Tracleer (bosentan) receives EU approval for pediatric formulation -
the first and only licensed pul
Tracleer (bosentan) receives EU approval for pediatric formulation - the first and only licensed pulmonary arterial hypertension therapy for children
(Thomson Reuters ONE) - Corporate news announcement processed and transmitted by Hugin AS.The issuer is solely responsible for the content of this announcement. ------------------------------------------------------------------------------------ ALLSCHWIL, SWITZERLAND - 06 July 2009 - Actelion Ltd (SIX: ATLN)announced today that the pediatric dispersible formulation ofTracleer® (bosentan) for the treatment of pulmonary arterialhypertension (PAH) in children has been approved in the EuropeanUnion.This approval makes Tracleer® the only PAH therapy with an approvedpediatric formulation for treating children from two years of age.PAH is a severe condition in children with an estimated mediansurvival of 10 months after diagnosis if left untreated [1].Martine Clozel, M.D., Pediatrician and Chief Scientific Officer atActelion, commented: "Our team is very proud of this achievement. Wedecided very early in the development of Tracleer® to devote time andeffort to the development of a formulation adapted to the dailytreatment of children suffering from PAH, even if it was representinga small population. It has been a long, but ultimately extremelyrewarding journey, which had begun even before the first marketingauthorization of Tracleer®."Isaac Kobrin, M.D. and Head of Clinical Development at Actelion,added: "Having the approval of Tracleer®, with an accurate dosing ina child-friendly form, is an important advancement in the treatmentof PAH in children. After receiving approval in the EU, we willcontinue our regulatory filings in other countries to ensure thatchildren can benefit from this pediatric formulation of Tracleer® ona worldwide basis."Professor Maurice Beghetti, Head of the Pediatric Cardiology Unit atHôpital des Enfants, Geneva, commented: "It is great to see Actelionleading the way in providing a treatment fully tailored for children,from the precise dosing to the flavor. Ensuring correct dosing forchildren is a challenge we face across all diseases but particularlyin orphan diseases that affect children. This pediatric formulationfor Tracleer® is a large step in the right direction towardsdeveloping treatment with the needs of children specifically inmind."Tracleer® is an oral, dual endothelin receptor antagonist, which iscurrently approved in Europe for the treatment of PAH; in WHOFunctional Class III to improve exercise capacity and symptoms and inWHO Functional Class II where some improvements have also been shown[2]. In the EU, Tracleer® is also indicated to reduce the number ofnew digital ulcers in patients with systemic sclerosis and ongoingdigital ulcer disease.The new quadrisect, dispersible 32mg tablet formulation of bosentan,which was specifically developed for children, allows a convenient,accurate and more flexible dosing regimen according to low bodyweight.The safety and tolerability profile of Tracleer® in children wasconsistent with that observed in previous placebo-controlled clinicaltrials in the adult population.The key trials in the pediatric research program include:* BREATHE-3 (Bosentan Randomized trial of Endothelin Antagonist THErapy for pulmonary hypertension): an open-label study that provided safety and efficacy data in children with PAH treated with Tracleer® with or without concomitant prostanoid therapy. It also provided important information on the dose required in the pediatric formulation.* FUTURE-1 (Pediatric FormUlation of bosenTan in pUlmonary arterial hypeRtEnsion): an open-label study that evaluated the safety and pharmacokinetics of a new dispersible tablet formulation of Tracleer®. This study provided important pharmacokinetic and dosing information using the new pediatric formulation of bosentan. In FUTURE-1, the observed exposure to Tracleer® was similar to that in children who participated in BREATHE-3.* FUTURE-2: an open-label safety extension study is ongoing to assess long-term safety and outcome data. ###Notes to Editor:About Pulmonary Arterial Hypertension (PAH)Pulmonary arterial hypertension (PAH) is a chronic, life-threateningdisorder characterized by abnormally high blood pressure in thearteries between the heart and lungs of an affected individual. Thefunction of the heart and lungs is severely compromised, manifestedby a limited exercise capacity, and, ultimately, a reduced lifeexpectancy. Approximately 100,000 people in Europe and the UnitedStates are afflicted with either primary or secondary forms of thedisease related to conditions or tissue disorders that affect thelungs, such as scleroderma, lupus, HIV/AIDS or congenital heartdisease.PAH is associated with structural changes in both the pulmonaryvasculature and the right ventricle. Recent advances [4] in theunderstanding of the pathogenic factors leading to the pulmonaryvascular disease have led to the development of new therapiestargeting specific pathways (the prostacyclin pathway; the endothelinpathway; and the nitric oxide pathway) [5]. The available therapieshave shown positive treatment effects in patients with PAH, but theydo not provide a cure, and in many patients the disease willprogress. PAH remains a serious life-threatening condition [5,6].Early recognition and an understanding of the selection and timing oftherapeutic options remain critical elements in the optimalmanagement of patients with this disorder.About PAH in ChildrenPAH is a severe condition in children with an estimated mediansurvival of 10 months after diagnosis, if left untreated [1].Online information on PAH is available at www.pah-info.com.PAH-info.com is part of an international PAH awareness campaignsupported by Actelion Pharmaceuticals and has been created to provideinformation to healthcare professionals and patients.About Tracleer® in Pulmonary Arterial Hypertension (PAH)Tracleer® (bosentan), the first oral dual endothelin receptorantagonist, is approved for the treatment of pulmonary arterialhypertension (PAH) and made available by Actelion subsidiaries in theUnited States, the European Union, Japan, Australia, Canada,Switzerland and other markets worldwide.About Actelion's pediatric programBREATHE-3 (Bosentan Randomized trial of Endothelin Antagonist THErapyfor pulmonary hypertension) an open-label study, provided safety andefficacy data in children with PAH, treated with the adultfilm-coated tablet formulation of Tracleer® with or withoutconcomitant prostanoid therapy. It also provided importantinformation on the dose required in the pediatric formulation.In the BREATHE-3 study, WHO functional class improved in five of the19 patients over the 12-week treatment period. Improvements wereobserved in each weight group, and only one patient deteriorated;13/19 remained stable. These clinical results were consistent withthose obtained in earlier studies conducted in adult patients andsupport that pediatric patients with PAH may derive significantclinical benefit from therapy with bosentan.FUTURE-1 (Pediatric FormUlation of bosenTan in pUlmonary arterialhypertension) an open-label study, evaluated the safety andpharmacokinetics of a new dispersible tablet formulation of bosentan.A new oral, dispersible, quadrisect tablet formulation of bosentandedicated to pediatric patients was investigated; patients initiallyreceived 2 mg/kg bid for 4 weeks followed by 4 mg/kg bid until Week12. The trial enrolled 36 patients aged from 2 years up to 12 yearswith idiopathic PAH or familial PAH.The main objective was to demonstrate that exposure to the pediatricformulation of bosentan in children with PAH is similar to the knownexposure of the adult formulation. The primary study endpoint wasAUC(tau) of bosentan, determined at Week 12. Secondary objectivesincluded evaluation of tolerability and safety of bosentan inpediatric patients with PAH. Secondary endpoints included additionalpharmacokinetic parameters of Cmax and Tmax. Exploratory endpointsincluded: changes from baseline to Week 12 in health-related qualityof life, WHO functional class, and Global Clinical Impression.In FUTURE-1, the observed exposure to bosentan was similar to that inchildren who participated in BREATHE-3. A subset of 11 patients hadpharmacokinetics evaluated for both 2 and 4 mg/kg doses. In thesepatients, exposure to bosentan was comparable at both doses:geometric mean AUC(tau) was 3577 ng x h/mL and 3371 ng x h/mL withbosentan 2 mg/kg and 4 mg/kg, respectively, and geometric mean Cmaxwas 583 ng/mL and 649 ng/mL, respectively.The safety and tolerability profile of bosentan was consistent withthat observed in previous placebo-controlled clinical trials in theadult population.An open-label safety extension, FUTURE-2, is ongoing to assesslong-term safety and outcome data.About Tracleer® in Digital Ulcers (DU)DUs are a manifestation of the underlying vasculopathy which iscentral to the pathophysiology of systemic sclerosis (SSc) andpivotal in the development of PAH in SSc, one of the leading causesof death in SSc. Endothelin, a pathogenic mediator, is implicated inthe underlying vasculopathy in SSc.DUs can be a frequent, persistent and debilitating complication ofSSc. They are caused by a reduction in the lumen of small bloodyvessels that decreases blood flow to the fingers and toes causingopen sores. DUs are painful, with a debilitating impact on patients'daily life, often making it impossible to work and undertake evensimple day-to-day activities, particularly those associated withfingertip function. Reducing the occurrence of new DUs is animportant and achievable treatment goal in SSc.In the EU, Tracleer® is indicated to reduce the number of new digitalulcers in patients with systemic sclerosis and ongoing digital ulcerdisease. Tracleer® has been shown to improve hand function (i.e.dressing and hygiene) in patients with scleroderma-induced digitalulcers.Requires attention to two significant safety concerns [2]: Potentialfor serious liver injury (including rare cases of liver failure andunexplained hepatic cirrhosis in a setting of close monitoring) -Liver monitoring of all patients is essential prior to initiation oftreatment and monthly thereafter. High potential for major birthdefects - Pregnancy must be excluded and prevented by two forms ofbirth control; monthly pregnancy tests should be obtained. Because ofthese risks, Tracleer® is only supplied through controlleddistribution.References1. Widlitz A, Barst RJ. Pulmonary Arterial Hypertension inchildren. Eur Respir J 2003;21:155-176.2. Tracleer® SmPC.3. Rosenzweig et al. Effects of long-term Bosentan in childrenwith pulmonary arterial hypertension. J. Am. Coll. Cardiol 2005;46:697-704.4. Farber HW; Loscalzo J. Mechanisms of disease: pulmonaryarterial hypertension. N. Eng. J. Med. 2004; 351:1655-65.5. Humbert M; Sitbon O; Simonneau G. Treatment of pulmonaryarterial hypertension. N. Eng. J. Med. 2004;351:1425-36.6. Humbert M; Morrell NW; Archer SL; et al. Cellular andmolecular pathobiology of pulmonary arterial hypertension. J. Am.Coll. Cardiol. 2004; 43: Suppl. 12: 13S-24S.Actelion LtdActelion Ltd is a biopharmaceutical company with its corporateheadquarters in Allschwil/Basel, Switzerland. Actelion's first drugTracleer®, an orally available dual endothelin receptor antagonist,has been approved as a therapy for pulmonary arterial hypertension.Actelion markets Tracleer® through its own subsidiaries in keymarkets worldwide, including the United States (based in South SanFrancisco), the European Union, Japan, Canada, Australia andSwitzerland. Actelion, founded in late 1997, is a leading player ininnovative science related to the endothelium - the single layer ofcells separating every blood vessel from the blood stream. Actelion'sover 2000 employees focus on the discovery, development and marketingof innovative drugs for significant unmet medical needs. Actelionshares are traded on the SIX Swiss Exchange (ticker symbol: ATLN) aspart of the Swiss blue-chip index SMI (Swiss Market Index SMI®).For further information please contact:Medical Media ContactElizabeth PerryPacker Forbes+44 208 772 1551elizabeth(at)packerforbes.comInvestor ContactRoland HaefeliVice President, Head of Investor Relations & Corporate CommunicationsActelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil+41 61 565 62 62+1 650 624 69 36http://www.actelion.comhttp://hugin.info/131801/R/1327164/312589.pdf --- End of Message ---Actelion Pharmaceuticals LtdGewerbestrasse 16 Allschwil SwitzerlandWKN: 936767; ISIN: CH0010532478; Index: SBIOM, SLIFE, SMCI, SMIEXP, SMIM, SPI, SPIEX;Listed: Main Market in SIX Swiss Exchange;
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