Burosumab-TWZA

XLH can lead to rickets, a condition that results in weak or soft bones. Credit: CGN089 via Shutterstock. The UK’s National Health Service (NHS) is rolling out Kyowa Kirin ’s Crysvita (burosumab) for the treatment of adults with a rare bone disease called X-linked hypophosphataemia (XLH). The drug has been available for children with XLH since 2018 but will now be available for eligible adults on the NHS. The service will initially roll the drug out through around 20 specialist centres across England. Additionally, patients will be taught to give Crysvita in their own homes to reduce “unnecessary visits to hospitals”. XLH is a genetic disorder that leads to low phosphate levels in the blood. Phosphate is essential for the normal formation of bones and teeth in childhood and helps maintain bone strength in adults. Deficiency of the mineral can result in rickets, an increased risk of fractures, muscle weakness, and dental problems such as abscesses and tooth loss. Patients often experience chronic pain and mobility issues in the most severe cases. This move follows the UK’s National Institute for Health and Care Excellence (NICE) recommendation in August, where the pricing watchdog said that the treatment is a clinically and cost-effective use of NHS resources. This wasn’t always the case, as NICE previously rejected the drug for adults on the NHS in draft guidance released in December 2023. In the guidance, NICE commented on pivotal clinical trial data, saying that patients on “burosumab may have less pain and fatigue, and improved physical functioning” but added it was “not certain”. This deliberation came from the results of the Phase III CL303 trial (NCT02526160), which compared Crysvita to a placebo for 24 weeks. After this period, people on placebo switched to Crysvita, where treatment was continued for another 72 weeks. The trial met its primary outcome – with 94.1% of the treatment arm meeting a mean serum phosphate concentration above the lower limit of normal (2.5mg/dL), compared to 7.6% in the placebo arm. In the announcement accompanying the roll-out, Oliver Gardiner, trustee at charity XLH UK, said: “This is fantastic news for adults with XLH in the UK. This life-changing treatment is the first to tackle the underlying problem and will lessen the significant physical and emotional challenges that adults with XLH face on a daily basis.” The standard of care for patients with XLH was phosphate supplements or vitamin D, which doesn’t target the cause of the disease and can cause side effects such as stomach problems and kidney stones. Crysvita works by suppressing the hormone that causes low levels of phosphate and stabilising levels in the blood. According to GlobalData’s Pharma Intelligence Center, Crysvita will generate $2.4bn in revenue in 2030. GlobalData is the parent company of Pharmaceutical Technology.

2024年9月30日，香港维健医药集团（以下简称“维健医药”）正式宣布，已成功完成对日本协和麒麟（以下简称“协和麒麟”）株式会社全资子公司——协和麒麟（中国）制药有限公司（以下简称“协和麒麟中国”）股权收购的交割。 On September 30, 2024, Hong Kong Winhealth Pharma Group (hereinafter referred to as "Winhealth Pharma") officially announced the successful completion of the equity acquisition of Kyowa Kirin (China) Pharmaceutical Co., Ltd. (hereinafter referred to as "Kyowa Kirin China"), a wholly-owned subsidiary of Kyowa Hakko Kirin Co., Ltd. (hereinafter referred to as "Kyowa Kirin"). 交割之后，维健医药将持有协和麒麟中国100%股权，并获得协和麒麟在中国上市商业化的五个原研品牌药品的永久商业化及生产权益以及未来在肾科和血液科管线的优先商业化权益。此外，维健医药还与协和麒麟签订了全球重磅产品麟平®和惠尔金®的商业化许可协议，获得其在中国的长期商业化权益。 Following the closing, Winhealth Pharma will hold 100% equity of Kyowa Kirin China and obtain the permanent commercialization and production rights of five original brand drugs commercialized in China by Kyowa Kirin, as well as the priority commercialization rights of future pipeline products in the nephrology and hematology fields. Additionally, Winhealth Pharma has also signed a commercial license agreement with Kyowa Kirin for the global blockbuster products Crysvita® and Poteligeo®, securing their long-term commercialization rights in China. 此次交易，自2024年8月1日宣布签约以来，历时两个月。在此期间内，项目得到了各方的高度重视与支持，双方团队能够在短短61天的时间里，完成项目所涉交割程序和附属协议的签署，使得项目高效、顺利、平稳的交割，得益于双方高度专业化的项目团队，以及内、外部团队、合作伙伴多方的积极努力和高效协同。 This transaction has spanned two months since the signing was announced on August 1, 2024. During this period, the project has received high-level attention and support from all parties. The project teams of both sides were able to complete the closing procedures and the signing of ancillary agreements involved in the project in just 61 days. The efficient, smooth, and stable closing of the project is attributable to the highly professional project teams of both sides and the active efforts and efficient collaboration of internal and external teams and multiple partners. 维健医药董事长/创始人兼CEO王威先生表示： Mr. Wang Wei, Chairman/Founder and CEO of Winhealth Pharma, said: “我们对股权收购的成功完成感到非常高兴，它将显著增强我们在创新药物研发和商业化领域的综合竞争力；意味着维健医药从一家医药商业化推广企业成为一家大型创新型生物制药企业的战略转型的顺利完成；更是维健医药真正迈向维健2.0时代的重大里程碑事件。未来，我们将始终秉持‘患者为先’的初心，致力于为全球患者提供更多更好的治疗方案。此交割的圆满落幕，维健医药将会继承和发扬协和麒麟中国的业务、持续扩大中国市场，满足更多未被满足的患者需求。” “We are extremely delighted with the successful completion of the equity acquisition. It will significantly enhance our comprehensive competitiveness in the fields of innovative drug research and development and commercialization. It means the smooth completion of Winhealth Pharma's strategic transformation from a pharmaceutical commercial promotion enterprise to a large innovative biopharmaceutical enterprise. It is also a significant milestone event as Winhealth Pharma truly steps into the Winhealth 2.0 era. In the future, we will always adhere to the original intention of ‘patient comes first’ and be committed to providing more and better treatment options for patients around the world. With the successful conclusion of this transaction, Winhealth Pharma will inherit and carry forward the business of Kyowa Kirin China, continuously expand the Chinese market, and meet more unmet patient needs.” 维健医药集团执行董事、联席CEO郝景辉先生表示： Mr. Hao Jinghui, Executive Director and Co-CEO of Winhealth Pharma, remarked,  “从8月1日签约到今天的高效交割，历时不到两个月，这一成就离不开双方管理层和交易团队的专业互信、一丝不苟与精诚合作。作为长期战略合作伙伴，维健医药与协和麒麟始终保持着紧密且良好的合作关系，共同构建了基于互惠互信共赢原则的共生关系。今天的成功交割，标志着双方合作迈入了全新的发展阶段。未来，维健医药将持续秉承对患者、合作伙伴和员工的承诺，携手协和麒麟及众多全球优秀战略合作伙伴，将更多领先的原研创新药品带给中国患者，共创更加辉煌的未来。” "From the signing on August 1 to today's efficient closing, it took less than two months. This achievement is inseparable from the professional mutual trust, meticulousness, and sincere cooperation of the management and transaction teams of both sides. As a long-term strategic partner, Winhealth Pharma and Kyowa Kirin have always maintained a close and excellent cooperative relationship and jointly established a symbiotic relationship based on the principles of mutual benefit, mutual trust, and win-win. Today's successful closing signals that the cooperation between the two sides has entered a new stage of development. In the future, Winhealth Pharma will continue to uphold its commitments to patients, partners, and employees. Together with Kyowa Kirin and many outstanding global strategic partners, we will bring more leading original innovative drugs to Chinese patients and create a more glorious future." 随着本次交割的圆满落幕，维健医药与协和麒麟的合作正式迈入了全新的发展阶段。这不仅是双方合作历程中的一个重要里程碑，更是标志着双方在战略层面的深度融合。维健医药将以此为契机，全面而深入地整合协和麒麟中国在研发、生产及商业化领域的丰富资源。通过资源的优化配置与共享，维健医药将构建起更为专业、完善和高效的全链路商业化体系，夯实公司的竞争优势，为公司的持续发展和转型注入了强大的发展动力。 With the successful closing of this transaction, the cooperation between Winhealth Pharma and Kyowa Kirin has officially entered a new stage of development. This is not only an important milestone in the cooperation process between the two sides but also indicates the deep integration of the two sides at the strategic level. Winhealth Pharma will seize this opportunity to comprehensively and deeply integrate the abundant resources of Kyowa Kirin China in the fields of research and development, production, and commercialization. Through the optimized allocation and sharing of resources, Winhealth Pharma will establish a more professional, complete, and efficient full-link commercialization system, consolidate the company's competitive advantages, and inject strong impetus for the company's continuous development and transformation. 展望未来，维健医药将依托这些优势，不断追求卓越。公司将始终以患者为中心，致力于满足患者日益增长的健康需求，持续扩展我们的自有和合作产品管线。通过不断的创新与突破，维健医药将和我们的优秀产业链伙伴一道共同开创医药产业的新篇章，为健康事业贡献更多的智慧与力量。 Looking ahead, Winhealth Pharma will rely on these advantages and continuously pursue excellence. The company will always center on patients, be committed to meeting the growing health needs of patients, and continuously expand our own and cooperative product pipelines. Through continuous innovation and breakthroughs, Winhealth Pharma will work together with our excellent industrial chain partners to jointly create a new chapter in the pharmaceutical industry and contribute more wisdom and strength to the health cause. 关于香港维健医药集团 About Hong Kong Winhealth Pharma Group 香港维健医药集团是一家总部位于香港的领先生物制药公司，在大中华地区开展商业运营，并在亚洲和欧洲区域不断扩展业务。公司专注于创新药物的商业化，致力于为心肾疾病、血液肿瘤、胃肠疾病、呼吸和抗感染疾病和罕见病以及其他未满足和严重未被满足的医疗需求患者提供突破性疗法。 Hong Kong Winhealth Pharma Group is a leading biopharmaceutical company based in Hong Kong, with commercial operations across the Greater China Area and an expanding regional footprint in Asia and Europe. Specializing in the commercialization of innovative medicines, the company is dedicated to delivering breakthrough therapies for patients with cardio-renal, gastrointestinal, respiratory and anti-infectious and rare diseases, as well as other unmet and severely underserved medical needs. 维健医药与众多世界领先的制药和生物技术公司建立了成功的长期合作伙伴关系，创造了一个独特而有意义的多元化产品组合，包括15种以上的专科产品，覆盖商业和后期临床阶段。维健医药始终以患者为中心，坚守对合作伙伴的承诺，不断扩展其产品组合，寻求通过其全球合作伙伴网络引进更多的变革性疗法。 WinHealth has established successful long-term partnerships with numerous world-leading pharmaceutical and biotechnology companies, creating a uniquely impactful and diversified portfolio that includes over 15 specialty products in both commercial and late clinical stages. Committed to patients and partners, WinHealth continuously expands its portfolio and seeks to bring more transformative therapies from its global network of partners. 欲了解更多信息，请访问www.winhealth.hk。 For more information, please visit www.winhealth.hk. 关于协和麒麟 About Kyowa Kirin 协和麒麟致力于研发具有改变生命价值的创新型药物。作为一家总部位于日本的全球化特药公司，我们在药物研发和生物技术创新等方面持续投入超过70年，目前，我们正致力于研发先进的抗体技术、细胞和基因疗法，以期帮助患有严重疾病或罕见疾病的患者。我们在全球四个地区——日本、亚太、北美和EMEA（欧洲、中东和非洲），因共同的价值观，对可持续发展的承诺以及致力于为人们带来笑容的使命而紧密相联。如需了解更多信息，请访问www.kyowakirin.com。 Kyowa Kirin aims to discover novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients affected by a severe or rare disease. A shared commitment to our values, to sustainable growth, and to making people smile unites us across our four regions – Japan, Asia Pacific, North America, and EMEA/International. You can learn more about the business of Kyowa Kirin at www.kyowakirin.com.