FDA Advisory Committee Unanimously Recommends Approval Of Bayer's Riociguat In Two Pulmonary Hypertension Indications

WHIPPANY, N.J., Aug. 6, 2013 /PRNewswire/ -- Bayer HealthCare today announced that the U.S. Food and Drug Administration's (FDA) Cardiovascular and Renal Drugs Advisory Committee recommended approval for investigational riociguat, proposed trade name Adempas™, in two forms of pulmonary hypertension.  The Committee voted 11 to 0 that riociguat should be approved for the treatment of pulmonary arterial hypertension [PAH] of WHO¹ Group 1.  The Committee also voted 11 to 0 that riociguat should be approved for the treatment of chronic thromboembolic pulmonary hypertension (CTEPH) of WHO Group 4.   

In February 2013, Bayer submitted a new drug application for riociguat in two indications: (i) the treatment of PAH (WHO Group 1) to improve exercise capacity, improve WHO functional class and delay clinical worsening; and (ii) the treatment of persistent/recurrent CTEPH (WHO Group 4) after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class.

"We appreciate the Committee's discussion today around the safe and appropriate use of riociguat and are pleased with the outcome of the votes," said Pamela A. Cyrus, MD, Vice President and Head of U.S. Medical Affairs, Bayer HealthCare Pharmaceuticals.  "If approved, riociguat will offer a new treatment option for patients with PAH and will also provide the first approved non-surgical treatment option for CTEPH patients who are inoperable or who have recurrent or persistent disease.  We look forward to continued dialogue with the FDA in order to make riociguat available to patients."

PAH and CTEPH are both life-threatening forms of pulmonary hypertension that cause significantly increased pressure in the pulmonary arteries. Riociguat is an investigational, oral medication for the treatment of adult patients with PAH or inoperable or persistent/recurrent CTEPH.  If approved by the FDA later this year, it would create a new class of therapy available in the U.S.  PH is associated with endothelial dysfunction, impaired synthesis of nitric oxide (NO) and insufficient stimulation of soluble guanylate cyclase (sGC). Riociguat stimulates sGC independent of NO and increases the sensitivity of sGC to NO.

Data presented at today's advisory committee meeting included results from the global Phase 3 clinical program, which enrolled 704 patients across two Phase 3 studies.  Both studies met their primary endpoint by demonstrating a statistically significant improvement in the six-minute walk test (6MWT), after 16 and 12 weeks respectively.  Riociguat was also associated with improvements across multiple, relevant, secondary endpoints in the studies.

The most common treatment-emergent adverse events with riociguat were headache, dizziness, dyspepsia, peripheral edema, nausea, diarrhea and vomiting. 

The advisory committee's vote will be taken into consideration by the FDA when making its decision on the approvability of Bayer's NDA for riociguat, which was submitted in February 2013. After acceptance of the NDA, the FDA granted riociguat priority review designation, which is given to drugs that have the potential to offer significant improvement in treatment or provide a treatment option where no adequate therapy exists.

About Pulmonary Arterial Hypertension (PAH)
In PAH, a rare and life-threatening disease, the blood pressure in the pulmonary arteries (the arteries that take de-oxygenated blood to the lungs from the heart) is significantly increased.  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. In most cases, PAH has no known cause and, in some cases, it can be inherited.

About Chronic Thromboembolic Pulmonary Hypertension (CTEPH)
CTEPH is also a rare and life-threatening disease in which it is believed that thromboembolic occlusion (organized 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, CTEPH is inoperable in an estimated 20 to 40 percent of patients, and, in some cases, the disease persists or reoccurs after surgery.

About Bayer HealthCare Pharmaceuticals Inc.
Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world's leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Medical Care, and Pharmaceuticals divisions. As a specialty pharmaceutical company, Bayer HealthCare provides products for General Medicine, Hematology, Neurology, Oncology and Women's Healthcare. The company's aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases.

Bayer^® and the Bayer Cross^® are registered trademarks of Bayer.

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 www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

¹ World Health Organization

Intended for U.S. media only

SOURCE Bayer HealthCare Pharmaceuticals

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