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Pharming announces presentation of new Ruconest(R) clinical data

June 18, 2012 - Leiden, The Netherlands

Findings presented at the annual meeting of the European Academy of Allergyand Clinical Immunology

Biotech company Pharming Group NV("Pharming" or "the Company") (NYSE Euronext: PHARM) today presentedclinicalsafety and efficacy data for Ruconest® (recombinant human C1inhibitor, orrhC1INH) at the annual meeting of the European Academy of Allergy andClinicalImmunology (EAACI), Geneva, Switzerland, 16 to 20 June 2012.

The new data covers a number of aspects that are relevant to theincreasingnumber of physicians that use Ruconest in the day-to-day treatmentof HAEpatients in the European Union: Efficacy and safety data for Ruconestin thetreatment of adolescents suffering from acute attacks of HAE;underpinning apotential extension of the European labeling of Ruconest and"real-life"experience by French physicians; building confidence in Ruconest bysuccessfultreatment with Ruconest of a HAE patient that previously failed variousothertreatments.

In addition an analysis of a previously reported open-label study toexplorepotential benefits of Ruconest in prophylaxis of HAE, potentially anadditionalindication that could be explored.

The headlines and authors of the poster presentations are:

Toubi et al: "Safety and Efficacy evaluation of rhC1INH for the treatmentof HAEattacks in adolescent patients". This analysis reviewed data from 16adolescentHAE patients who were treated for a total of 50 angioedema attackswithRuconest. Patients were treated up to 7 times for HAE attacks at alllocations.Median times to onset of symptom relief for successive attacks ranged from19 to123 minutes. Median times to minimal symptoms ranged from 120 to 650minutes.Ninety percent of the attacks responded within 4 hours after treatment, andnoneof the attacks relapsed. The most frequently reported adverseevent washeadache. No hypersensitivity reactions and nodrug-related serious adverseevents were observed. Notreatment-emergent antibodies developed in thesepatients.

Bouillet et al: "The case of a type III HAE patient who failed severaltherapiesand was successfully treated with Ruconest.

Reshef et al: "Safety and Efficacy of a weekly infusion of RecombinantHuman C1Inhibitor (rhC1INH) for Prophylaxis of Hereditary Angioedema (HAE)attacks".This was an open label study on the prophylactic effect ofonce-weeklyadministration of Ruconest in 25 HAE patients. Patients included in thisstudyhad a history of frequent HAE attacks, with a significant impact ontheirquality of life. The frequency of HAE attacks during the 8 week treatmentperiodwas reduced by approximately 50 percent, from a median of 0.6 attacks perweekto 0.3. The repeated administrations were generally safe andwell-tolerated.

About the EAACI Annual Meeting

The EAACI Congress 2012 will attract around 7000 international clinicians,researchers and allied health professionals. This is the most importantprofessional meeting for advances in research, treatment and prevention ofallergic and immunologic diseases (such as asthma, rhinitis, eczema andoccupational allergy, food and drug allergy, severe anaphylactic reactions,rheumatic and autoimmune diseases, AIDS). More information on the Congresscanbe found on

RUCONEST® Phase III StudyPharming is conducting a Phase III clinical study with RUCONEST® underaSpecial Protocol Assessment (SPA) that is intended to support thesubmission ofa Biologics License Application (BLA) to the U.S. Food and DrugAdministration(FDA). RUCONEST is being evaluated for the treatment of acute attacks ofangioedema in patients with HAE in an international, multicenter,randomized,placebo-controlled Phase III study at a dosage of 50 U/kg with a primaryendpoint of time to beginning of relief of symptoms. Santarus has licensedcertain exclusive rights from Pharming to commercialize RUCONEST in NorthAmerica for the treatment of acute attacks of HAE and other futureindications.Under the terms of the license agreement, a $10 million milestone ispayable toPharming upon successful achievement of the primary endpoint of the PhaseIIIclinical study. The study is expected to be completed in Q3 2012.

About RUCONEST® and Hereditary Angioedema

RUCONEST® (INN conestat alfa) is a recombinant version of the humanprotein C1inhibitor (C1INH). RUCONEST is produced through Pharming's proprietarytechnology in milk of transgenic rabbits and is approved in Europe fortreatmentof acute angioedema attacks in patients with HAE. RUCONEST® is aninvestigational drug in the U.S. and has been granted orphan drugdesignationfor the treatment of acute attacks of HAE, a genetic disorder in which thepatient is deficient in or lacks a functional plasma protein C1 inhibitor,resulting in unpredictable and debilitating episodes of intense swelling oftheextremities, face, trunk, genitals, abdomen and upper airway. The frequencyandseverity of HAE attacks vary and are most serious when they involvelaryngealedema, which can close the upper airway and cause death by asphyxiation.According to the U.S. Hereditary Angioedema Association, epidemiologicalestimates for HAE range from one in 10,000 to one in 50,000 individuals.

About Pharming Group NV

Pharming Group NV is developing innovative products for the treatment ofunmetmedical needs. RUCONEST® is a recombinant human C1 inhibitor approvedfor thetreatment of angioedema attacks in patients with HAE in all 27 EU countriesplusNorway, Iceland and Liechtenstein, and is distributed in the EU by SwedishOrphan Biovitrum (OMX: SOBI). RUCONEST® is partnered with Santarus,Inc(NASDAQ: SNTS) in North America where the drug is undergoing Phase IIIclinicaldevelopment. The product is also being evaluated forfollow-on indications inthe areas of transplantation and reperfusion injury. The advancedtechnologiesof the Company include innovative and validated platforms for theproduction ofprotein therapeutics, technology and processes for the purification andformulation of these products. A feasibility study, using the validatedtransgenic rabbit platform, aimed at the development of recombinant FactorVIIIfor the treatment of Haemophilia A is underway with partner, Renova Life,Inc.Additional information is available on the Pharming website, download the Pharming Group Investor Relations App, click here.

This press release contains forward looking statements that involveknown andunknown risks, uncertainties and other factors, which may cause theactualresults, performance or achievements of the Company to be materiallydifferentfrom the results, performance or achievements expressed or implied bytheseforward looking statements.

Press release (PDF):

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Source: Pharming Group N.V. via Thomson Reuters ONE


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