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2016-02-29

Actelion Pharmaceuticals Ltd: Actelion emphasizes commitment to advance research and care in rare diseases

Actelion Pharmaceuticals Ltd / Actelion emphasizes commitment to advance
research and care in rare diseases. Processed and transmitted by NASDAQ OMX
Corporate Solutions. The issuer is solely responsible for the content of this
announcement.
"Patient Voice - Join us in making the voice of rare diseases heard." This is
the theme of Rare Disease Day 2016 - and a cause for Actelion to emphasize
its long-term commitment to support the needs of the patient community.

ALLSCHWIL, SWITZERLAND - 29 February 2016
- Today, on Rare Disease Day 2016, patients, their families and caregivers,
patient organizations, medical professionals, politicians and researchers are
coming together to raise awareness for what living with a rare disease means
for those affected and for their families.

Patients play a crucial role in bringing both research and the standard of
care in rare diseases forward, which is recognized in the theme of this
year's Rare Disease Day, 'Patient Voice'. For Actelion, listening to
patients' voices and working closely with the rare disease community is key
to delivering on the company's mission to discover new innovative drugs for
rare diseases and to help more patients with groundbreaking therapies. At the
end of 2015, over 65,000 patients were benefiting from Actelion's rare
disease drugs, and the company continues to work with governments and other
stakeholders to widen accessibility around the world.

On February 23 in Brussels, our commitment to rare diseases was publicly
recognized by the European Organization for Rare Diseases (EURORDIS) as
Actelion was honored with the EURORDIS Company Award 2016. Presented to
pioneering companies for the development of treatments for rare diseases, the
award in particular acknowledges Actelion's work with patient groups to
support the patient and carer community.

Through the company's collaborative work with patient groups and healthcare
professionals, Actelion has become a trusted partner in supporting activities
that raise awareness of the challenges facing those living with rare diseases
and help bring about positive change. Activities include the development of
educational resources such as the innovative PAHuman® eBook to better explain
the disease and its impact, and the "Think Again. Think NP-C" campaign to
encourage earlier diagnosis of Niemann-Pick type C disease among healthcare
professionals unfamiliar with this rare condition.

Gerald Fischer, CEO of the European Pulmonary Hypertension Association (PHA
Europe), said: "Actelion has provided invaluable support to our activities
and, as a result, our collaboration has led to many great achievements over
the years. It's clear that Actelion's commitment extends beyond the discovery
and development of new medicines and includes initiatives that increase
knowledge and understanding of PAH and other rare diseases, which is crucial
to organizations like ours."

An example of Actelion's pioneering work in rare diseases is an ongoing
clinical study involving patients with Eisenmenger syndrome. Working with
ethics committees, patient advocacy, support groups and patients' families,
Actelion has extended the study on Eisenmenger syndrome to Down syndrome
patients, which will help to address the high unmet medical need for
effective, targeted PAH therapies in this vulnerable and too rarely
considered population.

Martine Clozel, MD, Chief Scientific Officer at Actelion, commented: "I am
very proud that, together with the PAH community, Actelion has brought about
not one, but several paradigm shifts in PAH care. The three established
pathways in PAH are now well served, but our discovery efforts continue. We
are turning to new pathways in PAH, helped by our strong connections with
academia, to further improve the prognosis for patients living with this
devastating disease. Actelion also plays a leadership role in Niemann-Pick
type C disease, where the lives of many patients have been positively
impacted. I am convinced that with our strong and innovative research,
Actelion will continue to advance care in many rare diseases."

###

NOTES TO EDITOR:

ABOUT RARE DISEASE DAY 2016
Rare Disease Day takes place on the last day of February each year. Its main
objective is to raise awareness amongst the general public and
decision-makers about rare diseases and their impact on patients' lives,
Since Rare Disease Day was first launched by EURORDIS and its Council of
National Alliances in 2008, thousands of events have taken place throughout
the world, reaching hundreds of thousands of people. The Rare Disease Day
2016 theme 'Patient Voice - Join us in making the voice of rare diseases
heard'' recognizes the crucial role that patients play in voicing their needs
and in instigating change that improves their lives and the lives of their
families and caregivers. The theme appeals to a wider audience, those that
are not living with or directly affected by a rare disease, to join the
community in making known the impact of rare diseases. To learn more,
visithttp://www.rarediseaseday.org.

ABOUT PAH
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 disease. 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 receptor
agonists, 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. Learn more
athttp://www.pahuman.com/

ABOUT NIEMANN-PICK TYPE C DISEASE (NP-C)
NP-C disease is a treatable rare, 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. 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.
Think Again. Think NP-C is a project coordinated by the International
Niemann-Pick Disease Alliance with the collaboration and financial support of
Actelion Pharmaceuticals Ltd. A multidisciplinary advisory committee of
leading clinical experts in the field of NP-C provided advice and input into
the content of the campaign to ensure it would resonate with healthcare
professionals.http://think-npc.com/

ABOUT EISENMENGER SYNDROME
Eisenmenger syndrome is the most advanced form of pulmonary arterial
hypertension (PAH) in conjunction with congenital heart disease (CHD). Down
syndrome patients represent an important subset of the Eisenmenger population
(between 25% and 50%, depending on cohort studied). To address the high unmet
medical need for effective, targeted PAH therapies in this vulnerable
population, Actelion has extended its MAESTRO study with macitentan in
Eisenmenger syndrome patients to the Down syndrome community. To ensure
proper safeguards are established to protect the patients' rights and safety,
we have been working with ethics committees, patient advocacy, support groups
and patients' families. Our outreach was incredibly well received by all
parties involved, and their support has enabled us to enroll a number of Down
syndrome patients into the study. This is the first time that Down syndrome
patients afflicted with Eisenmenger syndrome are included in a randomized
clinical trial.

ABOUT THE EURORDIS AWARDS
Actelion has been honored by EURORDIS, the European Organization for Rare
Diseases, with the EURORDIS Company Award 2016. EURORDIS is a
non-governmental, alliance of patient organizations representing 705 rare
disease patient organizations in 63 countries. The purpose of the EURORDIS
Awards is to recognize the commitment and achievements of patients'
organizations, volunteers, companies, scientists, media and policy makers who
have contributed to reducing the impact of rare diseases on people's lives.
Read more about the 2016 winners onhttp://www.eurordis.org/eurordis-awards

ACTELION LTD
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 also 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 2,500 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,
Switzerland.

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
http://www.actelion.com

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