Bayer receives approval for Adempas® as first drug to treat rare heart and lung disease

Bayer receives approval for Adempas® as first drug to treat rare heart and 
lung disease 

    --  Adempas® (riociguat) offers new hope for patients suffering
        from chronic thromboembolic pulmonary hypertension (CTEPH)
    --  Adempas(®) is now available in Canada as the first and only
        drug indicated to manage the treatment of chronic
        thromboembolic pulmonary hypertension (CTEPH)(1,2)

TORONTO, Sept. 23, 2013 /CNW/ - Bayer Inc. announced today the Health Canada 
approval of Adempas® (riociguat). Adempas® is the first drug indicated to 
manage the treatment of inoperable, or persistent /recurrent chronic 
thromboembolic pulmonary hypertension (CTEPH) after surgery in adult patients 
with World Health Organization Functional Class II or III pulmonary 

CTEPH is an uncommon form of pulmonary hypertension (PH), a severe, 
progressive and life-threatening condition of the heart and lungs that affects 
up to several thousand people in Canada.(3 )Patients with PH develop high 
blood pressure in the arteries of the lungs, which causes breathlessness and 
fatigue, hindering their ability to work and carry out everyday activities, 
like walking - even a short distance - in some cases.(4,5,6,7,8)

"Before today, we had no proven drug treatments for patients with inoperable 
CTEPH or patients in whom surgery was not successful in curing their CTEPH. 
Adempas gives us an effective drug treatment with proven clinical efficacy and 
good tolerability," said Dr. John Granton, Head, Division of Respirology 
University Health Network, Mount Sinai Hospital, and Women's College Hospital. 
He added that, because of the complex nature of PH, "Patients should be 
referred to an expert PH centre as soon as possible for a thorough assessment 
and timely treatment."

Results from a major clinical trial showed that riociguat is the first ever 
drug to provide statistically significant clinical improvement in patients 
with inoperable CTEPH or persistent/recurrent PH disease at the end of 16 
weeks of treatment. Improvements were seen in a range of disease-related 
measures such as reduction in patients' resistance to blood flow in the 
arteries of the lungs, and in markers of disease severity.(9) Riociguat also 
significantly improved their exercise capacity measured by a six-minute walk 
test (6MWT).(9)

"Riociguat will be welcomed by patients with CTEPH who, up to now, have not 
had a proven drug treatment option available to them if their disease is 
inoperable or for those experiencing residual PH following surgery. CTEPH is a 
devastating diagnosis, and the symptoms of breathlessness, dizziness and 
fainting can be frightening and have a severe impact on daily activities. To 
have a treatment that achieves meaningful clinical improvements is a much 
needed step forward," said Frank Poon, President, Pulmonary Hypertension 
Association of Canada.

The standard and potentially curative treatment option for patients who have 
developed CTEPH is a surgical procedure called pulmonary endarterectomy that 
mechanically clears the blood vessels of the lungs of scar tissue caused by 
the disease.(10) However, the disease persists or recurs after surgery in up 
to 35% of patients.(11 )Many patients (20%-50%)(12,13) with CTEPH are not 
candidates for surgery and, like patients with residual PH, urgently need 
effective new treatments to manage their disease.(14)

Long-term trials of the riociguat study program in CTEPH are ongoing, and 
first results show that safety and tolerability as well as efficacy (change in 
6MWT) are sustained over one year.(15 )

About Pulmonary Hypertension 
PH is a severe, progressive, life-changing and life-threatening disorder of 
the heart and lungs in which blood pressure in the pulmonary arteries is above 
normal, and which can lead to heart failure and death.(4,5) Patients with PH 
develop a markedly decreased exercise capacity and a reduced quality of 
life.(8) The most common symptoms of PH include shortness of breath, fatigue, 
dizziness and fainting, all of which are worsened by exertion.(6,7) As the 
symptoms of PH are non-specific, diagnosis can be delayed by as much as two 
years.(8,16,17) Early diagnosis is essential and accurate identification of 
the PH type is essential as a delay in treatment initiation can have a 
negative impact on survival.(8,18) Continuous treatment monitoring is then 
vital to ensure that patients are receiving optimal care for their particular 
type and stage of disease.(8)

There are five different types of PH and each can affect the patient in a 
different way and every patient may have a different etiology and 
manifestation of PH.(8,10,16) For the best chance of success patients need to 
be treated at an expert PH centre. (8,19)

About Chronic Thromboembolic Pulmonary Hypertension (CTEPH)
CTEPH is a progressive and life-threatening disease and a type of PH in which 
it is believed that thromboembolic occlusion (organized blood clots) of 
pulmonary vessels gradually lead to increased blood pressure in the pulmonary 
arteries, resulting in an overload of the right heart.(8,10) CTEPH may evolve 
after prior episodes of acute pulmonary embolism, but the pathogenesis is not 
yet completely understood.(10) The standard and potentially curative treatment 
for CTEPH is pulmonary endarterectomy (PEA), a surgical procedure in which the 
blood vessels of the lungs are cleared of clot and scar tissue.(10 )However, a 
considerable number of patients with CTEPH (20%-50%) are inoperable and in up 
to 35% of patients, the disease persists or recurs after PEA. These patients 
need an effective pharmacological treatment.(14)

About Riociguat
Riociguat is a soluble guanylate cyclase (sGC) stimulator, the first member of 
a novel class of compounds developed by Bayer to target a key molecular 
mechanism underlying PH. sGC is an enzyme found in the cardiopulmonary system 
and the receptor for nitric oxide (NO). When NO binds to sGC, the enzyme 
enhances synthesis of the signaling molecule cyclic guanosine monophosphate 
(cGMP). cGMP plays an important role in regulating vascular tone, 
proliferation, fibrosis, and inflammation.

PH is associated with endothelial dysfunction, impaired synthesis of NO and 
insufficient stimulation of sGC. Riociguat( )has a unique mode of action - it 
sensitizes sGC to endogenous NO by stabilizing the NO-sGC binding. Riociguat 
also directly stimulates sGC via a different binding site, independently of 
NO. Riociguat, as a stimulator of sGC, addresses the issue of NO deficiency by 
restoring the NO-sGC-cGMP pathway, leading to increased generation of cGMP.

With its novel mode of action, riociguat has the potential to overcome a 
number of limitations of currently approved PAH therapies, including NO 
dependence. It is the first drug which has shown clinical benefits in CTEPH, 
where, until the approval of riociguat, no indicated pharmacological treatment 
was available.(1,2)

About Bayer in Canada
Bayer Inc. is a Canadian subsidiary of Bayer AG and the headquarters for its 
Canadian operations. Celebrating its 150(th) anniversary, Bayer AG is an 
international research-based group with core businesses in healthcare, crop 
science and innovative materials committed to creating a better life for all 
through science. In Canada, Bayer operates its healthcare business - 
Pharmaceuticals, Consumer Care, Diabetes Care, Animal Health and Radiology & 
Interventional - from its headquarters in Toronto, ON and its CropScience 
business from Calgary, AB. With more than 1,300 employees across the country, 
in 2012, Bayer had sales of $1.6 billion and invested $55.9 million in 
research and development in Canada. Globally, Bayer AG had sales of €39.8 
billion and invested €3 billion in research and development.

For more information about Bayer Inc., please visit

Forward-Looking Statements
This release may contain forward-looking statements based on current 
assumptions and forecasts made by Bayer Group or subgroup management. Various 
known and unknown risks, uncertainties and other factors could lead to 
material differences between the actual future results, financial situation, 
development or performance of the company and the estimates given here. These 
factors include those discussed in Bayer's public reports which are available 
on the Bayer website at The company assumes no liability 
whatsoever to update these forward-looking statements or to conform them to 
future events or developments.

(1) Mayer, E. Surgical and post-operative treatment of chronic thromboembolic 
pulmonary hypertension. Eur Respir Rev 2010;19:64-67
(2) ADEMPAS Product Monograph, September 17, 2013
(3) Marc de Perrot, MD, Karen McRae, MD, Yaron Shargall, MD, Laura Pletsch, 
RN, Kongteng Tan, MD, Peter Slinger, MD, Martin Ma, MD, Narinder Paul, MD, 
Jakov Moric, MD, John Thenganatt, MD, Susanna Mak, MD, and John T. Granton, 
MD. Clinical Research Pulmonary Endarterectomy for Chronic Thromboembolic 
Pulmonary Hypertension: The Toronto Experience. Canadian Journal of Cardiology 
27 (2011) 692-697
(4) Rosenkranz, S. Pulmonary hypertension: current diagnosis and treatment. 
Clin Res Cardiol 2007;96:527-541
(5) Macchia, A et al. A meta-analysis of trials of pulmonary hypertension: A 
clinical condition looking for drugs and research methodology. Am Heart J 
(6) McKenna, S et al. The Cambridge Pulmonary Hypertension Outcome Review 
(CAMPHOR): A measure of health-related quality of life and quality of life for 
patients with pulmonary hypertension. Qual Life Res 2006;15:103-115
(7) PHA UK website. Available from: Last accessed: May 2013
(8) Galiè, N et al. 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;30:2493-2537
(9) Ghofrani, HA et al. Riociguat for the treatment of inoperable chronic 
thromboembolic pulmonary hypertension: a randomized, double-blind, 
placebo-controlled study (CHEST-1). ACCP 2012, Atlanta, USA. Oral abstract 
(10) Ali, JM et al Chronic thromboembolic pulmonary hypertension: An 
underdiagnosed entity? Hosp Pract 2012;40:71-9
(11) Rahnavardi M, Yan TD, Cao C, Vallely MP, Bannon PG, Wilson MK. Pulmonary 
thromboendarterectomy for chronic thromboembolic pulmonary hypertension: a 
systematic review. Ann Thorac Cardiovasc Surg. 2011;17(5):435-445.
(12) Mayer E, Jenkins D, Lindner J, et al. Surgical management and outcome of 
patients with chronic thromboembolic hypertension: results from an 
international prospective registry. J Cardiovasc. Surg. 2011. 702-710.
(13) Humbert, M. Pulmonary arterial hypertension and chronic thromboembolic 
pulmonary hypertension: pathophysiology. Eur Respir Rev 2010;19:59-63
(14) Humbert, M. Pulmonary arterial hypertension and chronic thromboembolic 
pulmonary hypertension: pathophysiology. Eur Respir Rev 2010;19:59-63
(15) Simonneau, G et al. Riociguat for the treatment of chronic thromboembolic 
pulmonary hypertension (CTEPH): A Phase III long-term extension study 
(CHEST-2). 5th World Symposium of Pulmonary Hypertension (WSPH) 2013, Nice, 
France. Poster Presentation.
(16) Armstrong, I et al. The trajectory to diagnosis with pulmonary arterial 
hypertension: a qualitative study. BMJ Open 2012; 2:e000806
(17) Peacock, JA. Treatment of pulmonary hypertension. BMJ 2003; 326:835-836
(18) Vachiery, J-L et al. How to detect disease progression in pulmonary 
arterial hypertension. Eur Respir Rev 2012; 21:40-47
(19) Ghofrani, HA et al. Treatment of pulmonary arterial hypertension (PAH): 
updated recommendations of the Cologne Consensus Conference 2011. Int J 
Cardiol 2011;154(Suppl 1):S20-S33

SOURCE  Bayer Inc. 
Laura Burns 416-240-5484 
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