without a HCP present) was granted immediately on approval in the US as a result of more safety data being available at the time of the Biologics License Application (BLA) and subsequent Food and Drug Administration (FDA) approved label in July 2014.  
Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published in the revised European public assessment report (EPAR), and will be available in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.
Please see Prescribing Information for RUCONEST(R) as applicable for various jurisdictions. 
EMA:  http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/001223/WC500098542.pdf
FDA:  http://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM405634.pdf  
About HAE 
Hereditary Angioedema (HAE) is a rare genetic disorder. The condition is caused by a deficiency of the C1 esterase inhibitor protein, which is normally present in blood and helps control inflammation (swelling) and parts of the immune system. Because defective C1-Inhibitor does not adequately perform its regulatory function, a biochemical imbalance can occur and produce unwanted peptides that induce the capillaries to release fluids into surrounding tissue, thereby causing swelling or edema.
HAE 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 angioedema 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(R) 
RUCONEST(R) (recombinant C1 esterase inhibitor) is indicated for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE). Effectiveness in clinical studies was not established in HAE patients with laryngeal attacks.
RUCONEST contains C1 esterase inhibitor at 50 IU/kg. When administered at the onset of HAE attack symptoms at the recommended dose, RUCONEST may help to return a patient's C1 esterase inhibitor levels to normal range and relieve the symptoms of an HAE attack with a low recurrence of symptoms within 24 hours.
RUCONEST is the only recombinant C1 esterase inhibitor approved by the U.S. Food and Drug Administration (FDA) and was approved in July 2014.
Recently RUCONEST(R) demonstrated very positive data for prophylaxis (prevention) in patients with HAE.  If approved for this indication, RUCONEST(R) will have access to this additional market. RUCONEST(R) therefore, has the potential to be the only recombinant C1 esterase inhibitor product approved to target both the acute market (worth approximately $845 million) and the HAE prophylaxis market (separately worth approximately $700 million).
About Pharming Group N.V. 
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(R) (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 and rest of the world. The product is available on a named-patient basis in other territories where it has not yet obtained marketing authorization.
RUCONEST(R) is commercialized by Pharming in Algeria, Andorra, Austria, Bahrain, Belgium, France, Germany, Ireland, Jordan, Kuwait, Lebanon, Luxembourg, Morocco, Netherlands, Oman, Portugal, Qatar, Syria, Spain, Switzerland, Tunisia, United Arab Emirates, United Kingdom and Yemen.
RUCONEST(R) 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(R) is distributed in the United States by a subsidiary of Valeant Pharmaceuticals International, Inc. , following Valeant's acquisition of Salix Pharmaceuticals, Ltd.
RUCONEST(R) is distributed in Argentina, Colombia, Costa Rica, the Dominican Republic, Panama and Venezuela by Cytobioteck, in South Korea by HyupJin Corporation and in Israel by Megapharm.
RUCONEST(R) is also being investigated 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's technology platform includes a unique, GMP-compliant, validated process for the production of pure recombinant human proteins that has proven capable of producing industrial quantities of high quality recombinant human proteins in a more economical and less immunogenetic way compared with current cell-line based methods. Leads for enzyme replacement therapy ("ERT") for Pompé and Fabry's diseases are being optimized at present, with additional programs not involving ERT also being explored at an early stage at present.
Pharming has a long term partnership with the China State Institute of Pharmaceutical Industry ("CSIPI"), a Sinopharm company, for joint global development of new products, starting with recombinant human Factor VIII for the treatment of Haemophilia A. Pre-clinical development and manufacturing will take place to global standards at CSIPI and are funded by CSIPI. Clinical development will be shared between the partners with each partner taking the costs for their territories under the partnership.
Pharming has declared that the Netherlands is its "Home Member State" pursuant to the amended article 5:25a paragraph 2 of the Dutch Financial Supervision Act.
Additional information is available on the Pharming website: http://www.pharming.com
Forward-looking Statements 
This press release of Pharming Group N.V. and its subsidiaries ("Pharming", the "Company" or the "Group") may contain forward-looking statements including without limitation those regarding Pharming's financial projections, market expectations, developments, partnerships, plans, strategies and capital expenditures. 
The Company cautions that such forward-looking statements may involve certain risks and uncertainties, and actual results may differ. Risks and uncertainties include without limitation the effect of competitive, political and economic factors, legal claims, the Company's ability to protect intellectual property, fluctuations in exchange and interest rates, changes in taxation laws or rates, changes in legislation or accountancy practices and the Company's ability to identify, develop and successfully commercialize new products, markets or technologies. 
As a result, the Company's actual performance, position and financial results and statements may differ materially from the plans, goals and expectations set forth in such forward-looking statements. The Company assumes no obligation to update any forward-looking statements or information, which should be taken as of their respective dates of issue, unless required by laws or regulations. 

 
Contacts: 

Pharming Group N.V. 
Sijmen de Vries, CEO, Tel: +31-71-524- 7400 
Bruno Gianetti, COO, Tel: +31-71-524-7400 

FTI Consulting: 
Julia Phillips/ Victoria Foster Mitchell, Tel: +44-203-727-1136 

Lifespring Life Sciences Communication 
Leon Melens, Tel: +31-20-705-95-90 


  PRN NLD



Pharming Group N.V.

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