US Food and Drug Administration grants priority review for Actelion's
miglustat in Niemann-Pick type
(Thomson Reuters ONE) - Corporate news announcement processed and transmitted by Hugin AS.The issuer is solely responsible for the content of this announcement. ------------------------------------------------------------------------------------ ALLSCHWIL/BASEL, SWITZERLAND - 19 November 2009 - Actelion Ltd (SIX:ATLN) announced today that a supplemental new drug application (sNDA)for an extension of indication for Zavesca® (miglustat) for thetreatment of progressive neurological manifestations in adult andpediatric patients with Niemann-Pick type C disease (NP-C) has beenaccepted by the U.S. Food and Drug Administration (FDA).In the US, Zavesca® is currently indicated for the oral treatment ofadult patients with mild to moderate type 1 Gaucher disease whereenzyme replacement therapy is unsuitable or is not a therapeuticoption.The sNDA, based on results from the clinical trial OGT 918-007, andtwo multicenter retrospective cohort studies in patients with NP-C,has been granted a priority review. A priority review designation isgiven to drugs that offer major advances in treatment, or provide atreatment where no adequate therapy exists. It also means that theFDA will aim to complete the review within 6 months.Actelion has been informed by the FDA that this sNDA will be reviewedby the Endocrine and Metabolic Drug Advisory Committee (EMDAC) on12th January 2010. The FDA often seeks the advice of an AdvisoryCommittee when evaluating potential treatments for diseases, forinstance when there is no approved therapy available.Jean-Paul Clozel, M.D. and Chief Executive Officer commented:"Actelion is working closely with the FDA to provide information asneeded to support the review process and make miglustat available inthe United States to patients suffering from this fatalneurodegenerative genetic disorder affecting both children andadults. Miglustat could become the first treatment for NP-C in theUSA, which would represent a major therapeutic breakthrough forpatients and their treating physicians".In 2008, FDA granted orphan drug status to miglustat for NP-C in theUnited States. ###Notes to the editorAbout Zavesca® (miglustat)Zavesca® (100 mg miglustat capsule) is indicated for the oraltreatment of adult patients with mild to moderate type 1 Gaucherdisease. Zavesca® may only be used in the treatment of type 1 Gaucherpatients for whom enzyme replacement therapy is unsuitable or is nota therapeutic option. It is approved in the European Union, theUnited States, Canada, Switzerland, Brazil, Australia, Turkey,Israel, South Korea, New Zealand and Russia.In the European Union, South Korea, Brazil and Russia, Zavesca® isalso indicated for the treatment of progressive neurologicalmanifestations in adult patients and pediatric patients withNiemann-Pick type C disease.About Niemann-Pick type C diseaseNP-C is a very rare, fatal, neurodegenerative, genetic condition,primarily affecting children and teenagers but which can strike atany age. The symptoms are caused by the storage of someglycosphingolipids within certain cells in the body, including thebrain. It is invariably progressive and most patients die within fiveto ten years of diagnosis; for the majority the disease is fatalduring childhood. Neurological deterioration is the key feature ofthe disease, and can manifest itself as clumsy body movements,balance problems, slow and slurred speech, difficulty in swallowing,problems with eye movements and seizures. Intellectual decline isalso common. In the final stages of the disease the child or youngadult is frequently bedridden, has little muscle control and isintellectually impaired. Diagnosis of the disease can be difficultand lengthy due to its rarity and heterogeneity.Zavesca® safety informationGastrointestinal events, mainly diarrhea, have been observed in morethan 80% of patients treated with Zavesca®, either at the onset oftreatment or intermittently during treatment. The majority of casesare mild and are expected to resolve after the first weeks ontherapy. In clinical practice, diarrhea has been observed to respondto diet modification (reduction of lactose and other carbohydrateintake), to taking Zavesca® away from meals, and/or to antidiarrhealmedicinal products such as loperamide. In some patients, temporarydose reduction may be necessary. Patients with chronic diarrhea orother persistent gastrointestinal events that do not respond to theseinterventions should be investigated according to clinical practice.Zavesca® has not been evaluated in patients with a history ofsignificant gastrointestinal disease, including inflammatory boweldisease.Cases of peripheral neuropathy have been reported in patients withtype 1 Gaucher disease treated with Zavesca®. Peripheral neuropathyseems to be more common in patients with type 1 Gaucher diseasecompared to the general population. All patients should undergobaseline and repeat neurological evaluation. Patients who developsymptoms such as numbness and tingling should have a carefulre-assessment of risk benefit.Zavesca® may cause fetal harm if administered to a pregnant woman andis contraindicated in women who are or who may become pregnant;patients should be informed of the potential hazard to the fetus.There is a risk of impaired fertility in men. Men should maintainreliable contraceptive methods and not plan to conceive while takingZavesca® and for three months thereafter.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 2'200 employees focus on the discovery, development andmarketing of innovative drugs for significant unmet medical needs.Actelion shares are traded on the SIX Swiss Exchange (ticker symbol:ATLN) as part of the Swiss blue-chip index SMI (Swiss Market IndexSMI®).For further information please contact:Roland HaefeliVice President, Head of Investor Relations & Public AffairsActelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil+41 61 565 62 62+1 650 624 69 36http://www.actelion.comhttp://hugin.info/131801/R/1356015/329390.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;
Datum: 19.11.2009 - 07:00 Uhr
Sprache: Deutsch
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