2009-11-19 07:04:03 -
London, November , 19, 2009
Corporate news announcement processed and transmitted by Hugin AS.
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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 the
treatment of progressive neurological manifestations in adult and
pediatric patients with Niemann-Pick type C disease (NP-C) has been
accepted by the U.S. Food and Drug Administration (FDA).
In the US, Zavesca® is currently indicated for the oral treatment of
adult patients with mild to moderate type 1 Gaucher disease where
enzyme replacement therapy is unsuitable or is not a therapeutic
option.
The sNDA, based on results from the clinical trial OGT 918-007, and
two multicenter retrospective cohort studies in patients with NP-C,
has been granted a priority review. A priority review designation is
given to drugs that offer major advances in treatment, or provide a
treatment where no adequate therapy exists. It also means that the
FDA will aim to complete the review within 6 months.
Actelion has been informed by the FDA that this sNDA will be reviewed
by the Endocrine and Metabolic Drug Advisory Committee (EMDAC) on
12th January 2010. 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.
Jean-Paul Clozel, M.D. and Chief Executive Officer commented:
"Actelion is working closely with the FDA to provide information as
needed to support the review process and make miglustat available in
the United States to patients suffering from this fatal
neurodegenerative genetic disorder affecting both children and
adults. Miglustat could become the first treatment for NP-C in the
USA, which would represent a major therapeutic breakthrough for
patients and their treating physicians".
In 2008, FDA granted orphan drug status to miglustat for NP-C in the
United States.
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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 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.
About Niemann-Pick type C disease
NP-C is a very rare, fatal, neurodegenerative, genetic condition,
primarily affecting children and teenagers but which can strike at
any age. The symptoms are caused by the storage of some
glycosphingolipids within certain cells 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 lengthy due to its rarity and heterogeneity.
Zavesca® safety information
Gastrointestinal events, mainly diarrhea, have been observed in more
than 80% of patients treated with Zavesca®, either at the onset of
treatment or intermittently during treatment. The majority of cases
are mild and are expected to resolve after the first weeks on
therapy. In clinical practice, diarrhea has been observed to respond
to diet modification (reduction of lactose and other carbohydrate
intake), to taking Zavesca® away from meals, and/or to antidiarrheal
medicinal products such as loperamide. In some patients, temporary
dose reduction may be necessary. Patients with chronic diarrhea or
other persistent gastrointestinal events that do not respond to these
interventions should be investigated according to clinical practice.
Zavesca® has not been evaluated in patients with a history of
significant gastrointestinal disease, including inflammatory bowel
disease.
Cases of peripheral neuropathy have been reported in patients with
type 1 Gaucher disease treated with Zavesca®. Peripheral neuropathy
seems to be more common in patients with type 1 Gaucher disease
compared to the general population. All patients should undergo
baseline and repeat neurological evaluation. Patients who develop
symptoms such as numbness and tingling should have a careful
re-assessment of risk benefit.
Zavesca® may cause fetal harm if administered to a pregnant woman and
is 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 maintain
reliable contraceptive methods and not plan to conceive while taking
Zavesca® and for three months thereafter.
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 2'200 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
www.actelion.com
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Actelion Pharmaceuticals Ltd
Gewerbestrasse 16 Allschwil
Switzerland
WKN: 936767; ISIN: CH0010532478; Index: SBIOM, SLIFE, SMCI, SMIEXP,
SMIM, SPI, SPIEX;
Listed: Main Market in SIX Swiss Exchange;
