Actelion Pharmaceuticals Ltd / Zavesca® (miglustat) receives positive vote from the FDA's Endocrinologic and Metabolic Drugs Advisory Committee 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 - 12 January 2010 -Actelion Ltd (SIX: ATLN)
announced today that United States (US) Food and Drug Administration's (FDA)
Endocrinologic and Metabolic Drugs Advisory Committee voted (10 yes to 3 no) in
its final vote in question that the benefit/risk profile of Zavesca® (miglustat)
supports its approval for the treatment of progressive neurological
manifestations in adult patients and pediatric patients with Niemann-Pick type C
(NP-C) disease. NP-C disease is a very rare, relentlessly progressive and
eventually fatal neurodegenerative genetic disorder for which no specific
treatment is currently approved in the US.
The decision was based on results from the clinical trial OGT 918-007 and two
multicenter NP-C disease cohort studies as well as other clinical trials in
related lysosomal storage disorders for the safety and tolerability evaluation.
Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: "We
are pleased that the Advisory Committee today recognized the importance of this
therapeutic advance and voted to recommend approval of the supplemental New Drug
Application (sNDA) of Zavesca® for the treatment of NP-C disease. We will
continue to work closely with the FDA to facilitate the completion of the
priority review of the sNDA."
Zavesca® is the only specific treatment available for patients with NP-C
disease. It received approval in the European Union (EU) and other countries in
2009. Zavesca® is also indicated in the US, the EU and other countries for the
oral treatment of adult patients with mild to moderate type 1 Gaucher disease
for whom enzyme replacement therapy is unsuitable or is not a therapeutic
option. The use of Zavesca® is supported by over 10 years of clinical trials and
post-marketing experience across indications.
The FDA often seeks the advice of an Advisory Committee when evaluating
potential treatments for diseases, for instance when there is no approved
therapy available. TheFDA is not bound by the committee's guidance, but takes
its recommendation into consideration.
The decision of the FDA for the sNDA is expected in March 2010.
About Niemann-Pick type C disease
NP-C disease is a very 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 this
condition.
For more information:
http://podcasts.mayoclinic.org/2008/07/11/niemann-pick-disease-type-c/
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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 and Russia, Zavesca® is also
indicated for the treatment of progressive neurological manifestations in adult
patients 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 1900 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< http://www.actelion.com/>
[HUG#1372896]
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Actelion Pharmaceuticals Ltd
Gewerbestrasse 16 Allschwil Switzerland
WKN: 936767;ISIN: CH0010532478;
Press Release PDF: http://hugin.info/131801/R/1372896/336308.pdf