Pharming gives update on Rhucin EU marketing authorization application
(Thomson Reuters ONE) - CHMP raises no 'major concerns' on D120 List of QuestionsLeiden, The Netherlands,January 22, 2010. Biotech company Pharming Group NV("Pharming" or "the Company") (NYSE Euronext: PHARM) today provided an update onthe regulatory review by the European Medicines Agency's Committee for MedicinalProducts for Human Use (CHMP) of its Marketing Authorization Application (MAA)for Rhucin for the treatment of acute attacks of Hereditary Angioedema (HAE).The Company reports that it has received the Day 120 List of Questions (LoQ)from the CHMP. At this stage of the procedure, no 'major concerns' have beenraised by the CHMP on the application. Pharming is confident on a positiveoutcome of the European MAA filing.Pharming submitted the MAA for Rhucin to the European Medicines Agency inSeptember 2009 and the CHMP has issued the Day 120 List of Questions on 21January 2010. The LoQ did not contain any 'major concerns'. The CHMP review ofthe MAA will continue as soon as the clock starts after the response to theCHMP's questions is submitted (Day 121). Pharming plans to submit its responsewithin the regular three month clock stop period. Another one month clock stopmay occur at Day 180 of the procedure to permit Pharming to respond to furtherquestions from the CHMP and, in line with the regulatory timetable, the CHMPwill reach its final opinion no later than Day 210. More information on thisprocedure can be found on www.ema.europa.eu .Dr. Bruno Giannetti, Chief Operations Officer of Pharming, said: "We are verypleased with the EMA feed back on our dossier and therefore we continue toremain confident that our new MAA dossier has addressed all issues from theprevious CHMP opinion in 2008. We believe that our current clinical databasestrongly supports the efficacy and safety of Rhucin for single and repeated usein the treatment of all types of acute angioedema attacks. We are confident thatwe will be able to satisfactorily address all outstanding CHMP questions in thecoming months."Efficacy of Rhucin has been demonstrated in two randomized controlled studiesand is supported by four open label studies, including the successful treatmentof potentially life-threatening laryngeal attacks.In the RCT studies patientsreceived Rhucin (100 U/kg or 50 U/kg body weight) or saline (placebo) controltreatment. Patients receiving Rhucin responded rapidly to treatment, withstatistically significant and clinically relevant shorter time to onset ofrelief and time to minimal symptoms compared to placebo. Following treatmentwith Rhucin, no relapse of attacks was recorded. Superiority over placebo wasalso consistently supported by exploratory endpoints, sensitivity analyses andsubgroup statistical analyses.The updated safety dataset, submitted to the European Medicines Agency inSeptember, comprises a total of 405 administrations in 139 subjects. Fourteenpatients received at least five repeat administrations of Rhucin and one patientreceived 20 administrations. Rhucin was well tolerated with the adverse eventprofile observed in the controlled studies was similar to that of placebo. Therewere no significant infusion site reactions such as pain. Immunogenicity testingrevealed a reassuring immuno-safety profile, without evidence for induction ofneutralizing antibodies against Rhucin or for the induction of allergicresponses.About Rhucin® and HAERhucin® (recombinant human C1 esterase inhibitor) is a human protein developedthrough Pharming's proprietary technology where the human protein is expressedin milk of transgenic rabbits. Pharming is developingRhucin® for treatment ofpatients with acute attacks of Hereditary Angioedema (HAE). HAE is a humangenetic disorder caused by a shortage of C1 inhibitor activity and results in anoverreaction of the immune system. The disease is characterized by acute attacksof painful and in some cases fatal swelling of several soft tissues (edema),which may last up to five days when untreated. In addition to thelife-threatening nature of the disease, quality of life for individuals with thedisease may be seriously impaired. Approximately one in 30,000 individualssuffers from HAE and has an average of seven acute attacks per year.About Pharming Group NVPharming Group NV is developing innovative products for the treatment of geneticdisorders, ageing diseases, specialty products for surgical indications, andnutritional products. Pharming's lead product Rhucin® for acute attacks ofHereditary Angioedema has passed clinical development stage and the MarketAuthorization Application is under review withthe European Medicines Agency.Prodarsan® is in early stage clinical development for Cockayne Syndrome andlactoferrin for use in food products. The advanced technologies of the Companyinclude innovative platforms for the production of protein therapeutics,technology and processes for the purification and formulation of these products,as well as technology in the field of DNA repair (via DNage). Additionalinformation is available on the Pharming website, http://www.pharming.com.This press release contains forward looking statements that involve known andunknown risks, uncertainties and other factors, which may cause the actualresults, performance or achievements of the Company to be materially differentfrom the results, performance or achievements expressed or implied by theseforward looking statements.Contact:Marjolein van Helmond, T: +31 (0)71 52 47 431 or +31 (0)6 109 299 54Samir Singh, T: + 1 908 720 6224[HUG#1375833] Press release (PDF): http://hugin.info/132866/R/1375833/337667.pdf
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Datum: 22.01.2010 - 02:01 Uhr
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