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U. S. FDA Grants Priority Review to Bayer’s Riociguat for the Treatment of
two Life-Threatening Pulmonary Hypertension Indications
Berlin, April 8, 2013 - Bayer HealthCare announced today that the U.S. Food
and Drug Administration (FDA) has granted priority review to the New Drug
Application (NDA) for the oral investigational drug riociguat. At the
beginning of February, Bayer HealthCare submitted riociguat, the first drug
to demonstrate efficacy in two distinct forms of pulmonary hypertension
(inoperable chronic thromboembolic pulmonary hypertension (CTEPH) and
pulmonary arterial hypertension (PAH) ) for regulatory approval in the
United States and in the European Union.
The FDA grants priority review to medicines that offer major advances in
care or that provide a treatment where no adequate therapy exists. Under
the Prescription Drug User Fee Act (PDUFA), the FDA aims to complete its
review within eight months from the submission of the NDA, rather than the
standard 12-month review cycle.
"In the pivotal Phase III studies, riociguat was shown to be the first drug
therapy to demonstrate robust efficacy in two potentially fatal pulmonary
hypertension indications," said Dr. Kemal Malik, member of the Bayer
HealthCare Executive Committee and Head of Global Development. "To date, no
approved pharmacological therapy exists for CTEPH and as a result there is
an urgent unmet medical need for patients who are unable to undergo
surgery. We hope that we will soon be in a position to provide patients and
doctors with a new treatment option."
The submission is supported by data from the two randomized, double-blind,
placebo-controlled pivotal, global Phase III studies, namely CHEST-1 and
PATENT-1. Results of both studies were presented at the 2012 annual meeting
of the American College of Chest Physicians (ACCP) in Atlanta, USA. Both
Phase III studies with riociguat met their primary endpoint, a change in
exercise capacity, after 12- or 16 weeks respectively. Riociguat was
generally well tolerated, with a good safety profile.
Bayer HealthCare announced positive data from the interim analysis of the
on-going CHEST-2 trial with riociguat, the open-label long-term extension
of the pivotal Phase III study CHEST-1, at the 5th World Symposium of
Pulmonary Hypertension (WSPH) in Nice, France. The results of the CHEST-2
trial support the positive data of the pivotal CHEST-1 trial, showing
long-term safety and sustained clinical benefits in patients with
inoperable chronic thromboembolic pulmonary hypertension (CTEPH).
Riociguat was discovered by Bayer and is the first member of a novel class
of compounds, the stimulators of soluble guanylate cyclase (sGC).
About Pulmonary Hypertension
Pulmonary hypertension (PH) is a severe, progressive and life-threatening
disorder in which the pressure in the pulmonary arteries is significantly
increased and which can lead to heart failure and death. Patients with PH
develop a markedly decreased exercise tolerance and reduced quality of
life. The most common symptoms of PH include shortness of breath, fatigue,
dizziness and fainting, all of which are worsened by exertion. As the
symptoms of PH are non-specific, diagnosis can be delayed by as much as two
years. Early diagnosis is essential as a delay in treatment initiation can
have a negative impact on survival. Continuous treatment monitoring is then
vital to ensure that patients are receiving optimal care for their
particular type and stage of disease.
According to the clinical classification of PH (Dana Point), there are five
different types of PH based on underlying causes which are: pulmonary
arterial hypertension (PAH), pulmonary hypertension owing to left heart
disease (e.g. PH-LVD), pulmonary hypertension owing to lung disease and/or
hypoxemia (e.g. PH-COPD or PH-ILD), chronic thromboembolic pulmonary
hypertension (CTEPH) and pulmonary hypertension with unclear multifactorial
mechanisms. Currently available pharmacological treatments are only
approved to treat one of the five types of PH, pulmonary arterial
hypertension. As a result, there is a strong need for more research to
improve understanding of how all five types of PH can be treated
About Pulmonary Arterial Hypertension (PAH)
PAH is a rare but life-threatening disease in which the pressure in the
pulmonary arteries is above normal. PAH is characterized by morphological
changes to the endothelium of the arteries of the lungs causing remodeling
of the tissue, vasoconstriction and thrombosis-in-situ. As a result of
these changes, the blood vessels in the lungs are narrowed, making it
difficult for the heart to pump blood through to the lungs. PAH affects an
estimated 52 people per million globally. It is more prevalent in younger
women than men. In most cases, PAH has no known cause and, in some cases,
it can be inherited.
Despite the availability of several approved therapies, the prognosis for
patients with PAH remains poor.
About Chronic Thromboembolic Pulmonary Hypertension (CTEPH)
CTEPH is a rare but life-threatening disease in which it is believed that
thromboembolic occlusion (blood clots) of pulmonary vessels gradually lead
to an increased pressure in the pulmonary arteries, resulting in an
overload of the right heart. CTEPH may evolve after prior episodes of acute
pulmonary embolism, but the pathogenesis is not yet completely understood.
The standard treatment for CTEPH is pulmonary endarterectomy (PEA), a
surgical procedure in which the blood vessels of the lungs are cleared of
clot and scar material. However, a considerable number of patients with
CTEPH are not operable and in some patients the disease persists or
reoccurs after PEA. Currently, there are no approved pharmacological
treatments available for CTEPH.
Riociguat (BAY 63-2521) is an oral agent being investigated as a new
approach to treating different types of pulmonary hypertension. Riociguat
is the first member of a novel class of compounds, the stimulators of
soluble guanylate cyclase (sGC). sGC is an enzyme found in the
cardiopulmonary system. When nitric oxide (NO) binds to sGC, the enzyme
catalyzes synthesis of the signaling molecule cyclic guanosine
monophosphate (cGMP). cGMP plays an important role in regulating vascular
tone, proliferation, fibrosis, and inflammation.
Pulmonary hypertension is associated with endothelial dysfunction, impaired
synthesis of NO and thus insufficient stimulation of sGC. Riociguat is
believed to have a unique mode of action: sensitizing sGC to endogenous NO
and also directly stimulating sGC independent of NO.
About Bayer HealthCare
The Bayer Group is a global enterprise with core competencies in the fields
of health care, agriculture and high-tech materials. Bayer HealthCare, a
subgroup of Bayer AG with annual sales of EUR 18.6 billion (2012), is one
of the world’s leading, innovative companies in the healthcare and medical
products industry and is based in Leverkusen, Germany. The company combines
the global activities of the Animal Health, Consumer Care, Medical Care and
Pharmaceuticals divisions. Bayer HealthCare’s aim is to discover, develop,
manufacture and market products that will improve human and animal health
worldwide. Bayer HealthCare has a global workforce of 55,300 employees (Dec
31, 2012) and is represented in more than 100 countries. More information
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