Pharming submits marketing authorisation application for Rhucin® to
the EMEA
(Thomson Reuters ONE) - Leiden, The Netherlands, September 3, 2009. Biotech company PharmingGroup NV ("Pharming" or "the Company") (NYSE Euronext: PHARM) todayannounced that it has submitted the Marketing AuthorizationApplication (MAA) for Rhucin for the treatment of acute attacks ofHereditary Angioedema (HAE) to the European Medicines Agency (EMEA).In this new MAA, Pharming has followed up on and addressed concernsraised by the CHMP during the former evaluation procedure in 2007. Inparticular, the size of the clinical database has been significantlyexpanded, additional clinical efficacy and safety data have beencollected and analyzed, and an analysis of immunogenicity data after(repeat) treatment with Rhucin has been completed.Efficacy of Rhucin was demonstrated in two randomized controlledstudies and is supported by four open label studies, including thesuccessful treatment of potentially life-threatening laryngealattacks. In the placebo controlled clinical studies a total of 70patients were randomized to Rhucin (100 U/kg or 50 U/kg body weight)or placebo control treatment. Patients with active Rhucin treatmentresponded rapidly to treatment, with statistically significant andclinically relevant shorter time to onset of relief and time tominimal symptoms compared to placebo. No relapses of attacks wererecorded. Superiority over placebo was also consistently supported byexploratory endpoints, sensitivity analyses and subgroup analyses.Additional data on 155 attacks in 79 patients collected in theongoing open-label studies furthermore confirmed Rhucin's efficacy.The updated safety dataset comprising 405 administrations in 164subjects, includes 14 patients with at least five repeatadministrations, Rhucin continued to be well tolerated, the adverseevent profile observed in the controlled studies, was similar to thatof placebo, and there were no significant infusion site reactionssuch as pain. The immunogenicity analysis of 621 plasma samplescovering 217 treatments in 119 patients showed a reassuringimmuno-safety profile, without evidence for induction of neutralizingantibodies against Rhucin, nor for induction of allergies."The MAA re-submission is a significant milestone in the developmentof Rhucin and demonstrates our commitment to provide a highlyeffective therapy for patients living with the burden of HereditaryAngioedema", said Dr. Bruno Giannetti, Chief Operations Officer. "Webelieve that our new MAA submission addresses all issues thatremained after the previous CHMP opinion in 2008. We are convincedthat our current clinical database adequately supports the efficacyand safety of Rhucin, in single and repeated use and in all types ofacute attacks including laryngeal attacks. Our next focus will be theadmission of a Biologic License Application (BLA) for Rhucin in theUSA and we will request a pre-BLA meeting by the end of 2009."Upon receipt of the MAA, the EMEA will validate the dossier and mayrequire additional data, information or clarification before thestart of evaluation of the scientific content of the dossier. Oncethe application is validated, the EMEA starts the CentralisedProcedure for scientific evaluation of the MAA by the Committee forMedicinal Products for Human Use (CHMP) according to a standardtimetable. At day 120, the CHMP produces a List of Questions to beanswered within 3 months by the Company. More information on thisprocedure can be found on www.emea.europa. The Company will providefurther information on progress of the review of the Rhucin MAAdossier at appropriate stages during the process.Background on Hereditary AngioedemaHAE is a genetic disorder caused by a shortage of C1 inhibitoractivity. Approximately one in 30,000 individuals suffers from HAEand has an average of seven acute attacks per year. HAE attacks thatare untreated can last up to five days. The disease is characterizedby acute attacks of painful swelling of soft tissues (edema),including regions of the skin, the intestine, and the mouth andthroat. If the soft tissue of the throat is involved, an attack ofangioedema can be fatal. In addition to the life-threatening natureof the disease, quality of life for individuals with the disease maybe seriously impaired.About Pharming Group NVPharming Group NV is developing innovative products for the treatmentof genetic disorders, ageing diseases, specialty products forsurgical indications, intermediates for various applications andnutritional products. Pharming has two products in late stagedevelopment - Rhucin® for Hereditary Angioedema and human Lactoferrinfor use in food products and one product in early stage clinicaldevelopment - Prodarsan® for Cockayne Syndrome. The advancedtechnologies of the Company include innovative platforms for theproduction of protein therapeutics, technology and processes for thepurification and formulation of these products, as well as technologyin the field of DNA repair (via DNage). Additional information isavailable on the Pharming website, http://www.pharming.com.This press release contains forward looking statements that involveknown and unknown risks, uncertainties and other factors, which maycause the actual results, performance or achievements of the Companyto be materially different from the results, performance orachievements expressed or implied by these forward lookingstatements.Contact:Sijmen de Vries, Pharming Group NV, T: +31 (0)71 52 47 400http://hugin.info/132866/R/1339193/319736.pdfThis announcement was originally distributed by Hugin. The issuer is solely responsible for the content of this announcement.
Datum: 03.09.2009 - 08:00 Uhr
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