Actelion receives FDA complete response letter for Zavesca (miglustat) for the treatment of Niemann-Pick type C disease
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Actelion Pharmaceuticals Ltd / Actelion receives FDA complete response letter for Zavesca (miglustat) for the treatment of Niemann-Pick type C disease processed and transmitted by Hugin AS. The issuer is solely responsible for the content of this announcement.
ALLSCHWIL/BASEL, SWITZERLAND - 09 March 2010 - Actelion Ltd (SIX: ATLN)
announced today that the company has received a complete response letter from
the U.S. Food and Drug Administration (FDA) for its supplemental New Drug
Application (sNDA) for Zavesca® (miglustat) for the treatment of progressive
neurological manifestations in adult and pediatric patients with Niemann-Pick
type C (NP-C) disease, a rare, neurodegenerative genetic disorder.
The FDA has requested additional preclinical and clinical information. Actelion
will work diligently with the FDA to explore the best ways to address the points
raised by the agency.
Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: "We
remain committed to bringing an approved treatment to patients suffering from
NP-C disease and in this spirit we will continue the dialogue with the FDA."
About Niemann-Pick type C disease
NP-C disease is a rare, fatal, neurodegenerative, genetic condition, primarily
affecting children and teenagers although the clinical manifestations can become
apparent at any age. The symptoms are caused by the storage of some lipids -
such as glycosphingolipids and cholesterol - within certain tissues in the body,
including the brain. It is invariably progressive and most patients die within
five to ten years of diagnosis; for the majority the disease is fatal during
childhood.
Neurological deterioration is the key feature of the 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 is also common. In the final stages of the disease the child or young
adult is frequently bedridden, has little muscle control and is intellectually
impaired. Diagnosis of the disease can be difficult and may take years due to
the rarity and heterogeneity of the condition. There is no specific drug
therapy approved in the United States to treat NP-C disease.
###
Notes to the editor
About Zavesca® (miglustat)
Zavesca® (100 mg miglustat capsule) is indicated for the oral treatment of adult
patients with mild to moderate type 1 Gaucher disease. Zavesca® may only be used
in the treatment of type 1 Gaucher patients for whom enzyme replacement therapy
is unsuitable or is not a therapeutic option. It is approved for this indication
in the European Union, the United States, Canada, Switzerland, Brazil,
Australia, Turkey, Israel, South Korea, New Zealand and Russia.
In the European Union, South Korea, Brazil, Russia, Australia and Canada,
Zavesca® is also indicated for the treatment of progressive neurological
manifestations in adult and pediatric patients with Niemann-Pick type C
disease.
Zavesca® safety information
In clinical studies, the most common adverse events due to Zavesca® included
weight loss, diarrhea, and tremor. Other common adverse reactions were
flatulence, abdominal pain, headache, and influenza-like symptoms. The most
common serious adverse reaction was peripheral neuropathy. Patients should
undergo neurological examination at the start of treatment and every 6 months
thereafter; Zavesca® should be reassessed in patients who develop symptoms of
peripheral neuropathy. Zavesca® should not be used in pregnant woman. Men should
maintain reliable contraceptive methods while taking Zavesca® and for 3 months
after discontinuing treatment.
Actelion Ltd
Actelion Ltd is a biopharmaceutical company with its corporate headquarters in
Allschwil/Basel, Switzerland. Actelion's first drug Tracleer®, 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 key markets worldwide, including the United States (based in
South San Francisco), the European Union, Japan, Canada, Australia and
Switzerland. Actelion, founded in late 1997, is a leading player in innovative
science related to the endothelium - the single layer of cells separating every
blood vessel from the blood stream. Actelion's over 2,300 employees focus on the
discovery, development and marketing 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 Index
SMI®).
For further information please contact:
Roland Haefeli
Vice President, Head of Investor Relations & Public Affairs
Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil
+41 61 565 62 62
+1 650 624 69 36
http://www.actelion.com
[HUG#1392243]
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Actelion Pharmaceuticals Ltd
Gewerbestrasse 16 Allschwil Switzerland
WKN: 936767;ISIN: CH0010532478;
Press Release PDF: http://hugin.info/131801/R/1392243/349854.pdf
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Datum: 09.03.2010 - 11:45 Uhr
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