Actelion receives marketing approval for Opsumit (macitentan) in pulmonary arterial hypertension in Japan

Actelion Pharmaceuticals Ltd / Actelion receives marketing approval for Opsumit (macitentan) in pulmonary arterial hypertension in Japan . Processed and transmitted by NASDAQ OMX Corporate Solutions. The issuer is solely responsible for the content of this announcement.

ALLSCHWIL, SWITZERLAND - 26 March 2015 - Actelion (SIX: ATLN) announced today that Japan's Ministry of Health, Labor and Welfare granted marketing approval for Opsumit^® (macitentan) for the treatment of pulmonary arterial hypertension (PAH).

The approval was based on data from a local study conducted in Japan and the landmark global Phase III SERAPHIN study. In both studies, improvements in pulmonary vascular resistance, 6MWD, and WHO function class were observed. In the SERAPHIN study, treatment with macitentan 10 mg per day resulted in a 45% risk reduction (p <0.0001) of the composite morbidity-mortality endpoint when compared to placebo.

Satoshi Tanaka, Dr. med Sci. President of Actelion Japan commented: "Opsumit represents a major step forward for the management of PAH as the first and only approved PAH treatment with proven long-term outcome efficacy. We are delighted to be able to add Opsumit to our PAH portfolio alongside epoprostenol 'ACT' and Tracleer as another crucial element in successful PAH management for physicians across Japan."

Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: "This is a great achievement for Actelion Japan. This approval is based on the SERAPHIN data and the local Japanese study performed by Actelion Japan. I am very happy that the Japanese PAH patients can soon benefit from Opsumit, an innovative product discovered in our laboratories."

The most common adverse events that were reported in the SERAPHIN study at a frequency at least 3% greater on macitentan than on placebo were nasopharyngitis, headache, anemia, bronchitis, urinary tract infection, pharyngitis and influenza.

Opsumit was approved by the US FDA in October 2013 and by the EU Commission in December 2013. Launch activities are progressing rapidly, with market introductions in the US, EU, Australia, Canada and Switzerland.

Actelion Pharmaceuticals Japan will co-promote Opsumit with Nippon Shinyaku in Japan and the companies will jointly ensure that Opsumit is made available to patients as soon as possible.

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NOTES TO THE EDITOR

ABOUT OPSUMIT^® (MACITENTAN)

Opsumit (macitentan) is a novel dual endothelin receptor antagonist (ERA) that resulted from a tailored drug discovery process with the target of developing an ERA to address efficacy and safety [2,3].

ABOUT THE SERAPHIN STUDY

SERAPHIN (Study with an Endothelin Receptor Antagonist in Pulmonary arterial Hypertension to Improve cliNical outcome) was the largest and longest randomized, controlled study in PAH patients to include a clearly defined morbidity/mortality primary endpoint [1]. The pivotal Phase III study was designed to evaluate the efficacy and safety of Opsumit (macitentan) - a novel dual endothelin receptor antagonist that resulted from a tailored drug discovery process - through the primary endpoint of time to first morbidity and all-cause mortality event in patients with symptomatic PAH.

Global enrolment was completed in December 2009 with a total of 742 patients. Patients were randomized 1:1:1 to receive two different doses of macitentan (3 mg and 10 mg once daily) or placebo. Patients were allowed to receive PAH background therapy throughout the study, either PDE-5 inhibitors or oral/inhaled prostanoids. This event-driven study was conducted in 151 centers from almost 40 countries in North America, Latin America, Europe, Asia-Pacific and Africa, and was completed in the first half of 2012, with 287 patients having an adjudicated event.

ABOUT SERAPHIN STUDY DATA                                     

Patients were randomized to placebo (n=250), macitentan 3 mg (n=250), or macitentan 10 mg (n=242). The primary end point occurred in 46.4%, 38.0%, and 31.4% of the patients in these groups, respectively. The hazard ratio for macitentan 3 mg versus placebo was 0.70 (97.5% CI, 0.52 to 0.96; p=0.0108) and the hazard ratio for macitentan 10 mg versus placebo was 0.55 (97.5% CI, 0.39 to 0.76; p<0.0001). Worsening of pulmonary arterial hypertension was the most frequent primary end point event. The effect of macitentan on this end point was observed irrespective of background therapy for pulmonary arterial hypertension. [3]

ABOUT THE SAFETY AND TOLERABILITY PROFILE

The most common adverse events that were reported in the SERAPHIN study at a frequency at least 3% greater on macitentan than on placebo were nasopharyngitis, headache, anemia, bronchitis, urinary tract infection, pharyngitis and influenza.

PULMONARY ARTERIAL HYPERTENSION (PAH)

Pulmonary arterial hypertension (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 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.

REFERENCES

1. Pulido T et al. Macitentan and Morbidity and Mortality in Pulmonary Arterial Hypertension. N Engl J Med 2013;369:809-18.
2. Bolli MH et al. The Discovery of N-[5-(4-Bromophenyl)-6-[2-[(5-bromo-2-pyrimidinyl)oxy]ethoxy]-4-pyrimidinyl]-N'-propylsulfamide (Macitentan), an Orally Active, Potent Dual Endothelin Receptor Antagonist. J Med Chem. 2012; 55:7849-61.
3. Iglarz M. et al. Pharmacology of macitentan, an orally active tissue targeting dual endothelin receptor antagonist. J Pharmacol Exp Ther. 2008;327(3):736-745.

NIPPON SHINYAKU

For further information on Nippon Shinyaku please visit:

http://www.nippon-shinyaku.co.jp/english/index.html

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 2,400 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, 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 Allschwi
[phone]
http://www.actelion.com

 

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Actelion Pharmaceuticals Ltd
Gewerbestrasse 16 Allschwil Switzerland

ISIN: CH0010532478;