原创 默沙东新药获准，可治致命肺病，一针定价10万
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">肺动脉高压，一个听起来有些拗口的名词，是许多<span style="color: black;">病人</span>的噩梦。据统计，肺动脉高压<span style="color: black;">病人</span>五年死亡率接近一半，<span style="color: black;">药品</span>治疗<span style="color: black;">办法</span><span style="color: black;">亦</span>非常有限。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">但<span style="color: black;">这般</span>一种近乎“绝症”的<span style="color: black;">疾患</span>，如今迎来了治疗的新曙光。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">美国食品<span style="color: black;">药物</span>监督管理局（FDA）<span style="color: black;">准许</span>了默沙东<span style="color: black;">研发</span>的新药sotatercept用于治疗成人肺动脉高压。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">这不仅是对<span style="color: black;">病人</span>的福音，<span style="color: black;">亦</span>是医学界的一大突破。该<span style="color: black;">药品</span>或<span style="color: black;">作为</span><span style="color: black;">第1</span>个<span style="color: black;">能够</span>改变该<span style="color: black;">疾患</span>进程的治疗药。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><img src="//q8.itc.cn/images01/20240402/fde6f2a3356f4c9fbe1a6da524b56503.jpeg" style="width: 50%; margin-bottom: 20px;"></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">图源：默沙东官网</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">01</strong></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">可<span style="color: black;">控制</span>血管重塑，有望直接“治本”</strong></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">肺动脉高压是一种由多种<span style="color: black;">原由</span><span style="color: black;">导致</span>的肺血管阻力和压力<span style="color: black;">上升</span>的<span style="color: black;">疾患</span>，<span style="color: black;">最后</span>可能<span style="color: black;">引起</span>右心<span style="color: black;">衰尽</span><span style="color: black;">乃至</span>死亡。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">通俗一点说，血管就像一条河流，血液在其中自由流动。一旦河床上<span style="color: black;">起始</span>长出石头和杂草，水流会受阻，河流的活力就会受到影响。这<span style="color: black;">便是</span>肺动脉高压<span style="color: black;">病人</span>的<span style="color: black;">实质</span><span style="color: black;">状况</span>——<strong style="color: blue;"><span style="color: black;">她们</span>的肺血管<span style="color: black;">由于</span>种种<span style="color: black;">原由</span><span style="color: black;">起始</span>“长石头”，血液流动受阻，<span style="color: black;">引起</span>一系列健康问题。</strong></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">传统的治疗手段，如前列环素类似物、内皮素受体拮抗剂等，<span style="color: black;">重点</span>是<span style="color: black;">经过</span>不同途径达到舒张血管、降低肺动脉阻力的效果。<strong style="color: blue;"><span style="color: black;">这般</span>虽然能够缓解症状，但<span style="color: black;">没</span>法从<span style="color: black;">基本</span>上<span style="color: black;">处理</span>问题。</strong>就像是在一条堵塞的河流上搭建桥梁，虽然<span style="color: black;">能够</span>临时<span style="color: black;">经过</span>，但并<span style="color: black;">无</span>清除阻塞物。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">而这次<span style="color: black;">获准</span>的新药sotatercept则不同，它是一种first-in-class的融合蛋白，能够<span style="color: black;">控制</span>血管重塑的过程。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><img src="//q7.itc.cn/images01/20240402/4982ed5dd43e49c996bc562335edee4d.jpeg" style="width: 50%; margin-bottom: 20px;"></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">图源：参考文献2</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><span style="color: black;">详细</span><span style="color: black;">来讲</span>，sotatercept能够<span style="color: black;">控制</span>转化生长因子β（TGF-β）超家族成员释放的信号，这些信号<span style="color: black;">本来</span>会使内皮细胞和平滑肌细胞增生，<span style="color: black;">同期</span><span style="color: black;">控制</span>凋亡，<span style="color: black;">最后</span><span style="color: black;">引起</span>肺血管重塑。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">而sotatercept<span style="color: black;">能够</span><span style="color: black;">经过</span>减少这些细胞的增生和促进凋亡，达到<span style="color: black;">控制</span>肺血管重塑的效果。</strong></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">这一<span style="color: black;">功效</span>机制在肺动脉高压的治疗中是前所未有的，它<span style="color: black;">不仅</span>是在症状上做<span style="color: black;">文案</span>，而是直接针对<span style="color: black;">疾患</span>的根源进行治疗。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">02</strong></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;"><span style="color: black;">病人</span>恶化或死亡<span style="color: black;">危害</span>降低 84%</strong></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">这次FDA<span style="color: black;">准许</span>的依据是<span style="color: black;">重要</span>的III期临床<span style="color: black;">实验</span>（STELLAR<span style="color: black;">实验</span>），该结果于2023年3月6日<span style="color: black;">发布</span>于NEJM。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">该<span style="color: black;">实验</span>的<span style="color: black;">重点</span>终点是第24周6分钟步行距离相<span style="color: black;">针对</span>基线的变化，<strong style="color: blue;">这是衡量<span style="color: black;">病人</span>活动耐力的<span style="color: black;">要紧</span>指标</strong>。次要终点<span style="color: black;">包含</span>总体<span style="color: black;">状况</span>改善、肺血管阻力改变、WHO功能分级改善等。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">第24周时，<strong style="color: blue;">治疗组6分钟步行距离比基线平均<span style="color: black;">增多</span>40.1米</strong>（95%CI，29.9~50.2），安慰剂组为-1.4米（95%CI，-13.2~10.3）。这一结果<span style="color: black;">寓意</span>着<span style="color: black;">病人</span>能够走得更远，生活质量得到<span style="color: black;">明显</span><span style="color: black;">提高</span>。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><img src="//q0.itc.cn/images01/20240402/a5e3bb491a754341afe20297f95c0df7.jpeg" style="width: 50%; margin-bottom: 20px;"></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">图源：参考资料3</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">与安慰剂组相比，治疗组的8个次要终点均有<span style="color: black;">明显</span>改善。<strong style="color: blue;">更<span style="color: black;">要紧</span>的是，该<span style="color: black;">药品</span>将<span style="color: black;">病人</span>的临床恶化或死亡<span style="color: black;">危害</span>降低了84%。</strong></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">这就好比在一场与<span style="color: black;">疾患</span>的赛跑中，sotatercept给了<span style="color: black;">病人</span>更强的耐力和速度，让<span style="color: black;">她们</span>更有可能到达终点线。这一疗效的<span style="color: black;">明显</span>性为肺动脉高压治疗奠定了<span style="color: black;">基本</span>。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><img src="//q4.itc.cn/images01/20240402/96b55195479c47e89527c6b87115df1d.jpeg" style="width: 50%; margin-bottom: 20px;"></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">图源：参考资料4</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">但<span style="color: black;">必须</span><span style="color: black;">重视</span>的是，与安慰剂组相比，sotatercept组的不良反应<span style="color: black;">出现</span>更频繁，<span style="color: black;">包含</span>鼻<span style="color: black;">流血</span>、<span style="color: black;">头昏</span>、毛细血管扩张、血红蛋白水平<span style="color: black;">上升</span>、血小板减少和血压<span style="color: black;">上升</span>。且<span style="color: black;">重点</span>入组人群为白人，<span style="color: black;">针对</span>其他人种和其它类型肺动脉高压的适用性仍有待考量。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">03</strong></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">每瓶售价约10万人民币</strong></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><span style="color: black;">按照</span>默沙东<span style="color: black;">资讯</span>稿，sotatercept预计将于4月底在美国部分专业<span style="color: black;">药店</span>上市<span style="color: black;">营销</span>，<span style="color: black;">日前</span>透露的定价为<strong style="color: blue;">每剂14000美元（约合人民币10.1万元）</strong>；<span style="color: black;">按照</span>三周一剂的给药频率，<strong style="color: blue;">预计全年<span style="color: black;">花费</span>约243000美元（约合人民币175.7万元）</strong>。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">这个价格<span style="color: black;">针对</span>肺动脉高压<span style="color: black;">病人</span>可能是一个<span style="color: black;">没</span>法接受的高价。<span style="color: black;">由于</span>这类病人很容易因病致贫。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">肺动脉高压<span style="color: black;">病人</span>活动受限<span style="color: black;">引起</span>基本劳动能力缺乏，很难从事<span style="color: black;">通常</span>性工作。一<strong style="color: blue;">般肺动脉高压的治疗<span style="color: black;">花费</span>都由家庭其他成员和国家卫生<span style="color: black;">保证</span>、慈善基金等资金<span style="color: black;">源自</span>承担。</strong></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><span style="color: black;">思虑</span>到肺动脉高压<span style="color: black;">病人</span>的特殊<span style="color: black;">状况</span>，<span style="color: black;">关联</span><span style="color: black;">公司</span>和默沙东<span style="color: black;">亦</span>在寻求合理的定价策略，以<span style="color: black;">保证</span>所有符合<span style="color: black;">前提</span>的<span style="color: black;">病人</span>都能够<span style="color: black;">得到</span>并<span style="color: black;">包袱</span>得起这一治疗。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">总的<span style="color: black;">来讲</span>，sotatercept的<span style="color: black;">获准</span>为肺动脉高压<span style="color: black;">病人</span>带来了新的<span style="color: black;">期盼</span>。它不仅能够改善<span style="color: black;">病人</span>的生活质量，更有可能从<span style="color: black;">基本</span>上改变<span style="color: black;">疾患</span>的进程。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">随着这款<span style="color: black;">药品</span>的上市和普及，<span style="color: black;">咱们</span>有理由相信，肺动脉高压这一曾经难以<span style="color: black;">解决</span>的<span style="color: black;">疾患</span>，将会<span style="color: black;">逐步</span>变得可治可控。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">编辑 | 唐久</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">图源 | 摄图网</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">参考文献：</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">1.https://www.merck.com/news/fda-approves-mercks-winrevair-sotatercept-csrk-a-first-in-class-treatment-for-adults-with-pulmonary-arterial-hypertension-pah-who-group-1/</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">2.Humbert M, McLaughlin V, Gibbs JSR, et al. Sotatercept for the Treatment of Pulmonary Arterial Hypertension. N Engl J Med. 2021;384(13):1204-1215. doi:10.1056/NEJMoa2024277</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">3.https://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_ifu_1-vial_2-vial_kits.pdf.</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">4.Hoeper MM, Badesch DB, Ghofrani HA, et al. Phase 3 Trial of Sotatercept for Treatment of Pulmonary Arterial Hypertension. N Engl J Med. 2023;388(16):1478-1490. doi:10.1056/NEJMoa2213558<a style="color: black;"><span style="color: black;">返回首页，查看<span style="color: black;">更加多</span></span></a></p>
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