拜耳（Bayer）3月31日宣布，肺动脉高压药物Adempas（riociguat）获欧盟委员会（EC）批准，作为单药疗法或联合内皮素受体拮抗剂，用于2种类型的肺动脉高压：a）用于肺动脉高压（PAH）成人患者；b）用于不能手术或手术后持续性、复发性慢性阻塞性肺动脉高压（CTEPH）成人患者。在欧盟，Adempas是首个也是唯一一种获批用于CTEPH的治疗药物。此前，欧盟已授予Adempas孤儿药地位。

Adempas的获批，是基于2项随机、双盲、安慰剂对照、全球性III期研究CHEST-1和PATENT-1、以及CHEST-2和PATENT-2目前获得的的长期数据。这些研究分别调查了口服riociguat治疗CTEPH和PAH的有效性和安全性。2项III期研究CHEST-1和PATENT-1均达到了研究的主要终点，此外riociguat在横跨多个相关次要终点均表现出持续改善，同时耐受性良好。

此前，Adempas已于2013年10月获FDA批准用于CTEPH和PAH的治疗。在加拿大，Adempas分别于2013年9月和2014年3月获批用于CTEPH和PAH的治疗。在瑞士和日本，Adempas分别于2013年11月和2014年1月获批用于CTEPH的治疗。

PAH和CTEPH是2种罕见的、危及生命的肺动脉高压，其特点是肺动脉压力明显增加。

     美国FDA去年10月已批准这款药物用于相同的适应症。拜耳将Adempas列入了其最有前景的新药名单之中，预测年销售峰值会超过5亿欧元（6.88亿美元）。

关于

Riociguat是拜耳开发的首个新一类可溶性鸟苷酸环化酶(sGC)激动剂，能够直接刺激sGC，增强其对低水平一氧化氮（NO）的敏感度，该药开发用于治疗2种肺动脉高压――慢性血栓栓塞性肺动脉高压(CTEPH)和肺动脉高压(PAH)，改善患者的运动能力，riociguat将以品牌名Adempas销售。

拜耳预计，该药的销售峰值将超过5亿欧元（约合6.46亿美元）。汤姆森路透发布分析报告称，预计该药在2017年的销售将达到6.79亿美元，同时将对市面上来自Actelion和吉利德（Gilead）的药物构成潜在的威胁。

Adempas® is approved for patients with chronic-thromboembolic pulmonary hypertension (CTEPH) and pulmonary arterial hypertension (PAH) / Adempas® is the first and only drug approved to treat patients with CTEPH

Berlin, March 31, 2014 C Bayer today announced that Adempas® (riociguat) has been approved by the European Commission for the treatment of chronic thromboembolic pulmonary hypertension (CTEPH) and pulmonary arterial hypertension (PAH). Adempas is indicated for the treatment of adult patients with inoperable chronic thromboembolic pulmonary hypertension (CTEPH) or persistent or recurrent CTEPH after surgical treatment, and for the treatment of adult patients with pulmonary arterial hypertension (PAH) as a monotherapy or in combination with endothelin receptor antagonists. Adempas is a soluble guanylate cyclase (sGC) stimulator, and the first member of a novel class of compounds. Adempas is the first and only drug treatment approved for patients with CTEPH.

“With the approval of Adempas by the European Commission, an imp0rtant new treatment option becomes available for patients with pulmonary hypertension in Europe,” said Dr. Jörg Möller, Member of the Bayer HealthCare Executive Committee and Head of Global Development. “Adempas is the first drug that has shown benefits across multiple clinically-relevant endpoints in two pulmonary hypertension indications, namely CTEPH and PAH. For the first time, there is a pharmacological treatment option for patients with CTEPH who are not eligible for surgery or whose disease persists or reoccurs.”

“The availability of riociguat is a significant step forward: It means that patients with CTEPH for the first time have an option if pulmonary endarterectomy (PEA), a highly specialized surgical procedure, is not an option for them or when the disease persists after surgery. Rather than struggling to breathe each day, with riociguat they can be more active again. The therapy enables patients to take part in everyday activities which many of us take for granted,” said Pisana Ferrari from the European Pulmonary Hypertension Assocation. “We also welcome the availability of a new class of medicines to help patients suffering from PAH meet their treatment goals.”

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 material. Riociguat is the first and only drug to demonstrate significant and sustained clinical efficacy in patients with inoperable CTEPH or persistent or recurrent CTEPH after surgical treatment. Riociguat is the first oral treatment to show early, significant and sustained clinical efficacy in Phase III clinical trials across multiple clinically relevant endpoints in patients with PAH, either as a monotherapy or in combination with certain other medicines used to treat PAH, such as endothelin receptor antagonists (ERAs) or non-intravenous prostacyclin analogue (PCA) therapies. So far no other oral drugs, including PDE5-inhibitors, have been able to show this. The consistency and robustness of the positive results in the Phase III trials demonstrated that riociguat relieved many of the symptoms experienced by people living with CTEPH or PAH. Riociguat significantly improved the patient’s ability to walk farther, helping the heart and lungs work better and making breathing easier when performing everyday basic tasks. Consequently, both CTEPH and PAH patients who received riociguat observed reductions in disease severity and these improvements are sustained over the long-term.

“With riociguat we have for the first time an effective drug treatment for two forms of PH. Under treatment with riociguat, the patient’s quality of life is rapidly improving, which is noticeable to them and clearly visible to us,” commented Professor Ardeschir Ghofrani, University Hospital Giessen and Marburg, Germany, Principal Investigator in the pivotal Phase III trials CHEST and PATENT.

The approval of Adempas is based on results from the two randomized, double-blind, placebo-controlled, global Phase III studies CHEST-1 and PATENT-1 as well as long-term data from CHEST-2 and PATENT-2 available at the time. These assessed the efficacy and safety of oral riociguat in the treatment of CTEPH and PAH respectively. Results of both studies were published in the New England Journal of Medicine (NEJM) in July 2013.

With the approval in the EU, the global prospective Adempas exposure registry EXPERT is started, which will collect data on the safety and clinical effect of this first-in-class sGC-stimulator in real-life clinical practice.

ab0ut Pulmonary Hypertension

Pulmonary hypertension (PH) is a severe, progressive, life-changing and life-threatening disorder of the heart and lungs in which the blood pressure in the pulmonary arteries is above normal, and which can lead to heart failure and death. Patients with PH develop a markedly decreased exercise capacity and a 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 and accurate identification of the PH type are 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.

There are five different types of PH; each can affect the patient in a different way and every patient may have a different etiology and manifestation of PH. For the best chance of success patients need to be treated at a PH specialist center.

ab0ut Pulmonary Arterial Hypertension (PAH)

PAH, one of the five types of pulmonary hypertension (PH), is a progressive and life-threatening disease in which the blood pressure in the pulmonary arteries is significantly increased due to vasoconstriction and which can lead to heart failure and death. PAH is characterized by morphological changes to the endothelium of the artery 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 is a rare disease and affects an estimated 15-52 people per million globally. It is more preva1ent in women than men. In most cases, PAH has no known cause and, in some cases, it can be inherited.

In spite of several pharmacological treatment options for PAH having been available for over a decade, the prognosis for these patients has remained poor and so new treatment options are needed. Currently, mortality of PAH patients remains high and is still 15% at 1 year and 32% at 3 years after diagnosis.

ab0ut 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 an increased blood pressure in the pulmonary arteries, resulting in an overload of the right heart. CTEPH is a rare disease and is comparable in terms of population size to PAH, as it affects an estimated 8-40 people per million globally. CTEPH may evolve after prior episodes of acute pulmonary embolism, but the pathogenesis is not yet completely understood. 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 material. However, a considerable number of patients with CTEPH (20%-40%) are not operable and in up to 35% of patients, the disease persists or reoccurs after PEA. These patients need an effective pharmacological treatment.

ab0ut Riociguat

Riociguat is a soluble guanylate cyclase (sGC) stimulator, the first member of a novel class of compounds, discovered and developed by Bayer as an oral treatment to target a key molecular mechanism underlying PH. Riociguat is being investigated as a new and specific approach to treat different types of PH. sGC is an enzyme found in the car

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