Sapropterin Dihydrochloride

先把时间拨回 2026 年 9 月 5 日。这一天上午八点零八分，全国人大会议中心西门，2026 年国家基本医保药品目录谈判竞价及商保创新药目录价格协商工作正式开谈，比往年 10 月底 11 月初的节奏提前了近一个半月。门口的工作人员开始点名核验，每半小时一轮、每轮五组并行。西装加口罩，是今年最常见的"国谈妆"。上午场第一批进场的是广誉远、康缘药业、华润三九等中药企业；下午场则是恒瑞、信达、辉瑞、诺和诺德、通化金马、勃林格殷格翰等 20 余家创新药企。 但我想讲的不是"哪些药跌了多少折"这个八卦。我更想说的是，今年的医保谈判，正在悄悄发生三件大事：基本医保和商保创新药目录第一次完整并行、谈判规则第一次被全面前置到审批阶段、以及首次正式启用"条件五"通道让 7 款高值商保品种重新冲击基本医保。这三件事叠在一起，意味着中国创新药"以价换量"的旧逻辑，已经走到必须重写的拐点。 补一句对很多读者最重要的话：医保谈判的结果要等到 11 月底才会通过"创新药高质量发展大会"一次性公布，2027 年 1 月 1 日起正式落地。所以现在你在网上看到的"某药谈成 / 谈崩"的消息，绝大多数是猜测和饭桌闲谈——现场签的是价格确认书，需经医保局复核、数据脱敏、目录统稿。下面讲的所有"砍价幅度"，也只是"降幅区间"的合理预期，不是既成事实。这是我今天想讲清楚的第一个原则。一、剧本：谁坐上了今年的谈判桌？ 先把今年的"演员表"理清楚。国家医保局 7 月 14 日公布的最终形式审查名单：基本医保目录通过初审 601 个药品通用名（总体通过率 91%），其中目录外新通用名 368 个、目录内续约 233 个；商保创新药目录通过初审 58 个通用名（通过率 94%）。目录外以近五年获批新通用名申报的药品达 343 个，创新药毫无疑问是今年调整的主角。 这意味着：每三个走进谈判桌的药里，就有一个是过去五年新上市的创新药。从 2018 年医保局成立时起，中国创新药"上市—进医保"的周期，从过去的平均 5 年缩短到了 1 年左右——80% 的创新药在上市两年内就被纳入医保支付范围。这条传导链是真金白银催出来的：医保基金每多报销一块钱，企业的研发意愿就多一分底气，2025 年医保新增 114 个药品里 50 个是 1 类创新药、谈判成功率 88%，创七年新高。 讲到这里必须说清楚一件容易被低估的事：今年的医保谈判，本质上是"基本医保+商保"两个目录同时谈判。基本医保坚持"保基本"底线，谈判失败的高价创新药可以转入商保目录继续价格协商；去年进入首版商保创新药目录的 7 款高值药品（多奈单抗、仑卡奈单抗、那西妥单抗、纳基奥仑赛、瑞基奥仑赛、泽尼达妥单抗、沙丙蝶呤），今年又反过来借助新增的"条件五"申报通道重新冲击基本医保。这种"基本医保保底、商保托底"的双层设计，是中国医疗保障制度演化的真正大事。二、AD 三剑客同台竞技——进口抗体 vs 国产胆碱酯酶抑制剂 阿尔茨海默病是今年国谈最具看点的赛道，没有之一。今年坐在谈判桌前的 AD 药一共有三款，作用机制几乎覆盖了三个完全不同的方向。补一句基础课：AD 过去 20 年的核心治疗手段是胆碱酯酶抑制剂（多奈哌齐、卡巴拉汀）和 NMDA 受体拮抗剂（美金刚）——只能缓解症状、不能延缓疾病进展。这两款药的有效性边界，是 Aβ 单抗（抗 β 淀粉样蛋白抗体）这种"对因治疗"想要打破的。 第一剑，是卫材/渤健的仑卡奈单抗（Lecanemab）。它是中国首个上市的 Aβ 单抗，2024 年 1 月获 NMPA 批准、6 月在复旦大学附属华山医院开出首针。它的 Ⅲ 期数据是：18 个月时认知功能下降速度减缓 27%，并把所有关键次要终点都做阳；模拟模型显示它能让早期 AD 患者在社区多停留 1.07 年、需要机构护理时间减少 0.11 年。临床意义要落到一句大白话：它不会让你记起来忘了什么，但能让"忘记"这件事发生得慢一点。挂网价约 2508 元/瓶（200mg/2mL），按体重 60kg 估算年费用约 18 万元。 第二剑，是礼来的多奈单抗（Donanemab）。中国 2024 年 12 月获批、2025 年 3 月正式上市，是全球首款靶向N3pG 修饰化 β 淀粉样蛋白斑块的单克隆抗体。它的"独门绝技"是有证据支持"可停药"——80.1% 的早期患者在治疗后达到淀粉样斑块清除标准，斑块平均减少 84%，相对安慰剂只有 1%。也就是说，用到"扫干净"就可以停。Ⅲ 期数据显示早期患者认知衰退减缓 35%、独立能力时间延长 37.3 个月，年费用约 22 万元，若按 18 个月停药策略全疗程 36 万元。 第三剑，是通化金马的琥珀酸安维吖啶片。它是中国首款自主研发的 AD 化学 1 类新药，2026 年 6 月 25 日获批上市，机制是同时抑制乙酰胆碱酯酶和丁酰胆碱酯酶（双胆碱酯酶抑制剂）。这个机制本质上属于"对症支持"——和二十年前的多奈哌齐属于同一家族，但选择性、可逆性、口服依从性都做了改良。通化金马的董事长张玉富亲自坐镇了 9 月 5 日的下午场。 这场三剑客对撞的最关键点不在价格，而在路径与定价。仑卡奈单抗和多奈单抗都已经进入 2025 年首版商保创新药目录——但运行半年的"成绩单"并不好看：截至 2026 年 1 月，仑卡奈单抗仅落地 198 家医院、多奈单抗仅 46 家，全国实际用药的患者加起来只有约 3000 人，不足 AD 患者总数的 0.1%。这印证了一个尴尬的事实：商保背书无法解决"支付深度"问题，不靠基本医保、20 万一年的年费就是绝大多数家庭够不到的天花板。 所以这次它们集体申请从商保转基本医保，是有备而来的。预计谈判后的目标区间会落在"年费用 10 万元以内"——但具体能谈到多少，等 11 月底。安维吖啶作为国产新药、未经过商保通道直接冲基本医保，定价空间相对宽裕；但如果仑卡奈单抗、多奈单抗最终都拿到了较低的医保价，对安维吖啶会形成"代差压制"。这场谈判表面是 Aβ 抗体 vs 胆碱酯酶抑制剂的机制对决，背后其实是进口抗体能否用"对因"溢价在基本医保里活下来的拷问。三、GLP-1 赛道——四款新品"神仙打架" 如果说 AD 是"机制的代际对决"，那 GLP-1 赛道就是"同代多点的血战"。今年谈判桌上一口气坐了四款肠促胰素类新药，分别是：信达生物的玛仕度肽（全球首个获批的 GCG/GLP-1 双激动剂）、辉瑞/先为达的埃诺格鲁肽（全球首款 cAMP 偏向型 GLP-1 受体激动剂）、派格生物的维培那肽（长效单靶 GLP-1）、诺和诺德的依柯胰岛素司美格鲁肽（依柯胰岛素+司美格鲁肽的复方制剂）。 为什么 GLP-1 这么热闹？因为中国糖尿病+肥胖合计潜在人群超过 2 亿。司美格鲁肽、替尔泊肽这些"前浪"已经在医保外稳坐百亿赛道，新一代"后浪"再不加医保，根本够不到中国基层的糖尿病人群。补一句行话：玛仕度肽是双靶点（同时激活 GCG 和 GLP-1 受体，减重效果比单靶更明显）；埃诺格鲁肽是"cAMP 偏向型"，意思是只激活 GLP-1 受体下游最有用的那条信号通路、副作用更少；维培那肽是国产长效，给药频次更低；依柯胰岛素司美格鲁肽是"复方"，把基础胰岛素和 GLP-1 装进同一支笔，主要给需要胰岛素强化治疗的 2 型糖尿病患者用。 临床价值侧，四个药的"创新价值"并不在同一档位：玛仕度肽是全球新机制，在双靶点里属于 first-in-class；埃诺格鲁肽是机理层面的"差异化"，强调减重同时不影响骨密度；维培那肽是国产长效化的工艺改进；依柯胰岛素司美格鲁肽是给药便捷性的组合创新。讲到这里得说清楚一件事：医保谈判里"创新"和"差异化"是两码事——前者决定能不能进、后者决定能谈到多少价。同一靶点、同类机制、同代际的"伪创新"，今年会被进一步收紧。国家医保局早已表态，"同质化"会被挡在形式审查之外。四、CAR-T 的百万天价——医保谈判的"终极考题" 如果说 AD 三剑客是 30 万级的"准奢侈品"，那 CAR-T 就是今年医保谈判的百万级"压轴考题"。今年坐在谈判桌前的 CAR-T 一共有四款：合源生物的纳基奥仑赛（定价 99.9 万元，是目前国内唯一低于百万元的 CAR-T）、药明巨诺的瑞基奥仑赛（定价 129 万元，国内最高）、恒润达生的雷尼基奥仑赛（未公开定价）、以及重庆精准生物的普基奥仑赛（首款儿童青少年复发难治 B 急淋 CAR-T，仅申报商保目录）。 为什么 CAR-T 是"终极考题"？因为 CAR-T 是一种活的细胞药——从患者身上抽出 T 细胞、在体外改造、扩增、回输，每个患者的药都是单独定制的。它不像化药或单抗可以工业化量产，成本曲线压不下来，强行砍价会打击整个行业的可持续研发。同时它的临床价值极其硬核：以纳基奥仑赛为例，2025 年 11 月新增大 B 细胞淋巴瘤第二适应症，复发难治患者中相当一部分可以"一次输注、长期缓解"——这种疗效用传统化疗、放疗、靶向药是做不到的。 讲到这里必须破除一个流传很广的误解："百万药"不等于"医保要全付"。基本医保的逻辑是按比例报销——以 99.9 万的纳基奥仑赛为例，若医保谈判降到 65 万、患者自付 30%，意味着每位入医保的患者医保实际支付 45 万左右、企业回款 65 万。医保谈判的真正难处不在"降到多少"，而在"多少患者能进这个池子"——纳基奥仑赛适用人群全国每年大约 2000-3000 人，这决定了医保基金可承受的总量。 因此今年 CAR-T 普遍选择"基本医保冲准入、商保扩覆盖"的双轨布局——基本医保谈出一个基准价锚定"医保患者"群体，商保创新药目录同步覆盖"商保患者"群体。这两条支付通道不是替代，而是互补。所以 CAR-T 能不能进医保、能不能谈到合理价，今年会成为中国创新药支付改革的标志性事件。五、双目录衔接——商保品种的"二次登陆" 讲到这里必须把今年最大的制度创新单独拎出来说一遍：基本医保+商保双目录并行，并通过"条件五"实现衔接。这件事的真正意义在于：它让过去"商保兜不住、医保进不来"的高值创新药，第一次有了完整的支付通路。 先说"条件五"是什么。今年国谈为已纳入 2025 年首版商保创新药目录的品种专门开辟了申报通道：这类药不再受"获批上市五年内"的时间门槛限制，可直接申报基本医保目录，同时支持商保目录品种新增适应症预申报。通过"条件五"通过初审的 7 款高值品种覆盖：神经退行性疾病（多奈单抗、仑卡奈单抗）、儿童罕见肿瘤（那西妥单抗）、罕见代谢病（沙丙蝶呤）、细胞治疗（纳基奥仑赛、瑞基奥仑赛）、HER2 高表达实体瘤（泽尼达妥单抗）。 这里得说清楚一件很多读者不会立刻反应过来的事："双目录衔接"不是"商保→医保"的单向阶梯。它是双向的——基本医保谈判失败的高价药，可以转入商保目录继续协商；商保目录已经成熟的品种，也可以反过来冲击基本医保。这种"双层支付"的真正含义，是给每一颗创新药都找到适合它的支付位置，不再用"保基本"这一根尺子量到底。 临床价值层面，7 款商保品种代表了高值创新药的几个典型方向：以仑卡奈单抗、多奈单抗为代表的慢病对因治疗（20 万/年级别，但能延缓疾病进展 25-35%）；以纳基奥仑赛、瑞基奥仑赛为代表的一次性细胞疗法（百万级，一次输注长期缓解）；以沙丙蝶呤为代表的罕见代谢病孤儿药（患者极少但无可替代）；以泽尼达妥单抗为代表的难治性实体瘤靶向治疗（HER2 高表达胆道癌，过去几乎无药可用）。它们的共同点是"无可替代的临床价值"——这不是"锦上添花"，而是"雪中送炭"。六、未来三年走向——准入前移、规则透明化、双目录成常态 讲完今年的精彩片段，我把视角拉到未来三年。这场谈判里藏着四个方向性的信号，每一个都和患者的钱袋子、企业的新药研发、医院的用药决策紧密相关。 信号一：准入节点全面前移。2026 年首次引入"预申报"机制——完成技术审评但尚未拿到注册批件的创新药可以先行申报，把上市和准入之间的"空窗期"压到最短。百利天恒的伦康依隆妥单抗（全球首个双抗 ADC）正是借助这一机制做到"6 月获批、9 月上谈判桌"的"无缝衔接"。配套的"参照药预沟通办法"今年 2 月施行后，企业可以在谈判前就和专家论证参照药、明确"跟谁比"。这两套组合拳把医保谈判从"事后博弈"变成了"事中共建"——规则前置、信息透明、企业不再"摸黑进场"。 信号二：续约规则"双算法取高值"。今年首次明确，协议期满 8 年的独家谈判药品在转入常规乙类目录时，支付标准核定采用"双算法取高值"原则——既不强行压价，也不让老独家品种"享受永恒红利"。同时谈判药品满 8 年且医保内销售额超 3 亿元的，统一降价 10% 简易续约后转入常规目录。这是中国医保首次明确"老品种退出谈判、转常规管理"的路径，对 2018 年前后那一批谈判成功的创新药来说，今年的"续约考"才是真正的"二次大考"。 信号三：双目录运行进入第二年，"保基本+商保衔接"成主旋律。李滔副局长在国新办发布会上明确表态："十五五"期间将持续坚持每年动态调整基本医保目录，将更多符合条件的肿瘤、罕见病、慢性病、儿童、老人等重点领域创新药纳入报销。换句话说，每年一调会成为常态。这对创新药企是好消息——不用再熬"四年一窗口"的焦灼；但对企业销售和合规也提出了更高要求——目录调整节奏和定价策略必须紧密配套。 信号四：商保兜底高值药、CAR-T 试水"百万级"谈判。商保创新药目录的扩容会让"百万级"细胞疗法、高值单抗、罕见病孤儿药进入"双层支付"体系。今年的纳基奥仑赛、瑞基奥仑赛、伦康依隆妥单抗都是试金石。如果双目录衔接机制跑通，中国未来 5 年可能会复制美国 PBM（药品福利管理）+ Medicare 的双层支付模式——基本医保托底、商保扩大覆盖、企业多渠道回款、患者多层次保障。这可能是中国医药行业未来十年最重要的支付改革。 讲到这里值得单独说一句创新药行业的"暗流"：医保打通"后端"回报，企业才有"前端"研发底气。国内市场的快速放量成了"压舱石"，让企业敢于用"凭底气闯世界"的方式去开拓海外授权、国际共同开发。这条传导链条如果断裂——比如同质化"伪创新"被一棍打死、真创新又谈不到合理价——中国的 first-in-class 管线就会失去资金回流。所以今年国谈最后的结果，无论谈成什么样，"创新药能不能拿到合理回报"这个核心命题，会比"具体降了多少幅"更值得被记住。七、给读者、给患者、给医保家庭的五条提醒 讲了这么多趋势和政策，最后还是得落回"和我有什么关系"。给读者五条提醒： 一、医保目录正式公布前，任何"已经进医保"的消息都不要轻信。今年的正式发布时间窗口是 11 月下旬创新药高质量发展大会，2027 年 1 月 1 日起正式落地。期间企业代表在谈判现场签的是价格确认书，结果前期严格保密。 二、不要把"进医保"等同于"用得起"。即使是仑卡奈单抗、多奈单抗这类已经商保覆盖的 AD 单抗，目前实际用药患者仍不足 AD 患者总数的 0.1%。医保谈判后患者自付部分仍是普通家庭需要认真规划的支出。先看疗效和适应症、再算经济账，是合理决策的核心。 三、"全球新"机制≠"对你更好"。玛仕度肽是 GCG/GLP-1 双激动剂，但具体是否适合你、是否能耐受得住胃肠反应、是否符合报销适应症，都是临床医生要评估的细节。不要在拿到处方前，仅凭宣传就锁定某款新药。 四、CAR-T 等细胞治疗不是"包治"。纳基奥仑赛、瑞基奥仑赛等 CAR-T 适用于复发难治的血液肿瘤患者，不是所有肿瘤都能用。具体是否适用、需要做哪些前期评估（T 细胞采集、淋巴细胞清除预处理方案），由血液科和移植科共同决策。 五、关注所在医院的"双通道"管理药品名单。医保谈判成功的药品在落地时往往通过"双通道"（定点医院 + 定点药店）保障供应，但具体哪些医院能用、是否能开出，需要看当地医保经办机构公布的"双通道"管理药品名单和医院药事管理委员会的决议。"进医保"不等于"进了你家门口的医院"。 讲到这里，我想把开头那个清晨七点的全国人大会议中心西门放回我们的视野。西装的领口、口罩的边缘、点名的节奏，每一年都在演同一出戏，但每年这出戏都换一些新角色。今年坐在桌前的，多了好几个从未出现的面孔：双抗 ADC、cAMP 偏向型 GLP-1、百万级的细胞疗法、双目录衔接机制下的高值药。"国谈"这两个字，从十年前"以价换量"的代名词，慢慢演化成"中国创新药支付改革"的代名词。 回看今年这场医保谈判，我最想留下的其实不是某个降幅数字，而是三件事。第一，医保谈判的真正主角从来不是"砍价幅度"，而是"创新药能不能拿到合理回报"——这件事决定了中国未来 10 年还能不能持续产出 first-in-class。第二，双目录衔接是中国医疗保障体系演化的关键一步，它让每一颗创新药都能找到适合它的支付位置，不再用"保基本"一根尺子量到底。第三，真创新和伪创新的分水岭正在医保谈判桌上变得越来越清晰——同质化"伪创新"会逐渐被拒于形式审查之外，真正不可替代的临床价值会被"双层支付"托底。 讲了这么多，最后想说的是：医保谈判的真正动人之处，不是"砍价"本身，而是它让中国患者和创新药企第一次站在同一个坐标系里——患者用得起、企业赚得到、医保可持续、临床不可替代——这是中国医药行业过去 10 年最大的一场"四向奔赴"。今年的谈判结果要等到 11 月底才会公布，但无论那张目录最终长什么样，这场"世纪大考"本身就已经是中国创新药行业最宝贵的成人礼。 作者：华北大药丸子，高级执业药师，您身边的药师朋友 | 专注用故事讲透每一颗药。 免责声明：本文为医保政策与药物科普，仅供信息交流，不构成任何诊疗或投资建议。本文提及的所有药品具体降价幅度、医保支付标准、报销比例、上市后实际可及性信息，以国家医保局 2026 年 11 月底正式发布的《国家基本医疗保险药品目录》以及《商保创新药目录》为准。文章中涉及的研发管线、临床试验数据、企业策略均综合自国家医保局、第一财经、21 世纪经济报道、财联社、搜狐、医药云端信息、Radix 药引、摩熵医药等公开报道。患者用药前请务必咨询相关科室医生，并结合自身情况、家庭经济能力、当地医保政策综合决策。

– 17 abstracts and presentations planned at SSIEM Symposium – – Real-world evidence demonstrates significant diet liberalization achieved with Sephience while maintaining target blood Phe levels – – Continued evidence of meaningful benefit across all patient subgroups – WARREN, N.J., Aug. 24, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that multiple Sephience™ (sepiapterin) scientific data presentations will be featured at the 2026 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, taking place in Helsinki, Finland, from Aug. 25-28. The presentations include new data from clinical trials and real-world evidence which reinforce the clinically meaningful benefits of Sephience on lowering phenylalanine (Phe), significant diet liberalization and sustained metabolic control for the full spectrum of individuals living with phenylketonuria (PKU) including those with classical PKU. In addition, Sephience studies continue to show a consistent and favorable safety profile. "The SSIEM presentations further demonstrate the broad clinical benefits of Sephience across the full spectrum of individuals living with PKU," said Matthew B. Klein, M.D., Chief Executive Officer. "In addition, new data to be presented at the PTC symposium show treatment with Sephience led to a large number of responsive participants achieving normalization of blood Phe levels (<120 µmol/L) in a rapid timeframe, including those with classical or non-BH4-responsive PKU. These impressive data support the potential benefits Sephience can deliver for individuals affected by PKU." Highlights of the data to be presented at SSIEM 2026 include: New analyses from the AMPLIPHY study demonstrate that Sephience treatment resulted in a 100% greater reduction in blood Phe for participants on sapropterin at screening after switching to Sephience. In an analysis of participants in Sephience studies with high baseline Phe levels (≥900 µmol/L), Sephience treatment resulted in clinically meaningful reductions in blood Phe levels within 14 days, with response rates comparable to the overall study population, and a safety profile consistent with prior studies. These findings support a trial of Sephience treatment in individuals with PKU regardless of severity or baseline Phe. An analysis of real-world data performed by a leading global key opinion leader shows that Sephience enabled significant dietary liberalization in adolescents, supporting greater independence, reduced dietary burden, and maintained metabolic control within recommended targets. These results were observed in individuals with both BH4-responsive and classical/non-BH4 responsive PKU mutations. About Sephience™ (sepiapterin) Sephience™ is indicated for the treatment of adult and pediatric patients with phenylketonuria (PKU). Sephience is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Through its unique dual mechanism of action, Sephience is able to effectively reduce blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients. Sephience is approved in the United States, the European Union/European Economic Area region, Japan and other countries. Indication and Important Safety Information Indication SEPHIENCE is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). SEPHIENCE is to be used in conjunction with a phenylalanine (Phe)-restricted diet. Contraindications None. Important Safety Information Treatment with SEPHIENCE should be directed by physicians knowledgeable in the management of PKU. Biochemical response to SEPHIENCE can only be determined by a therapeutic trial with careful monitoring of ongoing dietary and nutritional balance to ensure adequate Phe control. Warnings and Precautions Increased Bleeding: SEPHIENCE may increase the risk of bleeding. Bleeding events, including superficial hematomas, prolonged bleeding, and heavy menstrual bleeding have occurred in patients treated with SEPHIENCE. Inform patients about the risk of bleeding associated with SEPHIENCE and have patients follow up with their healthcare provider should such a bleeding event occur. Consider treatment interruption with SEPHIENCE in patients with active bleeding. Hypophenylalaninemia: Some pediatric patients receiving SEPHIENCE experienced hypophenylalaninemia. Monitor blood Phe levels during treatment and modify the dosage of SEPHIENCE and/or dietary protein and Phe intake as needed to ensure adequate blood Phe level control. Frequent blood monitoring is recommended in the pediatric population. Interaction with Levodopa: In a 10-year post-marketing safety surveillance program for a non-PKU indication using another drug that is a phenylalanine hydroxylase (PAH) activator, three patients with underlying neurological disorders experienced seizures, exacerbation of seizures, over-stimulation, and irritability during co-administration with levodopa. Monitor patients who are receiving levodopa for changes in neurological status during treatment with SEPHIENCE. Adverse Reactions Most common adverse reactions with SEPHIENCE (≥2% and > placebo) were diarrhea, headache, abdominal pain, hypophenylalaninemia, feces discoloration and oropharyngeal pain. Drug Interactions Avoid concomitant use of drugs known to inhibit folate synthesis dihydrofolate reductase (DHFR) (e.g., trimethoprim, methotrexate, trimetrexate, pemetrexed, pralatrexate, raltitrexed, and piritrexim) while taking SEPHIENCE. Concomitant administration of such drugs may reduce sepiapterin metabolism to BH4. If concomitant use is not avoidable, monitor blood Phe levels. SEPHIENCE and PDE-5 inhibitors (e.g., sildenafil, vardenafil, or tadalafil) induce vasorelaxation and may reduce blood pressure. Monitor for signs and symptoms of hypotension. For medical information, product complaints, or to report an adverse event, please call 1-866-562-4620 or email [email protected]. You may also report adverse events directly to FDA at 1-800-FDA-1088 or . Please see Full Prescribing Information. About Phenylketonuria Phenylketonuria (PKU) is a rare, inherited metabolic disease, characterized by the body's inability to break down an essential amino acid called phenylalanine (Phe) and which can result in neurological and other symptoms. If left untreated or poorly managed, Phe can build up to harmful levels in the body. This causes severe and irreversible disabilities, such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems. Newborns with PKU initially do not have any symptoms, but symptoms are usually progressive, and damage caused by toxic levels of Phe in the first few years of life is irreversible. Diagnosis of PKU usually takes place during newborn screening programs. There are an estimated 58,000 people living with PKU globally. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow us on LinkedIn, X, Instagram and Facebook. For More Information: Investors: Ellen Cavaleri +1 (615) 618-6228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, commercialization and other matters with respect to its products and product candidates; expectations with respect to Sephience; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including commercialization and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval, and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 21% more press release views with Request a Demo