Scoliosis

Biogen’s Skyclarys (omaveloxolone) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) as the first treatment for patients aged 16 years and older with the rare neurodegenerative movement disorder Friedreich’s ataxia (FA). FA is the most common inherited ataxia, a group of disorders affecting coordination, balance and speech, affecting at least one in every 50,000 people in the UK. Early symptoms of the life-shortening condition, such as progressive loss of coordination and muscle weakness, typically present in childhood and most patients will need to use a wheelchair within ten to 20 years of their first symptoms. Patients may also experience hearing loss, vision impairments, problems with speech and swallowing, scoliosis, diabetes and serious heart conditions as the disease progresses. Taken as an oral capsule, Skyclarys activates the Nrf2 pathway, which helps cells respond to a condition that can lead to cell and tissue damage. The MHRA’s decision on the drug was supported by positive results from the placebo-controlled MOXIe trial, in which Skyclarys-treated patients scored better on tests of neurological function compared to those in the placebo cohort. As previously reported by Biogen, all components of the modified FA Rating Scale (mFARS) assessment, including the ability to swallow, upper limb coordination, lower limb coordination and upright stability, favoured Skyclarys over placebo. Julian Beach, MHRA interim executive director of healthcare quality and access, said: “I am pleased to confirm the first UK approval for the treatment of FA, [Skyclarys]. “We’re assured that the appropriate regulatory standards of safety, quality and effectiveness for the approval of this new treatment have been met. As with all products, we will keep its safety under close review.” Biogen gained access to Skyclarys in September 2023 after completing its $7.3bn acquisition of Reata Pharmaceuticals. The drug had already been approved by the US Food and Drug Administration when the company announced its intended buyout of Reata in July, and gained EU approval in February last year. Although Skyclarys was the main asset included in the acquisition, Biogen also gained access to a portfolio of products that Reata was developing for a range of neurological diseases.

今日（4月22日），阿斯利康（AstraZeneca）宣布瑞利珠单抗注射液（ravulizumab）的上市申请已获得中国国家药监局（NMPA）批准，与常规治疗药物联合用于治疗抗乙酰胆碱受体（AChR）抗体阳性的成人全身型重症肌无力（gMG）患者。瑞利珠单抗是一款长效补体C5蛋白抑制剂。通过每八周一次的给药方式，可改善患者的生活质量，帮助减轻治疗负担。在美国，该产品已经获得FDA批准治疗多种自身免疫性疾病（商品名：Ultomiris），包括阵发性睡眠性血红蛋白尿（PNH）、非典型溶血性尿毒症综合征（aHUS）、重症肌无力、视神经脊髓炎谱系疾病（NMOSD）等。补体系统是先天免疫系统的一部分。当补体系统以不受控制的方式激活时，补体介导的免疫反应攻击患者自身的健康组织和细胞，导致不同组织和器官的损伤，引发多种免疫疾病。补体蛋白C5处于补体级联反应的末端，因此靶向这一蛋白可以调控所有3种不同通路激活的补体信号。在多种补体介导的免疫疾病中，抑制C5的活性可以抑制对自身的免疫攻击，从而缓解疾病症状。瑞利珠单抗是一款长效C5单克隆抗体，可提供即时、完全和持续的补体抑制。研究发现，该产品可通过高亲和力与C5结合来抑制终末补体途径，包括抑制C5分解为C5a（一种促炎和促血栓的过敏毒素）和C5b（终末补体复合物C5b-9的起始亚基，C5b促进细胞溶解）。此外，独特的设计使瑞利珠单抗具有比第一代C5补体抑制剂更长的半衰期。成人患者在初始剂量给药后，后续只需要每8周静脉注射一次。瑞利珠单抗最早于2018年获得美国FDA批准上市，用于治疗阵发性睡眠性血红蛋白尿症（PNH），患者只需要每8周接受一次注射就可以有效控制溶血的发生。此后，该药相继获FDA批准治疗多种自身免疫性疾病，包括非典型溶血性尿毒症综合征、重症肌无力、视神经脊髓炎谱系疾病（NMOSD）等。根据阿斯利康新闻稿介绍，此次NMPA的批准是基于CHAMPION-MG 3期研究的积极结果，该结果已发表于《新英格兰医学杂志-循证医学子刊》(NEJM Evidence)。研究显示，瑞利珠单抗在主要疗效终点即第26周重症肌无力日常生活活动（MG-ADL）评分较基线改善方面优于安慰剂。MG-ADL是一个患者自评量表，用于评估患者进行日常活动能力。此外，经开放标签扩展期研究的长期随访，瑞利珠单抗的临床获益持续至60周。该研究还观察到，与安慰剂相比，经瑞利珠单抗治疗的每百名患者每年的疾病加重发生率减少了71.1%。经美国神经病学学会（AAN）公布，并在《欧洲神经学杂志》发表的CHAMPION-MG 3期试验最终研究结果表明，瑞利珠单抗对重症肌无力（MG）症状‌具有临床意义的改善‌可维持长达164周‌。在中国，根据药物临床试验登记与信息公示平台信息，阿斯利康已经登记开展了多项瑞利珠单抗的国际多中心（含中国）3期临床试验，针对的适应症包括IgA肾病、成人和青少年造血干细胞移植（HSCT）后出现的血栓性微血管病（TMA）的治疗、预防体外心肺循环时慢性肾病患者的严重肾脏不良事件等。此外，研究人员还于2024年7月登记一项中国3期临床研究，旨在中国阵发性睡眠性血红蛋白尿症（PNH）成人受试者中评价瑞利珠单抗的有效性、安全性、药代动力学、药效学和免疫原性。参考资料：[1] 伟立瑞®在华获批用于治疗成人全身型重症肌无力(gMG)患者.From https://mp.weixin.qq.com/s/EZ83jDAe0UnyXmLP7Iw4RQ[2] 药物临床试验登记与信息公示平台.Retrieved Jul 30,2024,from http://www.chinadrugtrials.org.cn/clinicaltrials.searchlist.dhtml[3] New, long-term ALPHA Phase III trial data published in Blood show Voydeya as add-on to Ultomiris or Soliris maintained clinical improvements for the subset of people with PNH experiencing clinically significant extravascular haemolysis. Retrieved Jan 6, 2025, from  https://www.astrazeneca.com/media-centre/medical-releases/new-long-term-alpha-phase-iii-trial-data-published-in-blood-show-voydeya-as-add-on-to-ultomiris-or-soliris-maintained-clinical-improvements-for-the-subset-of-people-with-pnh-experiencing-clinically-significant-extravascular-haemolysis.html本文欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权及其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：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。

WARSAW, Ind., April 08, 2025 (GLOBE NEWSWIRE) -- OrthoPediatrics Corp. (“OrthoPediatrics” or the “Company”) (Nasdaq: KIDS), a company focused exclusively on advancing the field of pediatric orthopedics, today announced the U.S. launch of the new VerteGlide Spinal Growth Guidance System, used to treat Early Onset Scoliosis (“EOS”). This represents the company’s 80th system designed to treat musculoskeletal issues in children. VerteGlide is a guided growth technology designed for children with Early Onset Scoliosis, a severe and sometimes life-threatening condition affecting kids under the age of 10, who often suffer from pulmonary and digestive dysfunction because of the deformity. VerteGlide represents the second in a series of technologies OrthoPediatrics is developing to address the challenges of this special group of children suffering from debilitating and life-threatening consequences of Early Onset Scoliosis. With its unique design, the VerteGlide Spinal Growth Guidance System is designed to allow for deformity correction and minimize repeat surgeries needed until skeletal maturity. With the recent FDA clearance of this device, the Company is preparing for a limited launch and expects to be doing initial surgeries with select children's centers during summer of this year. Dr. Richard McCarthy, a pediatric orthopedic spine surgeon in Louisville, Kentucky is a pioneer in the field of growth guidance in scoliosis surgery; he and Dr. Scott Luhmann of St. Louis have been instrumental in partnering with the Company on the design and development of the system. McCarthy shared that "VerteGlide marks a breakthrough in technology for children." OrthoPediatrics Scoliosis division President, Greg Odle commented “We are excited to introduce another new product to treat children with EOS. The team has been working with key opinion leaders from the surgeon community to identify new technologies that will help this particularly vulnerable patient population. VerteGlide’s launch represents our commitment to treating the entire disease state of scoliosis.” CEO, David Bailey echoed those comments, saying “I’m incredibly proud of the Engineering team and the work the business teams have done to bring this product to market. It feels like we are making meaningful progress in providing healthcare professionals innovative treatment options for the patients they serve.” The VerteGlide Spinal Growth Guidance System is made possible with the Shilla™ technology licensed from Medtronic. About OrthoPediatrics Corp.Founded in 2006, OrthoPediatrics is an orthopedic company focused exclusively on advancing the field of pediatric orthopedics. As such it has developed the most comprehensive product offering to the pediatric orthopedic market to improve the lives of children with orthopedic conditions. OrthoPediatrics currently markets 80 products that serve three of the largest categories within the pediatric orthopedic market. This product offering spans trauma and deformity, scoliosis, and sports medicine/other procedures. OrthoPediatrics’ global sales organization is focused exclusively on pediatric orthopedics and distributes its products in the United States and over 70 countries outside the United States. For more information, please visit www.orthopediatrics.com. Investor ContactPhilip Trip TaylorGilmartin Groupphilip@gilmartinir.com415-937-5406