Ilaris® recommended for European approval as new biologic drug to
treat a rare but serious group of
(Thomson Reuters ONE) - Corporate news announcement processed and transmitted by Hugin AS.The issuer is solely responsible for the content of this announcement. ------------------------------------------------------------------------------------ * Set to become first medicine in EU to treat patients aged four and older suffering from life-long cryopyrin-associated periodic syndrome (CAPS)[1] * Ilaris targets interleukin-1 beta (IL-1ÿ), a key driver of inflammation[1],[2],[3] - studies ongoing in other diseases involving IL-1ÿ such as gout, COPD and type 2 diabetes * EU opinion follows US and Swiss approvals based on data showing Ilaris produced rapid and sustained remission in CAPS patients after one dose[2] * CAPS comprises three disorders of increasing severity with potentially fatal complications[2],[3] - most patients suffer from severe and disabling symptoms[1],[3]Basel, July 24, 2009 - The biotechnology medicine Ilaris®(canakinumab) has passed another major milestone with arecommendation for approval in the European Union to treat patientswith a life-long and potentially fatal auto-inflammatory diseasecalled cryopyrin-associated periodic syndrome (CAPS). When approved,Ilaris will be the only treatment in the EU indicated for CAPSpatients aged four years and older[1].Ilaris represents an important advance in the development ofpersonalized medicines because it targets a condition that istriggered by a specific genetic mutation. In CAPS patients, thismutation drives the overproduction of interleukin 1-beta (IL-1ÿ)which causes the widespread sustained inflammation and tissue damageassociated with the disease[3],[4],[5].Because Ilaris normalizes the production of IL-1ÿ[1],[2],[3], it isalso being studied in other diseases in which IL-1ÿ plays a pivotalrole such as systemic juvenile idiopathic arthritis (SJIA), gout,chronic obstructive pulmonary disorder (COPD), and type 2 diabetes."By concentrating initially on a rare syndrome with a well-defineddisease process such as CAPS, we have been able to demonstrate aclear therapeutic advantage with Ilaris," said Trevor Mundel, MD,Head of Global Development at Novartis Pharma AG. "Our focus now isto establish whether this could also provide a new approach to thetreatment of other diseases involving a similar underlying process."A positive opinion recommending the approval of Ilaris for CAPS wasissued by the Committee for Medicinal Products for Human Use (CHMP),which reviews medicines for the European Commission. Therecommendation comes shortly after approvals in the US andSwitzerland where Ilaris was granted priority review based on itspotential to fulfil an important unmet need for CAPS patients.The EU submission was supported by data showing that Ilaris, amonoclonal antibody formerly known as ACZ885, produced rapid andsustained remission of symptoms in up to 97% of CAPS patients, withmost responding from the first injection[2].Ilaris is given by subcutaneous injection only once every two monthsmaking it a convenient treatment, especially for younger patients[2].More than 90% of patients studied were free from painfulinjection-site reactions[2].CAPS includes three distinct auto-inflammatory disorders. These arefamilial cold auto-inflammatory syndrome (FCAS) which is the mildestform of CAPS, Muckle-Wells syndrome (MWS), and neonatal-onsetmultisystem inflammatory disease (NOMID, also known as chronicinfantile neurological cutaneous articular syndrome or CINCA) - themost severe form of the disease[2],[3]."CAPS is a life-long and potentially fatal condition for which thereare currently no approved medications in the European Union," saidHelen J. Lachmann, MD of the UK National Amyloidosis Centre at UCLMedical School in London, UK. "In clinical trials, canakinumab hasbeen shown to switch off disease activity in as little as 24 hoursfollowing a single dose. It has the potential to transform patients'lives, not only providing relief from their debilitating dailysymptoms but also offering the possibility of long-term control ofthe disease."The symptoms of CAPS, such as debilitating fatigue, rash, fever,headaches, joint pain and conjunctivitis, can be present from birthor infancy, and can occur daily throughout patients' lives[2],[3].Serious long-term consequences may include deafness, bonedeformities, erosive joint destruction, and central nervous systemdamage leading to loss of vision[1],[2],[3]. Around 25% of CAPSpatients develop amyloidosis, a condition in which the build-up ofproteins can cause vital organs to fail, resulting in renal failureand death within five to 10 years[1].CAPS is believed to occur in around 6,500 patients worldwide and2,500 in the EU[3],[6]. However due to lack of diagnosis ormisdiagnosis, fewer than 1,000 cases have been officially reportedworldwide[1],[3].The Ilaris filing was based on a clinical trial program involvingmore than 100 CAPS patients. The pivotal study is a three-part,one-year Phase III study involving 35 patients aged nine to 74 yearsold with varying degrees of disease severity[2]. Data published inThe New England Journal of Medicine in June 2009 show that Ilarisproduced a rapid, complete and sustained response in the majority ofpatients[2].Results for the primary endpoint showed that none of the patientstreated with Ilaris (0 out of 15) experienced a disease outbreak or'flare' compared to 13 of the 16 patients who received placebo (0%vs. 81% respectively, p<0.001)[2].In general, Ilaris was well tolerated with no consistent pattern ofadverse events apart from a slight increase in infections[2]. Twopatients experienced serious adverse events, namely a lower urinarytract infection and vertigo[2]. The most common adverse eventsreported in Ilaris-treated patients were nasopharyngitis, diarrhea,influenza, headache and nausea[2].No impact on the type or frequency of adverse events was seen withlonger-term treatment[2]. Ilaris was not associated with any severereactions at the injection site, and those that did occur weremild-to-moderate in nature[2].The CHMP recommended approval under exceptional circumstances,granted when comprehensive data are not yet available due to therarity of the disease or limited scientific knowledge. The approvalis subject to certain obligations for the company and is re-assessedeach year until normal approval can be given.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. Astudy in NOMID patients is under way in the US and priority reviewsare being conducted in other countries, including Australia andCanada.In addition to orphan drug status for CAPS, Ilaris has also beendesignated as an orphan drug for treating systemic juvenileidiopathic arthritis (SJIA) 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)[7] or fewer than five out of 10,000 people (in the EU)[8].DisclaimerThe foregoing release contains forward-looking statements that can beidentified by terminology such as "potentially," "will," "could,""potential," "can," "may," or similar expressions, or by express orimplied discussions regarding potential future regulatory filings ormarketing approvals for Ilaris, or the timing of any such potentialfilings or approvals, or regarding potential future revenues fromIlaris. You should not place undue reliance on these statements. Suchforward-looking statements reflect the current views of the Companyregarding future events, and involve known and unknown risks,uncertainties and other factors that may cause actual results withIlaris to be materially different from any future results,performance or achievements expressed or implied by such statements.There can be no guarantee that Ilaris will be approved for sale inany additional market, or for any additional indication, or that anysuch approvals will occur at any particular time. Nor can there beany guarantee that Ilaris will achieve any levels of revenue in thefuture. In particular, management's expectations regarding Ilariscould be affected by, among other things, unexpected clinical trialresults, including unexpected new clinical data and unexpectedadditional analysis of existing clinical data; unexpected regulatoryactions or delays or government regulation generally; the company'sability to obtain or maintain patent or other proprietaryintellectual property protection; competition in general; government,industry and general public pricing pressures; the impact that theforegoing factors could have on the values attributed to the Group'sassets and liabilities as recorded in the Group's consolidatedbalance sheet, and other risks and factors referred to in NovartisAG's current Form 20-F on file with the US Securities and ExchangeCommission. Should one or more of these risks or uncertaintiesmaterialize, or should underlying assumptions prove incorrect, actualresults may vary materially from those anticipated, believed,estimated or expected. Novartis is providing the information in thispress release as of this date and does not undertake any obligationto update any forward-looking statements contained in this pressrelease as a result 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 these areas. In 2008, theGroup's continuing operations achieved net sales of USD 41.5 billionand net income of USD 8.2 billion. Approximately USD 7.2 billion wasinvested in R&D activities throughout the Group. Headquartered inBasel, Switzerland, Novartis Group companies employ approximately99,000 full-time-equivalent associates and operate in more than 140countries around the world. For more information, please visithttp://www.novartis.com.References1. 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 April 21, 2009.2. Lachmann HJ, Kone-Paut I, Kuemmerle-Deschner JB, et al. Use ofCanakinumab in the Cryopyrin-Associated Periodic Syndrome. N Engl JMed, 360;23. June 4, 2009.3. 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 April 19,2009.4. Joost PH, Drenth MD, Jos W.M. van der Meer. The Inflammasome -A Linebacker of Innate Defense. N Engl J Med 2006. Vol 355:730-732.Number 7.5. 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.20082481.6. 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 14 July 2009.7. Orphan Drug Act. US Food and Drug Administration. Section 526(2).8. The orphan drug strategy. Europa: Gateway to the EuropeanUnion. Paragraph 1, Line 1. # # #Novartis Media RelationsEric Althoff Irina FerlugaNovartis Global Media Novartis Pharma CommunicationsRelations +41 61 324 2422 (direct)+41 61 324 7999 (direct) +41 79 824 1121 (mobile)+41 79 593 4202 (mobile) irina.ferluga(at)novartis.comeric.althoff(at)novartis.come-mail: media.relations(at)novartis.comNovartis Investor RelationsCentral phone: +41 61 324 7944Ruth +41 61 324 North America:Metzler-Arnold 9980Pierre-Michel +41 61 324 1065 Richard Jarvis +1 212 830Bringer 2433John Gilardi +41 61 324 3018 Jill Pozarek +1 212 830 2445Thomas +41 61 324 8425 Edwin Valeriano +1 212 830Hungerbuehler 2456Isabella Zinck +41 61 324 7188e-mail: e-mail:investor.relations(at)novartis.com investor.relations(at)novartis.comhttp://hugin.info/134323/R/1330801/314694.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: 24.07.2009 - 12:45 Uhr
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