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Actelion Pharmaceuticals Ltd: Actelion receives Health Canada approval for Uptravi (selexipag) for the long-term treatment of pulmonary arterial hypertension

Actelion Pharmaceuticals Ltd / Actelion receives Health Canada approval for
Uptravi (selexipag) for thelong-term treatment of pulmonary arterial
hypertension . Processed and transmitted by NASDAQ OMX Corporate
Solutions.The issuer is solely responsible for the content of this
ALLSCHWIL, SWITZERLAND - 26 January 2016 -
Actelion (SIX: ATLN) announced today that Health Canada has granted a Notice
of Compliance (NOC) approving the orally active, selective IP prostacyclin
receptor agonist Uptravi (selexipag),originally discovered and synthesized by
Nippon Shinyaku, for the treatment of pulmonary arterial hypertension.

Uptravi®is indicated for the long-term treatment of idiopathic pulmonary
arterial hypertension (iPAH), heritable pulmonary arterial hypertension
(HPAH), PAH associated with connective tissue disorders and PAH associated
with congenital heart disease, in adult patients with WHO functional class
(FC) II-III to delay disease progression. Disease progression included
hospitalization for PAH, initiation of intravenous or subcutaneous
prostanoids, or other disease progression events (decrease of 6-minute walk
distance [6MWD] associated with either worsened PAH symptoms or need for
additional PAH-specific treatment). Uptravi is effective in combination with
an endothelin receptor antagonist (ERA) or a phosphodiesterase-5 (PDE-5)
inhibitor, or in triple combination with an ERA and a PDE-5 inhibitor, or as

Jean-Paul Clozel, MD and Chief Executive Officer of Actelion commented: "This
approval represents a major milestone: the approval of a new oral medication
that effectively targets the prostacyclin pathway. Uptravi is supported by
robust long-term outcome results in combination with an ERA, or a PDE-5
inhibitor, and, for the first time in PAH, in triple combination with both an
ERA and a PDE-5 inhibitor. We are now working diligently to make Uptravi
available to patients in Canada as soon as possible, since we know how
eagerly anticipated it is."

The safety of selexipag has been evaluated in a long-term, Phase III
placebo-controlled study enrolling 1,156 patients with symptomatic PAH. The
mean treatment duration was 76.4 weeks (median 70.7 weeks) for patients
receiving selexipag versus 71.2 weeks (median 63.7 weeks) for patients on
placebo. The exposure to selexipag was up to 4.2 years.

The most commonly reported adverse drug reactions related to the
pharmacological effects of Uptravi are headache, diarrhoea, nausea and
vomiting, jaw pain, myalgia, pain in the extremity, flushing, and arthralgia.
These reactions are more frequent during the dose titration phase. The
majority of these reactions are of mild to moderate intensity.


GRIPHON was a long-term, global Phase III study in 1,156 patients treated for
up to 4.2 years. The GRIPHON study, in which more than 80% of patients were
already receiving PAH-specific therapies, showed that the risk of the primary
composite endpoint was reduced by 40% (p<0.0001) with selexipag compared to

The benefit of selexipag was consistent across pre-specified patient subgroups
such as disease etiology, functional class and baseline PAH therapy,
including patients already receiving combination therapy with an ERA and a
PDE-5 inhibitor.

Titrating selexipag to an individualized maintenance dose based on
tolerability was effective in achieving long-term outcome benefits across the
tested dose range. The dosing in GRIPHON was initiated at 200 micrograms
(mcg) twice daily (b.i.d.) and increased weekly in steps of 200 mcg up to a
maximum of 1600 mcg b.i.d. or to the patient's individual highest tolerated
dose. The benefit was consistent across the pre-specified low (200, 400 mcg
b.i.d.), medium (600, 800, 1'000 mcg b.i.d.) and high maintenance (1'200,
1'400, 1'600 mcg b.i.d.) dose groups.




In December 2014, Actelion submitted the registration dossier for selexipag to
both the US FDA and Europe's EMA. Approval from the FDA was received on 21
December 2015, approval was also received from Health Canada on 21 January
2016. Submission of the registration dossier to other health authorities is
ongoing, with regulatory reviews underway in Australia, Japan, New Zealand,
South Korea, Switzerland, Taiwan and Turkey.


Uptravi (selexipag), originally discovered and synthesized by Nippon Shinyaku,
is a potent, oral, selective IP prostacyclin receptor agonist formulated as a

Uptravi and its major metabolite selectively target the prostacyclin receptor
(also called IP receptor). The IP receptor is one of 5 major types of
prostanoid receptor (IP, EP, DP, TP, FP). Prostacyclin activates the IP
receptor to induce vasodilation and inhibit proliferation of vascular smooth
muscle cells.


PAH is a chronic, life-threatening disorder characterized by abnormally high
blood pressure in the arteries between the heart and lungs of an affected
individual. The symptoms of PAH are non-specific and can range from mild
breathlessness and fatigue during normal daily activity to symptoms of right
heart failure and severe restrictions on exercise capacity and ultimately
reduced life expectancy.

PAH is one group within the classification of pulmonary hypertension (PH).
This group includes idiopathic PAH, heritable PAH and PAH caused by factors
which include connective tissue disease, HIV infection and congenital heart

The last decade has seen significant advances in the understanding of the
pathophysiology of PAH, which has been paralleled with developments of
treatment guidelines and new therapies. Drugs targeting the three pathways
that have been established in the pathogenesis of PAH are endothelin receptor
antagonists (ERAs), prostacyclin analogs and phosphodiesterase-5 inhibitors.
PAH treatments have transformed the prognosis for PAH patients from
symptomatic improvements in exercise tolerance 10 years ago to delayed
disease progression today. Improved disease awareness and evidence-based
guidelines developed from randomized controlled clinical trial data have
highlighted the need for early intervention, goal-oriented treatment and
combination therapy.

1 Sitbon O et al. Selexipag for the Treatment of Pulmonary Arterial
Hypertension. N Engl J Med 2015; 373:2522-33.
2 Kuwano et al. A long-acting and highly selective prostacyclin receptor
agonist prodrug, NS-304, ameliorates rat pulmonary hypertension with unique
relaxant responses of its active form MRE-269 on rat pulmonary artery. J
Pharmacol Exp Ther 2008;326:691-699.
3 Kuwano K et al.
cetamide (NS-304), an orally available and long-acting prostacyclin
receptor agonist prodrug. J Pharmacol Exp Ther 2007;322(3):1181-1188.
4 Tetsuo Asaki et al. Selexipag: an oral and selective IP prostacyclin
receptor agonist for the treatment of pulmonary arterial hypertension. J.
Med. Chem., Just Accepted Manuscript. DOI: 10.1021/acs.jmedchem.5b00698.
Web: 20 Aug 2015.
5 Morrison et al. Selexipag: a selective prostacyclin receptor agonist that
does not affect rat gastric function. J Pharmacol Exp Ther
6 Morrison et al. Differential effects of selexipag and prostacyclin analogs
in rat pulmonary artery. J Pharmacol Exp Ther 2012;343:547-555.
7 Simonneau G, Lang I, Torbicki A, Hoeper MM, Delcroix M, Karlocai K, Galie
N. Selexipag, an oral, selective IP receptor agonist for the treatment of
pulmonary arterial hypertension Eur Respir J 2012; 40: 874-880.
8 Mubarak KK. A review of prostaglandin analogs in the management of patients
with pulmonary arterial hypertension. Respir Med 2010;104:9-21.


For further information on Nippon Shinyaku please visit:


Actelion Ltd. is a leading biopharmaceutical company focused on the discovery,
development and commercialization of innovative drugs for diseases with
significant unmet medical needs.

Actelion is a leader in the field of pulmonary arterial hypertension (PAH).
Our portfolio of PAH treatments covers the spectrum of disease, from WHO
Functional Class (FC) II through to FC IV, with oral, inhaled and intravenous
medications. Although not available in all countries, Actelion has treatments
approved by health authorities for a number of specialist diseases including
Type 1 Gaucher disease, Niemann-Pick type C disease, Digital Ulcers in
patients suffering from systemic sclerosis, and mycosis fungoides type
cutaneous T-cell lymphoma.

Founded in late 1997, with now over 2,400 dedicated professionals covering all
key markets around the world including Europe, the US, Japan, China, Russia
and Mexico, Actelion has its corporate headquarters in Allschwil / Basel,

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®). All
trademarks are legally protected.

For further information please contact:

Andrew C. Weiss
Senior Vice President, Head of Investor Relations&Corporate Communications
Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil
+41 61 565 62 62

The above information contains certain "forward-looking statements", relating
to the company's business, which can be identified by the use of
forward-looking terminology such as "estimates", "believes", "expects",
"may", "are expected to", "will", "will continue", "should", "would be",
"seeks", "pending" or "anticipates" or similar expressions, or by discussions
of strategy, plans or intentions. Such statements include descriptions of the
company's investment and research and development programs and anticipated
expenditures in connection therewith, descriptions of new products expected
to be introduced by the company and anticipated customer demand for such
products and products in the company's existing portfolio. Such statements
reflect the current views of the company with respect to future events and
are subject to certain risks, uncertainties and assumptions. Many factors
could cause the actual results, performance or achievements of the company to
be materially different from any future results, performances or achievements
that may be expressed or implied by such forward-looking statements. Should
one or more of these risks or uncertainties materialize, or should underlying
assumptions prove incorrect, actual results may vary materially from those
described herein as anticipated, believed, estimated or expected.

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