Actelion obtains "Epoprostenol ACT" label extension for pediatric PAH patients in Japan

ALLSCHWIL, SWITZERLAND - 02 March 2017 - Actelion (SIX: ATLN) announced today that Japan's Ministry of Health, Labor and Welfare granted a label extension for dosage and administration for "Epoprostenol ACT" (0.5 mg and 1.5 mg) for the treatment of pediatric patients with pulmonary arterial hypertension (PAH).

Dr. Satoshi Tanaka, President of Actelion Japan and Chairman of Actelion South Korea, commented: "Epoprostenol ACT" is the first epoprostenol to receive the pediatric indication in Japan. Adding this patient population to the product label allows us to offer physicians in Japan an option for intravenous therapy for young patients with advanced pulmonary arterial hypertension. "Epoprostenol ACT" will provide these patients and their families and caretakers with benefits that will make managing the disease easier, including greater stability of the medication and more flexibility with its preparation and administration."

Actelion Pharmaceuticals Japan will now work closely with all partners involved to ensure that "Epoprostenol ACT" is made available to pediatric patients as soon as possible.

Marketed in Japan as "Epoprostenol ACT", Veletri® (epoprostenol for injection), an intravenous prostacyclin, is commercially available in 17 markets worldwide, including the US (since 2010), Switzerland and - marketed as Caripul® - Canada (since 2012), as well as Japan and some European markets (since 2013). The registration process for other countries is ongoing.

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Notes to the Editor

ABOUT PULMONARY ARTERIAL HYPERTENSION (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 at http://www.pahuman.com/ 

ABOUT 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 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 2,600 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 by their respective owners.

For further information, please contact: 

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

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