ICONIC AS

▎药明康德内容团队报道10月9日，北海康成在研罕见病新药氯马昔巴特口服溶液迎来两项重要进展。一是向中国国家药监局药品审评中心（CDE）申报新适应症上市申请，并获得受理。二是在中国台湾地区获得批准上市，治疗1岁及以上阿拉杰里综合征（ALGS）患者胆汁淤积性瘙痒。氯马昔巴特口服溶液（maralixibat，迈芮倍）是一种几乎不被吸收的回肠胆汁酸转运蛋白（IBAT）抑制剂，可阻断胆汁酸肠肝回圈，降低肝内和血清中的胆汁酸水平，减少由此介导的肝脏损伤，缓解胆汁淤积瘙痒（极度瘙痒）。根据与Mirum公司的协议，北海康成拥有氯马昔巴特口服溶液在大中华区开发和商业化以及在特定条件下生产的独家授权，用于治疗包括胆道闭锁（BA）、ALGS和进行性家族性肝内胆汁淤积症（PFIC）在内的三种罕见肝病以及其他可选择的适应症。此前，氯马昔巴特口服溶液已在中国大陆和香港地区、加拿大、美国、欧盟等地获批上市，用于治疗ALGS患者胆汁淤积性瘙痒。此外，该药还获得FDA治疗ALGS和2型PFIC的突破性疗法认定，以及治疗ALGS、PFIC和BA的孤儿药物资格。在中国递交新适应症上市申请公开资料显示，此次为氯马昔巴特口服溶液在中国递交的第二项上市申请。今年9月，氯马昔巴特口服溶液已被CDE纳入优先审评，用于治疗2月龄及以上进行性家族性肝内胆汁淤积症（PFIC）患者的胆汁淤积瘙痒症。根据北海康成发布的2023年半年报，该公司预计于2023年下半年在中国大陆和台湾地区提交该产品用于治疗PFIC的新药申请。截图来源：CDE官网PFIC是一种可导致进行性肝病的罕见遗传性疾病，已在中国被纳入《第一批罕见病目录》。PFIC患者的肝脏分泌胆汁的能力较差，胆汁在肝内积聚会导致肝病，严重的会导致肝衰竭。该病的体征和症状通常始于婴儿期，患者会出现严重的瘙痒、黄疸、生长缓慢或无法生长，以及肝功能的逐渐丧失。目前已鉴定出6种类型的PFIC，均以胆汁流动受损和进展性肝病为特征。此前，氯马昔巴特口服溶液已在治疗PFIC的3期MARCH研究中取得了积极顶线数据。该研究评估了候选药在广泛亚型PFIC患者的疗效，共有93例年龄介于1-17岁的患者入组。结果显示，与接受安慰剂的患者相比，接受了氯马昔巴特口服溶液治疗的所有PFIC亚型的患者，主要与次要终点均获得了高度的统计学意义上的改善。其中主要终点为与基线时相比瘙痒严重程度的改善，而次要终点则是与基线相比的血清胆汁酸水平。在中国台湾地区获批上市根据北海康成新闻稿，氯马昔巴特口服溶液已在中国台湾地区获批上市，用于治疗1岁及以上阿拉杰里综合征（ALGS）患者胆汁淤积性瘙痒。ALGS是一种罕见遗传性疾病，可导致末期肝病甚至死亡。该病的特点是由于胆管缺乏而引起胆汁淤积，并累及肝外器官，例如肾脏、眼睛、骨骼和心血管系统。胆汁淤积性瘙痒是ALGS中最严重的症状之一，会大大降低患者生活质量。此次氯马昔巴特口服溶液的获批基于Mirum公司开展的关键ICONIC研究和RISE婴儿安全性研究的数据，以及多年对ALGS患者数据的收集。ICONIC研究显示，与安慰剂相比，氯马昔巴特口服溶液对于瘙痒症具有统计学及临床意义的减少，血清胆汁酸亦明显减少，两者在数年治疗内均可持久得到改善。RISE研究的中期数据支持该药用于小于12个月婴儿组的适应症。北海康成创始人、董事长兼首席执行官薛群博士表示，他们非常高兴迈芮倍在中国大陆和香港获批后六个月内，继续在中国台湾地区获批，这对于改善罕见病患者的生活具有重大的意义。将这一重要疗法引入中国台湾地区不仅践行了公司不断寻求创新突破的承诺，也展现了他们致力于为全球有需要的患者带来先进疗法的决心。随着迈芮倍在中国台湾地区上市，相信阿拉杰里综合征的认知度将得到大幅提高，诊断流程会更加简化并极大改善患者的护理现状。在此，衷心感谢合作伙伴Mirum在这一重要过程中所给予的大力支持。参考资料：[1]中国国家药监局药品审评中心（CDE）官网.Retrieved Oct 9，2023, From https://www.cde.org.cn/main/xxgk/listpage/2f78f372d351c6851af7431c7710a731[2]北海康成宣布治疗阿拉杰里综合征（ALGS）胆汁淤积性瘙痒的迈芮倍®在台湾获得批准上市.Retrieved Oct 9，2023, From https://mp.weixin.qq.com/s/mRibmyCYQIUyg2x5SC00Mw[3]北海康成发布截至2023年6月30日止六个月中期业绩公告. Retrieved Sep 4，2023. From https://mp.weixin.qq.com/s/Qqn7PTZCmxkq2zyBadqWHQ本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈。转发授权请在「医药观澜」微信公众号留言联系我们。其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。

▎药明康德内容团队报道9月28日，北海康成宣布，迈芮倍（氯马昔巴特口服溶液）已在中国香港地区获批上市。氯马昔巴特口服溶液是一种几乎不被吸收的回肠胆汁酸转运蛋白（IBAT）抑制剂，本次在中国香港地区获批治疗3个月及以上阿拉杰里综合征（ALGS）患者胆汁淤积性瘙痒。ALGS是一种罕见遗传性疾病，可导致末期肝病甚至死亡。ALGS的特点是由于胆管发育异常而引起胆汁淤积，并累及肝外器官，例如肾脏、眼睛、骨骼和心血管系统。胆汁淤积性瘙痒是ALGS中最严重的症状，会大大降低患者生活质量。氯马昔巴特口服溶液是一种口服、每日一次的回肠胆汁酸转运蛋白（IBAT）抑制剂，可以阻断胆汁酸肠肝回圈，降低肝内和血清中的胆汁酸水平，减少由此介导的肝脏损伤，缓解胆汁淤积瘙痒（极度瘙痒）。根据与Mirum制药公司的协议，北海康成拥有氯马昔巴特口服溶液在大中华区开发和商业化以及在特定条件下生产的独家授权，用于治疗包括阿拉杰里综合征（ALGS）、进行性家族性肝内胆汁淤积症（PFIC）和胆道闭锁（BA）在内三种罕见肝病适应症以及其他可选择的适应症。据北海康成新闻稿介绍，氯马昔巴特口服溶液已经在已在美国、加拿大、欧洲、中国大陆及其它地区获批用于治疗1岁及以上ALGS患者的胆汁淤积性瘙痒。该药的获批基于Mirum制药公司的关键ICONIC研究和RISE婴儿安全性研究的数据，以及多年对ALGS患者数据的收集。ICONIC研究显示，与安慰剂相比，迈芮倍对于瘙痒症具有统计学及临床意义的减少，血清胆汁酸亦明显减少，两者在数年治疗内均可持久得到改善。RISE研究的中期数据支持小于12个月的婴儿组的适应症。此外，氯马昔巴特口服溶液目前正在进行用于治疗BA的2期全球EMBARK研究，并于2023年5月完成在中国地区的患者招募。北海康成创始人、董事长兼首席执行官薛群博士表示：“迈芮倍在香港获批上市是北海康成取得的又一里程碑。很高兴我们现在能够为香港的医务人员和ALGS患者提供这一变革性疗法。最近ALGS被列入中国《第二批罕见病目录》，凸显了中国患者存在大量未满足的医疗需求。迈芮倍是北海康成重要的管线产品，今年在中国大陆和香港相继获得上市批准。我们期待在2024年启动其商业化进程，同时继续开发迈芮倍的其他适应症，最大限度地发挥对北海康成和胆汁淤积性肝病患者的价值。”参考资料：[1]北海康成宣布治疗阿拉杰里综合征（ALGS）胆汁淤积性瘙痒的迈芮倍®在香港获得批准上市. Retrieved Sep 28， 2023, from https://mp.weixin.qq.com/s/WHiUvGfV9Bfx9ZeGM3YgxA本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈。转发授权请在「医药观澜」微信公众号留言联系我们。其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。

Iconic electronics company to sponsor the training of a service dog to support a deserving war hero NEW YORK, Nov. 29, 2022 /PRNewswire/ -- RCA, the renowned electronics company founded more than a century ago, has announced a charitable partnership with K9s For Warriors, the nation's largest provider of trained service dogs for military veterans. The original RCA Victor brand logo – a charming Jack Russell terrier named Nipper tilting his head as he hears his master's voice from a gramophone – is associated by many with warm, nostalgic feelings for another era. It is with a nod to Nipper that RCA introduces the world to Chips, a sweet yellow Labrador retriever puppy that will be trained and paired with a veteran experiencing service-connected trauma. "From Korea and Vietnam to Iraq and Afghanistan, there are many U.S. veterans whose well-being has been deeply affected from their service to our country in military theaters overseas," said Tony Bozzini, representative of the RCA brand. "As these heroes face challenges, it's important to do what we can to help. Knowing of K9s For Warriors' powerful and impactful work, we recognized the opportunity to partner with them to provide the kind of help that comes on four legs. We are honored to sponsor Chips and his training so that he can serve a veteran who has so bravely served our country." RCA will introduce Chips and share K9s For Warriors's mission, as well as unveil and demonstrate their new consumer electronics at the 2023 CES (Consumer Technology Association) exposition in Las Vegas in January. K9s For Warriors was founded in 2011 by Shari Duval after her son, a civilian K9 police bomb dog handler who served in Iraq, returned home with PTSD. When doctors were unable to help her son mentally and emotionally heal, Duval and her family discovered that a service dog provided the support he desperately needed. Duval began the organization on her own and watched it become the largest provider of service dogs for veterans in the United States. To date, K9s For Warriors has been able to pair a highly trained service dog to 805 veterans suffering from PTSD, traumatic brain injury and/or military sexual trauma. Chips has already started his training at just 16 weeks and will one day go on to save a veteran's life. "We are incredibly grateful for the support of RCA to further our mission to end veteran suicide," said Carl Cricco, CEO of K9s For Warriors. "K9s For Warriors has the privilege every day to see the impact a service dog has on a veteran. With a battle buddy by their side, the veteran returns to a life of dignity and independence. RCA's generosity allows us to continue changing veterans' lives." A top-rated, four star charity on CharityNavigator.org, K9s For Warriors was named one of Good Housekeeping's "50 Best Charities to Give to Right Now" and was the recipient of the Florida Impact Award (Leadership Florida) and the 2020 Platinum Seal of Transparency (GuideStar.) In addition to sponsoring the training of Chips, RCA and K9s For Warriors will share the special journey with followers on social media at K9s for Warriors and RCA. RCA will also donate electronics to the war hero and looks forward to fostering a longtime future relationship with K9s For Warriors. About RCA RCA is one of the most recognized names in the electronics industry, delivering innovative and reliable technology that has been entertaining families for over 100 years. Consumers throughout the world depend on the RCA brand to provide products and services that feature the latest technology and design, are easy to use, and deliver the highest value and longevity. Generations of families continue to rely on the RCA brand for their home entertainment and lifestyle. RCA is a global trademark of Established. For more information about RCA brand, please visit: . About K9s For Warriors Determined to end veteran suicide, K9s For Warriors is the nation's largest provider of trained Service Dogs to military veterans suffering from PTSD, traumatic brain injury and/or military sexual trauma. With most dogs coming from high-kill rescue shelters, this innovative program allows the K9/Warrior team to build an unwavering bond that facilitates their collective healing and recovery. This treatment method is backed by scientific research (from University of Arizona's OHAIRE Lab) demonstrating Service Dogs' ability to help mitigate their veteran's symptoms of PTSD while simultaneously restoring their confidence and independence. Founded in 2011 as a 501 (c)(3) nonprofit organization, K9s For Warriors remains committed to bringing widespread awareness to veterans' mental health and contributing to policy-level reform. The organization's facilities include: K9s For Warriors National Headquarters (Ponte Vedra, FL) Davis Family Mega Kennel (Ponte Vedra, FL) Gold Family Campus (Alachua, FL) Petco Love K9 Center (San Antonio, Texas) Helotes Warrior Ranch (Helotes, TX) and K9s For Warriors at Heidi's Village (Phoenix, AZ). Find more information about K9s For Warriors, please visit: | Facebook | Twitter SOURCE RCA