Novartis biological drug Ilaris® approved in EU to treat children and
adults with CAPS, a rare debil
(Thomson Reuters ONE) - Corporate news announcement processed and transmitted by Hugin AS.The issuer is solely responsible for the content of this announcement. ------------------------------------------------------------------------------------ * First drug approved in EU for patients as young as four years old with cryopyrin-associated periodic syndrome (CAPS)[1] * Approval based on positive data showing Ilaris produced rapid and sustained remission of CAPS symptoms in up to 97% of patients[2] * Ilaris is a monoclonal antibody that selectively targets and blocks interleukin-1 beta (IL-1ÿ), the trigger for inflammation and tissue damage in CAPS patients[1],[2],[3] * Studies ongoing in groups of patients with other diseases involving IL-1ÿ such as COPD, type 2 diabetes and gout - one of the most painful forms of arthritisClick here for multimedia content:http://www.novartis.com/newsroom/news/spotlight-ilaris-caps.shtmlBasel, October 28, 2009 - The new biological medicine Ilaris®(canakinumab) has been approved in the European Union (EU) to treatadults and children as young as four years old withcryopyrin-associated periodic syndrome (CAPS), a rare life-longauto-inflammatory disease with debilitating symptoms and fewtreatment options[1],[2],[3].The accelerated EU decision follows approvals in the US andSwitzerland, where Ilaris was granted priority review in view of thesignificant unmet medical need. Ilaris is the only medicine approvedin the EU for CAPS patients as young as four years old, and forpatients with the most debilitating form of CAPS, neonatal-onsetmultisystem inflammatory disease (NOMID)[4]. It is a fully humanmonoclonal antibody given by injection under the skin once every twomonths - the longest dosing interval of any availabletreatment[2],[5],[6]."We are excited by the latest approval because Ilaris represents asignificant therapeutic advance for patients with this debilitatingand sometimes fatal disease," said Joe Jimenez, CEO of the NovartisPharmaceuticals Division. "Ilaris is the outcome of ourpathways-driven search for innovative medicines that are tailored tothe needs of patients. Initially we studied Ilaris in a very raredisease with a well-understood genetic profile, and now that itsefficacy has been proven, we are able to move ahead rapidly withdevelopment in other diseases characterized by the same inflammatoryprocess."The regulatory submission was supported by data showing that Ilarisproduced rapid and sustained remission of symptoms in up to 97% ofCAPS patients, with most of them responding within hours of the firstinjection[2].Ilaris, previously known as ACZ885, targets and normalizes theproduction of a protein within the immune system called interleukin-1beta (IL-1ÿ)[1],[3],[7]. In CAPS patients, IL-1ÿ is overproducedcausing widespread inflammation and tissue damage[3],[8],[9].Symptoms, such as debilitating fatigue, fever, joint pain andconjunctivitis, can be present from infancy and continue throughoutthe patient's life[2],[3].If left untreated, CAPS may have serious consequences such asdeafness, bone deformities, erosive joint destruction, and centralnervous system damage leading to loss of vision[1],[2],[3]. Around25% of patients develop amyloidosis, a condition in which thebuild-up of proteins can cause vital organs to fail, resulting inrenal failure and requiring kidney transplantation. Approximately 20%of patients with NOMID, the most severe form of CAPS, die beforereaching adulthood[2],[3],[10].CAPS is believed to occur in around one in 2,500 people in theEU[3],[11], but fewer than 1,000 cases have been reported worldwidedue to poor diagnosis[1],[3]. CAPS includes three distinctautoinflammatory disorders of increasing severity: familial coldauto-inflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS), andNOMID[2],[3]. Ilaris is the only treatment indicated in the EU andSwitzerland to treat all three disorders[1],[4].Studies with ACZ885 are ongoing in other diseases in which IL-1ÿplays an important role, such as chronic obstructive pulmonarydisease (COPD), type 2 diabetes, systemic juvenile idiopathicarthritis (SJIA), and gout - one of the most painful forms ofarthritis. Not all potential patients with these diseases would beeligible for treatment with ACZ885, if approved.The CAPS filing was based on a clinical trial program involving morethan 100 patients. Data from a pivotal study published in The NewEngland Journal of Medicine show that Ilaris produced a rapid,complete and sustained response in most patients[2]. None of thepatients treated with Ilaris (0 out of 15) experienced a diseaseoutbreak or 'flare' compared to 13 of the 16 patients who receivedplacebo (0% vs. 81% respectively, p<0.001)[2]."In CAPS studies, symptoms improved within 24 hours after patientsreceived a single dose of Ilaris. The disease was barely detectablein the blood after two weeks and the remission of symptoms wassustained for six months," said Helen J. Lachmann, MD of the UKNational Amyloidosis Centre at the Royal Free and University CollegeMedical School in London, UK. "By effectively turning off the diseaseactivity, Ilaris has the potential to transform patients' lives byoffering long-term control of their disease."Ilaris was generally well tolerated and there was no consistentpattern of adverse events apart from a slight increase ininfections[2]. Two patients experienced serious adverse events,namely a lower urinary tract infection and vertigo[2]. The mostcommon adverse events were nasopharyngitis, diarrhea, influenza,headache and nausea[2]. Ilaris was not associated with any severeinjection-site reactions and those that did occur were classified asmild-to-moderate[2].The EU approval was granted under exceptional circumstances, a commonpractice with so-called orphan drugs. This reflects a need foradditional data due to factors such as the rarity of the disease orlack of scientific knowledge. The situation is reviewed every yearuntil the European Medicines Agency (EMEA) is able to grant a normalapproval.In addition to its orphan drug status for CAPS, Ilaris has beendesignated as an orphan drug for treating SJIA, the most severe formof arthritis in children, in the US, EU and Switzerland, and hasfast-track status for SJIA in the US. Orphan drugs are thosedeveloped to treat diseases affecting fewer than 200,000 people (inthe US)[12] or fewer than five out of 10,000 people (in the EU)[13].Ilaris was approved in Switzerland in July 2009 to treat all threeforms of CAPS in adults and children over four years old, and in theUS in June 2009 to treat two forms of CAPS, namely FCAS and MWS,while a study in NOMID patients is under way. Priority reviews areongoing in other countries including Australia, Brazil and Canada.DisclaimerThe foregoing release contains forward-looking statements that can beidentified by terminology such as "potential," "long-term," orsimilar expressions, or by express or implied discussions regardingpotential additional indications for Ilaris, or regarding potentialfuture revenues from Ilaris. You should not place undue reliance onthese statements. Such forward-looking statements reflect the currentviews of the Company regarding future events, and involve known andunknown risks, uncertainties and other factors that may cause actualresults with Ilaris to be materially different from any futureresults, performance or achievements expressed or implied by suchstatements. There can be no guarantee that Ilaris will be approvedfor any additional indication. Nor can there be any guarantee thatIlaris will achieve any levels of revenue in the future. Inparticular, management's expectations regarding Ilaris could beaffected by, among other things, unexpected clinical trial results,including unexpected new clinical data and unexpected additionalanalysis of existing clinical data; unexpected regulatory actions ordelays or government regulation generally; the company's ability toobtain or maintain patent or other proprietary intellectual propertyprotection; competition in general; government, industry and generalpublic pricing pressures; the impact that the foregoing factors couldhave on the values attributed to the Group's assets and liabilitiesas recorded in the Group's consolidated balance sheet, and otherrisks and factors referred to in Novartis AG's current Form 20-F onfile with the US Securities and Exchange Commission. Should one ormore of these risks or uncertainties materialize, or shouldunderlying assumptions prove incorrect, actual results may varymaterially from those anticipated, believed, estimated or expected.Novartis is providing the information in this press release as ofthis date and does not undertake any obligation to update anyforward-looking statements contained in this press release as aresult of new information, future events or otherwise.About NovartisNovartis provides healthcare solutions that address the evolvingneeds of patients and societies. Focused solely on healthcare,Novartis offers a diversified portfolio to best meet these needs:innovative medicines, cost-saving generic pharmaceuticals, preventivevaccines, diagnostic tools and consumer health products. Novartis isthe only company with leading positions in each of these areas. In2008, the Group's continuing operations achieved net sales of USD41.5 billion and net income of USD 8.2 billion. Approximately USD 7.2billion was invested in R&D activities throughout the Group.Headquartered in Basel, Switzerland, Novartis Group companies employapproximately 99,000 full-time-equivalent associates and operate inmore than 140 countries around the world. For more information,please visit http://www.novartis.com.References[1.] National Horizon Scanning Centre. Canakinumab for cryopyrinassociated periodic syndrome. November 2008. Available at:http://www.pcpoh.bham.ac.uk/publichealth/horizon/outputs/documents/2008/sept-dec/Canakinumab.pdfLast accessed September 23, 2009.[2.] Lachmann HJ, Kone-Paut I, Kuemmerle-Deschner JB, et al. Use ofCanakinumab in the Cryopyrin-Associated Periodic Syndrome. N Engl JMed 2009; 360: 2416 - 2425[3.] Durrant KLW, Goldbach-Mansky R, Hoffman H, Leslie K, Rubin B.CAPS Cryopyrin-Associated Periodic Syndromes 2008. Available at:http://www.nomidalliance.net/Download1.html. Last accessed September23, 2009.[4.] Ilaris (canakinumab) prescribing information.[5.] Arcalyst (rilonacept) prescribing information.[6.] Kineret (anakinra) prescribing information.[7.] Kastner DL. Hereditary Periodic Fever Syndromes. Hematology 2005- American Society of Hematology Education Program. 2005: 74-81.Available at:http://asheducationbook.hematologylibrary.org/cgi/reprint/2005/1/74[8.] Joost PH, Drenth MD, Jos W.M. van der Meer. The Inflammasome - ALinebacker of Innate Defense. N Engl J Med 2006 355(7): 730-732.[9.] Lachmann HJ, Lowe P, Felix SD, et al. In vivo regulation ofinterleukin 1 in patients with cryopyrin-associated periodicsyndromes. J Exp Med 2009. Published online April 13, 2009. Availableat: www.jem.org/cgi/doi/10.1084/jem.20082481.[10.] Prieur A-M. CINCA syndrome. October 2003. Orphanet. Availableat: http://www.orpha.net/data/patho/Pro/en/CINCA-FRenPro3395.pdfLast accessed September 23, 2009.[11.] European Medicines Agency (EMEA). Pre-authorisation evaluationof medicines for human use. Committee for orphan medicinal products.Available at:http://www.emea.europa.eu/pdfs/human/comp/opnion/17086808en.pdf. Lastaccessed September 23, 2009.[12.] Orphan Drug Act. US Food and Drug Administration. Section 526(2), Line 2.[13.] The orphan drug strategy. Europa: Gateway to the EuropeanUnion. Paragraph 1, Line 1. # # #Novartis Media RelationsEric Althoff Irina FerlugaNovartis Global Media Relations Novartis Pharma Communications+41 61 324 7999 (direct) +41 61 324 2422 (direct)+41 79 593 4202 (mobile) +41 79 824 1121 (mobile)eric.althoff(at)novartis.com irina.ferluga(at)novartis.come-mail: media.relations(at)novartis.comNovartis Investor RelationsCentral phone: +41 61 324 7944Ruth Metzler-Arnold +41 61 324 North America: 9980Pierre-Michel +41 61 324 Richard Jarvis +1 212 830Bringer 1065 2433John Gilardi +41 61 324 Jill Pozarek +1 212 830 3018 2445Thomas Hungerbuehler +41 61 324 Edwin Valeriano +1 212 830 8425 2456Isabella Zinck +41 61 324 7188e-mail: e-mail:investor.relations(at)novartis.com investor.relations(at)novartis.comhttp://hugin.info/134323/R/1350596/325952.pdf --- End of Message ---Novartis International AGPosfach Basel WKN: 904278; ISIN: CH0012005267; Index: SLCI, SMI, SPI, SLIFE;Listed: Main Market in SIX Swiss Exchange, ZLS in BX Berne eXchange;
Datum: 28.10.2009 - 07:15 Uhr
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