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Actelion receives Therapeutic Goods Administration (TGA) approval in Australia for Opsumit (macitentan) in Pulmonary Arterial Hypertension (PAH)


February 7, 2014 - London

Actelion Pharmaceuticals Ltd /Actelion receives Therapeutic Goods Administration (TGA) approval in Australia for Opsumit (macitentan) in Pulmonary Arterial Hypertension (PAH) . Processed and transmitted by NASDAQ OMX Corporate Solutions.The issuer is solely responsible for the content of this announcement.

ALLSCHWIL/BASEL, SWITZERLAND - 07 February 2014 - Actelion Ltd (SIX: ATLN) today announced the approval of Opsumit® (macitentan) 10 mg for the treatment of pulmonary arterial hypertension (PAH) by the Therapeutic Goods Administration (TGA) of Australia.

Opsumit, as monotherapy or in combination with approved PAH treatments (phosphodiesterase-5 inhibitors or inhaled prostanoids), is indicated for the treatment of idiopathic and heritable PAH as well as PAH associated with connective tissue disease and PAH associated with congenital heart disease with repaired shunts in patients with WHO Functional class II,III or IV symptoms.

Professor Trevor Williams, Clinical Director of the Department of Allergy, Immunology and Respiratory Medicine at Alfred Hospital and Monash University, Melbourne, Australia commented, "The approval of Opsumit is a key milestone in the management of PAH. We now have confidence that we can improve long term clinical outcomes both in treatment naïve patients and those on combination therapy. Hopefully all new pulmonary arterial hypertension drugs will be subjected to such extensive level of rigorous clinical evaluation and demonstrate clinically important results."

The TGA's approval was based on data from the landmark Phase III SERAPHIN study. In the SERAPHIN study, treatment with macitentan 10 mg resulted in a 45% risk reduction (hazard ratio [HR] 0.55; 97.5% CI: 0.39 to 0.76; logrank p < 0.0001) of the composite morbidity-mortality endpoint when compared to placebo.

Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion gave his reaction to the news: "This approval marks a significant moment for the PAH community in Australia as Opsumit is the first and only PAH treatment with proven long-term efficacy in controlled clinical trials in PAH, and will now offer hope of a better future to those living with this disease. Actelion is now working to make this important medicine available to patients around Australia in the coming months."

The most common adverse events that were reported at a frequency at least 3% greater on macitentan than on placebo were nasopharyngitis (14.0% macitentan vs 10.4% placebo), headache (13.6% vs 8.8%) and anaemia (13.2% vs 3.2%)

Opsumit was approved by the US FDA in October 2013, Health Canada in November 2013 and by the EU Commission in December 2013. It is also undergoing regulatory assessment in other countries including Switzerland. 

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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 commonly reported adverse drug reactions with Opsumit are nasopharyngitis (14.0%), headache (13.6%) and anaemia (13.2%). 

ABOUT OPSUMIT (MACITENTAN) SUBMISSIONS TO HEALTHCARE AUTHORITIES

The first approval globally of the new drug application for Opsumit (macitentan) was issued by the US Food and Drug Administration (FDA) on 18 October 2013 for the treatment of pulmonary arterial hypertension (PAH, WHO Group I) to delay disease progression. Disease progression included: death, initiation of intravenous (IV) or subcutaneous prostanoids, or clinical worsening of PAH (decreased 6-minute walk distance, worsened PAH symptoms and need for additional PAH treatment). Opsumit also reduced hospitalization for PAH.

Regulatory reviews are ongoing in Switzerland, Taiwan, Mexico, Korea and Israel.

ABOUT PULMONARY ARTERIAL HYPERTENSION [4, 5, 6]

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), prostacyclins 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.

In PAH, survival rates are unacceptably low and PAH remains incurable.

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.
  4. Proceedings of the 4th world symposium on pulmonary hypertension. J Am CollCardiol 2009;54(1 Suppl).
  5. Galiè N, Hoeper MM, Humbert M, et al; ESC Committee for Practice Guidelines (CPG). Guidelines for the diagnosis and treatment of pulmonary hypertension: the Task Force for the Diagnosis and Treatment of Pulmonary Hypertension of the European Society of Cardiology (ESC) and the European Respiratory Society (ERS), endorsed by the International Society of Heart and Lung Transplantation (ISHLT). Eur Heart J 2009;30:2493-537.
  6. Benza RL, Miller DP, Barst RJ, Badesch DB, Frost AE, McGoon MD. An evaluation of long-term survival from time of diagnosis in pulmonary arterial hypertension from REVEAL. Chest 2012;142:448-56.

 

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 in patients with cutaneous T-cell lymphoma.

Founded in late 1997, with now over 2,400 dedicated professionals covering all key markets around the world including 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:

Roland Haefeli
Senior 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
www.actelion.com

 

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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Source: Actelion Pharmaceuticals Ltd via Globenewswire

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