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/
                                      ###

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