, April 8, 2013
/PRNewswire/ -- Bayer HealthCare announced today that the New Drug Application (NDA) for its oral investigational compound riociguat has been accepted for filing and granted priority review by the U.S. Food and Drug Administration (FDA) for the treatment of inoperable chronic thromboembolic pulmonary hypertension (CTEPH) or with persistent or recurrent CTEPH after pulmonary endarterectomy (PEA) and pulmonary arterial hypertension (PAH).
Both CTEPH and PAH are life-threatening diseases. CTEPH is a form of pulmonary hypertension in which blood clots and thromboembolic occlusion of pulmonary vessels leads to increased pressure in the pulmonary arteries. PAH is a disease characterized by elevated pressure in the pulmonary arteries.
"We are pleased that the FDA has granted priority review of the riociguat NDA for two distinct forms of pulmonary hypertension," said Pamela A. Cyrus, MD, Vice President and Head of U.S. Medical Affairs, Bayer HealthCare Pharmaceuticals. "This milestone reinforces our commitment to advancing treatment options for cardio-pulmonary diseases, and brings us one step closer to potentially providing the first pharmacological treatment for inoperable or persistent/recurrent CTEPH and a new treatment option for PAH."
The FDA grants priority review to medicines that provide a treatment where little or no adequate therapy exists. Under the Prescription Drug User Fee Act (PDUFA), the FDA aims to complete its review within six months of the 60-day filing receipt of the NDA submission (eight months total), rather than the standard 12-month review cycle.
The NDA submission is supported by data from two global Phase III studies of riociguat CHEST-1 and PATENT-1. Data from these two studies were presented in October 2012 at CHEST, the annual meeting of the American College of Chest Physicians (ACCP). Both Phase III studies on riociguat met their primary endpoint.
CHEST (Chronic Thromboembolic Pulmonary Hypertension sGC-Stimulator Trial) is a Phase III trial to assess the efficacy and safety of oral riociguat in the treatment of patients with either inoperable CTEPH or CTEPH which has persisted or reoccurred after pulmonary endarterectomy (PEA). CHEST is a multi-center, multi-national program with active centers in 26 countries. The program includes a randomized, double-blinded, placebo-controlled trial phase (CHEST-1) and an open label extension trial phase (CHEST-2).
In the CHEST-1 study, 261 patients with inoperable CTEPH or with persistent or recurrent CTEPH after PEA were randomized and treated with either riociguat or placebo orally for
16 weeks. The primary endpoint of the trial was improvement in 6-minute walking distance. Riociguat was titrated, over a period of eight weeks in doses of 0.5 mg increments, from 1.0 mg up to 2.5 mg, three times a day. After the titration phase, patients were followed up for another eight weeks on their last dose to complete CHEST-1. Patients from both arms then had the option of participating in the open label extension study (CHEST-2) after completing an eight-week blinded sham titration. CHEST-2 is continuing to investigate riociguat in CTEPH patients.
PATENT (Pulmonary Arterial Hypertension sGC-Stimulator Trial) is a Phase III trial to assess oral riociguat in the treatment of treatment naive patient and pre-treated patients with symptomatic PAH. PATENT is a multi-center, multi-national program with active centers in
32 countries. The program includes a randomized, double-blind, placebo controlled trial phase (PATENT-1) and an open label extension trial phase (PATENT-2).
In the PATENT-1 study, 445 patients with symptomatic PAH were randomized to receive either placebo or two different doses of riociguat orally over a period of 12 weeks. The primary endpoint of the trial was improvement in 6-minute walking distance. The riociguat dose was titrated, over a period of eight weeks in doses of 0.5 mg increments from 1.0 mg up to 2.5 mg three times daily. Patients were then followed for an additional four weeks to complete the study. Patients then had the option of participating in the open label extension study, after completing an eight-week blinded sham titration.
Riociguat (BAY 63-2521), discovered and developed at the Bayer research laboratories, is an investigational oral soluble guanylate cyclase (sGC) stimulator that is being studied in CTEPH and PAH, as well as other forms of pulmonary hypertension. Riociguat is an investigational agent and is not approved by the FDA, EMA or other health authorities.
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.
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SOURCE Bayer HealthCare