The objective of this post-marketing surveillance (PMS) study is to assess the safety and effectiveness of CRYSVITA injection 10, 20, and 30mg, equivalent to in routine clinical settings

开始日期2023-08-11

申办/合作机构

Kyowa Kirin Korea Co., Ltd.

NCT05509595 / Active, not recruiting临床2期IIT

A Phase 2 Study of Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia

Background:
Fibrous dysplasia (FD) is a disorder that affects bone growth. Affected bone tissue is weakened, and people with FD are prone to deformities, fractures, and other problems. People with FD may also have low blood phosphate levels. This can make bones even weaker. Better treatments are needed.
Objective:
To test a study drug (burosumab) in people with FD who have low blood phosphate levels.
Eligibility:
People aged 1 year or older who have FD and low blood phosphate levels.
Design:
Participants will visit the NIH 3 times in 48 weeks. Each visit will last 5 to 7 days.
Participants will self-inject burosumab under the skin in their belly, upper arm, or thigh. They (or a caregiver) will do this at home 1 or 2 times a month. They will be trained in person on how to inject the drug. Home injections will be guided via telehealth.
During NIH visits, participants will have a physical exam with blood and urine tests. They will have x-rays of different parts of their body. They will have a radioactive tracer injected into their vein; then they will have a bone scan. They will have tests to assess their strength, walking, and movement. They will complete questionnaires about their pain, mobility, and fatigue levels.
Adult participants may have bone biopsies. These will be done under anesthesia with sedation. Small samples of FD-affected bone will be removed for study.
Between NIH visits, participants will go to a local laboratory for blood and urine tests.
Child participants will have an additional follow-up visit 2 weeks after the final NIH visit.

开始日期2022-12-07

申办/合作机构

National Institute of Dental & Craniofacial Research

NCT05357573 / Completed临床4期

An Open-Label, Multi Center, Single-Cohort, Post-Marketing Phase 4 Study to Evaluate the Efficacy, Pharmacodynamics, and Safety of the Anti-FGF23 Antibody, KRN23, in Adult Chinese Patients With Tumor-Induced Osteomalacia (TIO)

The purpose of this study is to assess the safety, pharmacokinetics and efficacy of KRN23 in adult Chinese patients with TIO

开始日期2022-09-07

申办/合作机构

Kyowa Kirin Co., Ltd.

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项与布罗索尤单抗相关的文献（医药）

2024-11-01·Current pediatric reviews

Diagnostic and New Therapeutic Approaches to Two Challenging Pediatric Metabolic Bone Disorders: Hypophosphatasia and X-linked Hypophosphatemic Rickets

Review

作者: Aljuraibah, Fahad ; Alalwan, Ibrahim ; Habeb, Abdelhadi

Abstract::

The diagnosis and management of metabolic bone disease among children can be challenging. This difficulty could be due to many factors, including limited awareness of these rare conditions, the complex pathophysiology of calcium and phosphate homeostasis, the overlapping phenotype with more common disorders (such as rickets), and the lack of specific treatments for these rare disorders. As a result, affected individuals could experience delayed diagnosis or misdiagnosis, leading to improper management. In this review, we describe the challenges facing diagnostic and therapeutic approaches to two metabolic bone disorders (MBD) among children: hypophosphatasia (HPP) and X-linked hypophosphatemia (XLH). We focus on explaining the pathophysiological processes that conceptually underpin novel therapeutic approaches, as well as these conditions’ clinical or radiological similarity to nutritional rickets. Particularly in areas with limited sun exposure and among patients not supplementing vitamin D, nutritional rickets are still more common than HPP and XLH, and pediatricians and primary physicians frequently encounter this disorder in their practices. More recently, our understanding of these disorders has significantly improved, leading to the development of novel therapies. Asfotas alfa, a recombinant, human- tissue, nonspecific alkaline phosphatase, improved the survival of patients with HPP. Burosumab, a human monoclonal anti-FGF23 antibody, was recently approved as a specific therapy for XLH. We also highlight the current evidence on these two specific therapies’ safety and effectiveness, though long-term data are still needed. Both HPP and XLH are multisystemic disorders that should be managed by multidisciplinary teams. Finally, recognizing these conditions in early stages will enable affected children and young adults to benefit from newly introduced, specific therapies.

2024-08-01·European Journal of Medical Genetics

Safety and efficacy of burosumab in improving phosphate metabolism, bone health, and quality of life in adolescents with X-linked hypophosphatemic rickets

Article

作者: Comberiati, Pasquale ; Aversa, Antonio ; Sessa, Maria Rita ; Michelucci, Angela ; Baroncelli, Giampiero I ; Pelosini, Caterina ; Toschi, Benedetta ; Grandone, Anna ; Isola, Alessandro ; Manca, Mario

BACKGROUND AND OBJECTIVE:

X-linked hypophosphatemic rickets (XLH) is due to loss-of-function mutations in the phosphate-regulating endopeptidase homologue on the X chromosome (PHEX) that lead to increased fibroblast growth factor 23 (FGF23) production. FGF23 excess causes renal phosphate wasting and insufficient 1,25-dihydroxyvitamin D (1,25(OH)₂D) synthesis with reduced intestinal phosphate absorption, ultimately resulting in chronic hypophosphatemia. Children with XLH show typical skeletal lesions of rickets, deformities of the lower limbs, stunted growth with disproportionate short stature, bone pain, and physical dysfunctions. Burosumab, a fully human IgG1 monoclonal antibody that binds to FGF23 to inhibit its activity, is more effective to improve the biochemical and clinical signs of XLH than conventional treatment with phosphate supplements and vitamin D active metabolites. Data on adolescents with XLH during the transition period to young adulthood are few. In this prospective case series, we aimed to assess safety and efficacy of burosumab in adolescents with XLH who discontinued long-term conventional therapy.

METHODS:

Five Caucasian adolescents (4 males, 1 female; mean age 15.4 ± 1.5 years) with XLH were recruited and switched from conventional treatment to burosumab (0.8-1.2 mg/kg, s. c. QW2). Burosumab was continued for 12-48 months and, once discontinued, patients were followed-up for 6-12 months. In all patients, serum calcium, phosphate, alkaline phosphatase (ALP), parathyroid hormone (PTH), and 1,25(OH)₂D levels, and renal tubular reabsorption of phosphate (TmP/GFR) values were assessed at entry and during burosumab. Intact FGF23 plasma levels were measured at entry. Patient-reported outcomes (PROs) were assessed at entry and every 3-6 months to evaluate the impact of low extremity pain, stiffness, and difficulties performing daily activities.

RESULTS:

At entry, all patients showed hypophosphatemia, increased intact FGF23 levels, reduced TmP/GFR, insufficient 1,25(OH)₂D levels, and in four out of five increased ALP levels. Two patients had radiological signs of rickets. During burosumab, all patients showed a significant increase in serum phosphate and 1,25(OH)₂D levels, and in TmP/GFR values (P < 0.05 - P < 0.0001). Serum ALP levels significantly declined (P < 0.05) to normal values. No changes of serum calcium and PTH levels (PNS) were found during burosumab. PROs significantly improved (P < 0.02 - P < 0.0001) in all patients. Four patients discontinued burosumab when they turned 18 or 19, whereas one continued the treatment since he was still younger than 18 during the study period. Four patients who suspended burosumab showed a rapid decline in serum phosphate and 1,25(OH)₂D levels and in TmP/GFR values; serum ALP levels increased, and PROs progressively worsened with a significant reduction in quality of life. These consequences were not observed in the patient who continued burosumab treatment.

DISCUSSION:

Our data showed that conventional treatment improved only in part the signs and symptoms of XLH. Burosumab was well tolerated and was effective in improving phosphate metabolism, bone health, and PROs. All the benefits of burosumab were lost after its discontinuation. These results suggested that continuing burosumab is required to achieve and maintain the clinical benefits of the treatment during the transition to young adulthood in patients with XLH.

2024-07-01·ENDOCRINOLOGY

NFATc1 Is Required for Vitamin D- and Phosphate-Mediated Regulation of Osteocyte Lacuno-Canalicular Remodeling

Article

作者: Liu, Eva S ; Divieti Pajevic, Paola ; Manoochehri Arash, Niusha ; Brooks, Daniel J ; Sorsby, Melissa ; Hughes, Ashleigh ; Jagga, Supriya

Abstract:

Osteocytes are embedded in lacunae and connected by canaliculi (lacuno-canalicular network, LCN). Bones from mice with X-linked hypophosphatemia (Hyp), which have impaired production of 1,25 dihydroxyvitamin D (1,25D) and hypophosphatemia, have abnormal LCN structure that is improved by treatment with 1,25D or an anti-FGF23 targeting antibody, supporting roles for 1,25D and phosphate in regulating LCN remodeling. Bones from mice lacking the vitamin D receptor (VDR) in osteocytes (Vdrf/f;Dmp1Cre+) and mice lacking the sodium phosphate transporter 2a (Npt2aKO), which have low serum phosphate with high serum 1,25D, have impaired LCN organization, demonstrating that osteocyte-specific actions of 1,25D and hypophosphatemia regulate LCN remodeling. In osteoclasts, nuclear factor of activated T cells cytoplasmic 1 (NFATc1) is critical for stimulating bone resorption. Since osteocytes also resorb matrix, we hypothesize that NFATc1 plays a role in 1,25D and phosphate-mediated LCN remodeling. Consistent with this, 1,25D and phosphate suppress Nfatc1 mRNA expression in IDG-SW3 osteocytes, and knockdown of Nfatc1 expression in IDG-SW3 cells blocks 1,25D- and phosphate-mediated suppression of matrix resorption gene expression and 1,25D- and phosphate-mediated suppression of RANKL-induced acidification of the osteocyte microenvironment. To determine the role of NFATc1 in 1,25D- and phosphate-mediated LCN remodeling in vivo, histomorphometric analyses of tibiae from mice lacking osteocyte-specific Nfatc1 in Vdrf/f;Dmp1Cre+ and Npt2aKO mice were performed, demonstrating that bones from these mice have decreased lacunar size and expression of matrix resorption genes, and improved canalicular structure compared to Vdrf/f;Dmp1Cre+ and Npt2aKO control. This study demonstrates that NFATc1 is necessary for 1,25D- and phosphate-mediated regulation of LCN remodeling.

项与布罗索尤单抗相关的新闻（医药）

2024-09-19

·PRNewswire

Kyowa Kirin to Present New Research Spotlighting Global Efforts to Advance Science and Patient Care in X-linked Hypophosphatemia at ASBMR Annual Meeting

One oral presentation and nine posters will shed light on real-world experiences of people living with XLH and the impact of burosumab treatment TOKYO, Sept. 19, 2024 /PRNewswire/ -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE:4151) will share new research that enhances understanding about the use of burosumab in real-world clinical practice as well as the burden of those living with X-linked hypophosphatemia (XLH), a rare genetic metabolic bone disease. The findings will be presented at the American Society for Bone and Mineral Research (ASBMR) 2024 annual meeting, which takes place September 27-30 in Toronto. The oral presentation of "Patient reported outcomes (PRO) from a real-world study of burosumab treatment in adults with X-linked hypophosphatemia in the UK" will be made by Judith Bubbear, MD, Royal National Orthopaedic Hospital (UK) on Saturday, September 28, 11:30 am EDT (Presentation #1022). "As we learn more about XLH and how it impacts people's lives, real-world evidence can provide much-needed insight into potential strategies for managing this debilitating disease," said Angela Williams, PhD, Vice President and Global Head of Health Economics and Outcomes Research at Kyowa Kirin. "The breadth of our ASBMR data reflects our continued focus on advancing the science and understanding of XLH and improving the lives of people living with XLH." ASBMR XLH poster presentations: Real-world impact of burosumab treatment in people living with XLH: "Effectiveness of burosumab versus conventional therapy in adults with X-linked hypophosphatemia in a real-world setting from the XLH Disease Monitoring Program" (Poster#SAT-430) Lead author: Pablo Florenzano, MD, Pontificia Universidad Católica de Chile Poster Session I: Saturday, September 28, 2:15 – 3:45 pm EDT "Biochemical measurements according to age group in burosumab-treated patients with X-linked hypophosphatemia (XLH) in a real-world setting: an analysis of the XLH Disease Monitoring Program" (Poster #Fri-424, #Sun-424) Lead author: Leanne M. Ward, MD, Children's Hospital of Eastern Ontario Welcome Reception/Plenary Poster Session: Friday, Sept. 27, 5:30 – 7:30 pm EDT Poster Session II; Sunday, Sept. 29, 2:15 – 3:45 pm EDT "Symptoms experienced by adolescents living with X-linked hypophosphatemia at the end of skeletal growth (EOSG) treated with burosumab—a mixed methods analysis" (Poster #SAT-445) Lead author: Vrinda Saraff, MD, Birmingham Women's and Children's Hospital (UK) Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT "Efficacy and safety of burosumab in adult Chinese patients with X-linked hypophosphatemic rickets/osteomalacia (XLH): an open-label, multi-center, single-cohort, post-marketing phase IV study" (Poster#SUN-119) Lead author: Wei Liu, MD, Peking Union Medical College Hospital Poster Session II: Sunday, Sept. 29, 2:15 – 3:45 pm EDT "Efficacy and safety of burosumab in pediatric Chinese patients with X-linked hypophosphatemic rickets/osteomalacia (XLH): an open-label, multi-center, single-cohort, post-marketing phase IV study" (Poster#SAT-121) Lead author: Xiaoping Luo, MD, Tongji Medical College, Huazhong University of Science and Technology Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT XLH burden on multiple aspects of patients' lives: "Association between pain medication use and patient-reported outcomes in adults with X-linked hypophosphatemia: an exploratory analysis of a phase 3 study" (Poster #Sat-025) Lead author: Angela Williams, PhD, Kyowa Kirin International Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT "Individuals with hereditary hypophosphatemia are not prone to early death despite significant earlier development of co-morbidities: a retrospective Danish register study" (Poster #Sun-024) Lead author: Signe Sparre Beck-Nielsen, MD, PhD, Aarhus University Hospital (Denmark) Poster Session II: Sunday, Sept. 29, 2:15 – 3:45 pm EDT "X-linked Hypophosphatemia Community Impact Survey: psychosocial health, symptoms, and self-care" (Poster #SAT-LB 543) Lead author: Jill Simmons, MD, Vanderbilt University Medical Center Late Breaking Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT Bridging evidence gaps to improve clinical decision-making in XLH: "Advancing Patient Evidence in XLH (APEX): rational and design of real-world XLH global data unification program" (Poster #SAT-427) Lead author: Maria Luisa Brandi, MD, PhD, FIRMO Foundation, Florence, Italy and the University Vita-Salute San Raffaele, Milan, Italy Poster Session I: Saturday, Sep 28, 2:15-3:45 pm EDT About X-linked hypophosphatemia X-linked hypophosphatemia is a rare, lifelong, genetic disease that can impact the bones and muscles in both children and adults. In individuals with XLH, the body doesn't hold on to enough phosphorus, which is an essential mineral for bone health. This is due to the production of excess fibroblast growth factor 23 (FGF23), causing the body to release too much phosphorus through the urine. When phosphorus levels are too low (hypophosphatemia), it can cause the softening and weakening of growing bones in children (rickets) and of mature bones in adults (osteomalacia). In children, XLH typically appears as bowed legs or knock knees. Over time, bone weakening can also lead to bone abnormalities in the legs, delayed growth, and short stature. In adults, XLH may cause osteomalacia, fractures and pseudo-fractures, and hypophosphatemia. About CRYSVITA® (burosumab-twza) Injection CRYSVITA is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, which binds to and inhibits the biological activity of FGF23, the underlying cause of hypophosphatemia in XLH. By blocking FGF23, CRYSVITA helps to restore phosphorus reabsorption in the kidneys and increase the production of active vitamin D, which enhances intestinal absorption of phosphate and calcium. U.S. CRYSVITA Indication CRYSVITA is a fibroblast growth factor (FGF23) blocking antibody indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older. Important Safety Information CONTRAINDICATIONS CRYSVITA is contraindicated: In concomitant use with oral phosphate and/or active vitamin D analogs (e.g., calcitriol, paricalcitol, doxercalciferol, calcifediol) due to the risk of hyperphosphatemia. When serum phosphorus is within or above the normal range for age. In patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism. WARNINGS AND PRECAUTIONS Hypersensitivity Hypersensitivity reactions (e.g., rash, urticaria) have been reported in patients with CRYSVITA. Discontinue CRYSVITA if serious hypersensitivity reactions occur and initiate appropriate medical treatment. Hyperphosphatemia and Risk of Nephrocalcinosis Increases in serum phosphorus to above the upper limit of normal may be associated with an increased risk of nephrocalcinosis. For patients already taking CRYSVITA, dose interruption and/or dose reduction may be required based on a patient's serum phosphorus levels. Injection Site Reactions Administration of CRYSVITA may result in local injection site reactions. Discontinue CRYSVITA if severe injection site reactions occur and administer appropriate medical treatment. ADVERSE REACTIONS Pediatric Patients Adverse reactions reported in 10% or more of CRYSVITA-treated pediatric XLH patients across three studies are: pyrexia (55%, 44%, and 62%), injection site reaction (52%, 67%, and 23%), cough (52%), vomiting (41%, 48%, and 46%), pain in extremity (38%, 46%, and 23%), headache (34% and 73%), tooth abscess (34%, 15%, and 23%), dental caries (31%), diarrhea (24%), vitamin D decreased (24%, 37%, and 15%), toothache (23% and 15%), constipation (17%), myalgia (17%), rash (14% and 27%), dizziness (15%), and nausea (10%). Postmarketing experience reported in CRYSVITA-treated pediatric XLH patients: blood phosphorus increased. Adult Patients Adverse reactions reported in more than 5% of CRYSVITA-treated adult XLH patients and in at least 2 patients more than placebo in one study are: back pain (15%), headache (13%), tooth infection (13%), restless legs syndrome (12%), vitamin D decreased (12%), dizziness (10%), constipation (9%), muscle spasms (7%), and blood phosphorus increased (6%). Spinal stenosis is prevalent in adults with XLH, and spinal cord compression has been reported. It is unknown if CRYSVITA therapy exacerbates spinal stenosis or spinal cord compression. USE IN SPECIFIC POPULATIONS There are no available data on CRYSVITA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-844-768-3544. There is no information regarding the presence of CRYSVITA in human milk or the effects of CRYSVITA on milk production or the breastfed infant. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for CRYSVITA and any potential adverse effects on the breastfed infant from CRYSVITA or from the underlying maternal condition. PATIENT COUNSELING INFORMATION Advise patients not to use any oral phosphate and/or active vitamin D analog products. Instruct patients to contact their physician if hypersensitivity reactions, injection site reactions, and restless legs syndrome induction or worsening of symptoms occur. You may report side effects to the FDA at (800) FDA-1088 or . You may also report side effects to Kyowa Kirin, Inc. at 1-844-768-3544. For important risk and use information, please see the full Prescribing Information for CRYSVITA. About Kyowa Kirin Kyowa Kirin aims to discover and deliver novel medicines and treatments 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 with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato oncology, and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites us across the globe. Learn more about Kyowa Kirin at: kyowakirin.com. COR-US-CRY-0049 September 2024 SOURCE Kyowa Kirin WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床3期临床结果

2024-09-10

·Business Wire

New Analysis Demonstrates Impaired Work Productivity and Increased Unemployment Rates in Adults with X-linked Hypophosphataemia (XLH)

GALASHIELS, Scotland & MARLOW, England--( BUSINESS WIRE )--Kyowa Kirin International (KKI), a wholly owned subsidiary of Kyowa Kirin Co., Ltd. (TSE:4151, Kyowa Kirin) and a Japan-based global specialty pharmaceutical company, announced today the publication of a new analysis showing low levels of full-time employment and impaired work productivity among working age adults enrolled in the X-linked hypophosphataemia (XLH) Disease Monitoring Program (DMP), a prospective, 10-year, observational study of adults and children with XLH in the US, Canada, and Latin America. 1 XLH is a rare, genetic, progressive, phosphate-wasting disorder that can cause skeletal abnormalities, stiffness, pain, and impaired physical function. 2 This analysis of baseline data from the XLH DMP (n=281) showed that 31% of working age adults with XLH were not employed—a rate eight times higher than in the US general population and five times higher than in the EU— and 15% were receiving disability payments. 1,3,4 Additionally, individuals with a higher number of past orthopaedic surgeries, and those with worse physical function, were less likely to be employed. Among those employed (n=193), the majority (60%) were working in light or sedentary roles and those in heavier work roles reported worse pain on average. 1 “These findings highlight the substantial burden of XLH, which impacts multiple aspects of affected individuals' lives. Exploring real-world experiences helps to expand understanding of the disease beyond clinical endpoints to outcomes that are meaningful to patients,” said lead author, Professor Aliya Khan, MD, McMaster University, Canada. “Here, we learn that adults with XLH experienced challenges in the workplace. Understanding the factors underpinning this is an important consideration when planning patient care.” A disease monitoring programme provides an alternative to a traditional registry and extensive post-marketing studies and uses a collaborative, multi-stakeholder approach to monitor disease manifestations over an extended period, without limiting participants based on treatment received. “Kyowa Kirin is committed to generating real-world evidence that builds greater understanding of the XLH patient experience and the effectiveness of treatment on outcomes relevant to patients and clinicians, in both the short- and long-term,” said Ben Johnson, study author and Director of Global Health Economics and Outcomes Research at Kyowa Kirin. “We look forward to sharing further findings from the XLH DMP and other real-world studies at the American Society for Bone and Mineral Research annual meeting in September.” About the XLH Disease Monitoring Program (DMP) 1 The XLH DMP is an international, prospective, 10-year, longitudinal, observational study of adults and children with XLH (NCT03651505) designed to characterise XLH disease presentation and progression, and prospectively assess changes over time in biochemical, clinical, and patient/caregiver-reported outcomes in a representative population. The DMP steering committee includes clinical experts in XLH, patient advocates from the XLH Network, and representatives from Kyowa Kirin and Ultragenyx Pharmaceutical. This study was designed to investigate the association of patient characteristics and work productivity; causality was not assessed and should not be inferred based on these findings. The study population was predominantly from the US (80.8%, with the remaining 19.2% from Brazil, Canada, and Chile), which may have different employment profiles from other countries, limiting the generalisability of the results outside the US. About X-linked hypophosphataemia (XLH) XLH is caused by a genetic mutation which leads to overexpression of the protein FGF23, a protein involved in the regulation of phosphate concentration in the blood. In XLH, FGF23 is produced in excess leading to depletion of phosphate in the blood, known as hypophosphataemia. 2 Individuals living with the disease may display a multitude of symptoms including short stature, limb deformities, bone and joint pain, oral abscesses, and hearing loss. 5 To manage this wide variety of symptoms, the disease is managed through multi-disciplinary teams. 6 About Kyowa Kirin 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 severe and rare diseases. 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: https://www.kyowakirin.com References 1 Khan A, et al. Association between work productivity and characteristics of adults with X-linked hypophosphatemia: an analysis of the XLH disease monitoring program. JBMR Plus. 2024:ziae102. 2 Beck-Nielsen SS, et al. 2019. FGF23 and its role in X-linked hypophosphatemia-related morbidity. Orphanet Journal of Rare Diseases. 2022;14:1-25. 3 US Bureau of Labor Statistics. Labor Force Statistics from the Current Population Survey. 2022. 4 Unemployment rates, Euro Area Unemployment Rate. June 2024 5 Kubota T. X-linked hypophosphatemia transition and team management. Endocrines. 2022;3(3):411-418. 6 European Medicines C. Summary of product characteristics, Crysvita 10 mg solution for injection. 2023. Available here . [Last accessed September 2024].

临床结果临床研究

2024-08-08

·上海市生物医药科技发展中心

7.2亿，一家跨国药企出售中国子公司给中国药企

近日，协和麒麟（Kyowa Kirin）宣布与香港维健医药集团有限公司（以下简称“维健医药”）达成协议，将中国子公司协和麒麟（中国）制药有限公司（以下简称“协和麒麟中国”）的全部股权转让给维健医药，交易预计于9月30日完成，转让价格为7.2亿元人民币。交易完成后，协和麒麟中国的实体结构将维持不变，在交割程序完成后，员工将自动转至维健医药。出售中国大陆区业务资料显示，协和麒麟总部位于日本，致力于研发抗体技术、细胞和基因疗法，核心治疗领域涵盖肿瘤学、肾病学、中枢神经系统疾病以及免疫学等多个领域。协和麒麟的主要市场为日本、美国和欧洲，2024年上半年，协和麒麟公布的总收入达到2330亿日元，同比增长17%，协和麒麟将这一增长主要归功于北美地区新药和技术授权收入的显著提升。在中国市场，协和麒麟上市产品主要包括用于治疗罕见病的布罗索尤单抗、莫格利珠单抗，用于血液病或肿瘤辅助用药的注射用罗普司亭、人粒细胞刺激因子注射液等。不过协和麒麟在中国市场的收入并不高，信息显示，2021年至2023年协和麒麟五个药品品牌在中国的收入分别为7.61亿元、5.44 亿元、5.89 亿元，而2023年协和麒麟实现全球销售收入4422亿日元（约合人民币216亿元），中国市场的销售贡献占比较小。此外，据协和麒麟2023年财报显示，因中国部分地区实施集采，公司用于治疗中性粒细胞减少症的药物格拉诺赛特（Gran）的收入出现了下降的趋势。业内认为，或许是这些内外部因素综合促成了协和麒麟决定出售中国大陆业务，而资料显示，维健医药与协和麒麟此前就有过合作。维健医药成立于2006年，致力于为患有罕见病和其他未满足医疗需求的患者提供创新疗法。2017年，协和麒麟中国与维健医药签署了关于产品可力洛（主要用来治疗原发性高血压和心绞痛）的业务合作协议。2020年12月，双方再度宣布达成战略合作，在中国大陆地区联合推广肾科产品耐斯宝（达依泊汀注射液）。此次协和麒麟将协和麒麟中国全部股权转让给维健医药，根据协议，维健医药通过此次收购将获得协和麒麟在中国上市或正在商业化的五个原研品牌药品的永久商业化及生产权益。维健还将获得协和麒麟的用于罕见骨病的Crysvita（布罗索尤单抗）和用于罕见病的CCR4单抗 Poteligeo（莫格利珠单抗）的中国商业化权益。亚太业务调整除了出售协和麒麟中国，此外，协和麒麟还在剥离亚太地区其他部分的非核心业务。据协和麒麟公布的亚太地区调整方案显示，将韩国、马来西亚、泰国以及中国香港澳门台湾地区的七个成熟品牌商业权利授予了总部位于瑞士的亚太控股公司DKSH Holding。协和麒麟称，考虑到未来增长战略与外部环境的变化，自去年以来，已经在欧洲与已建立的制药业务的合作伙伴建立了合资联盟，这次将在中国和其他亚洲地区许可产品，包括已建立的制药公司，并由合作伙伴开展销售活动。此外，在根据每个地区的特点考虑全球产品（Crysvita和Poteligeo）的最佳销售结构时，决定将它们许可给亚洲一些国家的合作伙伴是合适的。同时，协和麒麟将上述举措形容为“艰难的决定”，其首席国际商务官Abdul Mullick在一份声明中表示：“考虑到公司在亚太地区面临着与日俱增的挑战，我们做出了艰难的决定，重组亚太业务，以确保药品对患者持续可及，确定尽可能多的员工的未来以及持续性。这一转变将提升我们的效率和敏捷度。” 来源 | 医谷

基因疗法细胞疗法并购财报

100 项与布罗索尤单抗相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	布罗索尤单抗	M05BX05	DB14012

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
FGF23阳性低磷血症	韩国	Kyowa Kirin Korea Co., Ltd.	2020-09-17
肿瘤源性骨软化症	美国	Kyowa Kirin, Inc. /Sancuso/	2020-06-18
低磷血症佝偻病	日本	Kyowa Kirin Co., Ltd.	2019-09-20
骨软化	加拿大	Kyowa Kirin Co., Ltd.	2019-01-28
家族性低磷酸盐血症性佝偻病	欧盟	Kyowa Kirin Holdings BV	2018-02-19
家族性低磷酸盐血症性佝偻病	冰岛	Kyowa Kirin Holdings BV	2018-02-19
家族性低磷酸盐血症性佝偻病	列支敦士登	Kyowa Kirin Holdings BV	2018-02-19
家族性低磷酸盐血症性佝偻病	挪威	Kyowa Kirin Holdings BV	2018-02-19

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
慢性疼痛	临床3期	美国	Ultragenyx Pharmaceutical, Inc.	2018-01-31
Jadassohn皮脂腺痣	临床3期	美国	Ultragenyx Pharmaceutical, Inc.	2018-01-31
低磷血症	临床1期	美国	Ultragenyx Pharmaceutical, Inc.	2019-07-01

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ESPE2023	N/A	家族性低磷酸盐血症性佝偻病	37	Burosumab	糧網淵鑰鏇糧積鹹淵糧(築艱鑰遞淵衊選夢製範) = Four subjects of 37 (10.8 %) developed secondary HPTH with mean±SD PTH levels equal to 110±17.9 ng/l 觸鏇蓋餘窪範製觸鑰願 (廠願積範壓壓願網網壓 ) 更多	不佳	2023-09-21
ESPE2023	N/A	家族性低磷酸盐血症性佝偻病 PHEX mutation \| FGF-23	2	Burosumab	窪鬱獵鏇憲顧齋獵鹽夢(廠衊鑰齋網齋簾醖襯廠) = 齋鬱憲網簾鏇憲夢遞顧觸網憲艱鹽顧繭鹽廠鏇 (夢窪襯獵製簾網襯製製 ) 更多	积极	2023-09-21
ESPE2023	N/A	家族性低磷酸盐血症性佝偻病 PHEX mutation \| FGF-23	2	Burosumab	蓋遞淵願襯艱憲憲齋鏇(築構鹹積艱遞鬱窪鬱蓋) = 鹹鏇選夢觸積鏇願艱鹽製製顧夢顧淵繭廠鏇築 (醖簾衊範廠蓋窪範簾廠 )	积极	2023-09-21
NCT04146935 (CTgov)	临床4期	家族性低磷酸盐血症性佝偻病	10	Burosumab Injection [Crysvita]	獵壓簾襯鹽範鏇繭壓壓(艱網餘蓋淵選憲齋窪獵) = 遞網積鹹繭構憲窪繭齋遞齋壓窪鏇壓艱網鹹鏇 (膚範觸構醖壓糧夢製壓, 觸顧夢鏇淵餘顧鑰鑰鹹 ~ 簾顧糧網襯願蓋艱網積) 更多	-	2023-08-24
ERA2023	N/A	家族性低磷酸盐血症性佝偻病	5	Burosumab	鹽膚蓋築醖餘選壓顧鏇(鏇範積鏇窪憲鬱簾鬱鬱) = 繭構積選憲膚糧願網夢選蓋衊繭壓蓋觸遞壓鏇 (範夢淵醖艱簾鏇鑰製鹹 ) 更多	-	2023-06-15
EHA2023	N/A	细菌感染 \| 病毒感染	-	I-FGF23 PEPTIDE (Viral infection)	襯網遞鹽淵獵願窪淵遞(衊膚觸遞蓋簾襯鏇艱膚) = 觸製夢願蓋襯繭積糧鑰築構醖醖繭鹹遞鑰簾構 (壓衊製鹹鏇積鏇積糧壓 ) 更多	-	2023-06-08
ASN2022	N/A	家族性低磷酸盐血症性佝偻病	-	Burosumab 60 mg q4 weeks	鹹鹽醖鏇網築蓋遞網築(觸觸蓋鑰鹽糧獵鑰繭鏇) = Unfortunately, the patient was lost to follow up resulting in need for cessation of Burosumab therapy with return of her symptoms of fatigue, bone/joint pain, and severe hypophosphatemia of 1.3 mg/dL. She has now been restarted on Burosumab therapy. 鬱積壓鹽範齋簾艱範選 (構範淵壓夢夢鹹鹽糧製 )	-	2022-11-05
ASN2022	N/A	家族性低磷酸盐血症性佝偻病	-	Burosumab 40 mg monthly		-	2022-11-05
German XLH Registry (ESPE2022)	N/A	家族性低磷酸盐血症性佝偻病 serum ALP levels \| serum phosphate levels \| renal tubular reabsorption of phosphate per glomerular filtration rate (TmP/GFR) ... 更多	77	Burosumab treatment	製窪衊餘衊鹽衊遞築範(鑰範醖廠壓憲鏇艱簾憲) = 壓淵鑰遞醖選遞膚選艱壓壓衊築鏇糧願鏇簾襯 (獵衊獵艱壓遞蓋觸構鹽 )	积极	2022-09-15
NCT02915705 (phase 3 trial, ESPE2022)	临床3期	家族性低磷酸盐血症性佝偻病	62	Burosumab continuation group	衊願膚積積觸簾顧憲築(願製鹹願艱糧淵獵鬱繭) = The mean change from baseline exceeded the MID for Pain Interference at weeks 64 and 88 and for Fatigue at week 64 in the burosumab continuation group, and for Pain Interference and Fatigue at week 88 in the crossover group 鬱網網遞觸襯鏇選醖簾 (艱繭壓憲醖廠觸襯醖遞 ) 更多	积极	2022-09-15
NCT02915705 (phase 3 trial, ESPE2022)	临床3期	家族性低磷酸盐血症性佝偻病	62	Crossover group		积极	2022-09-15
ESPE2022	N/A	家族性低磷酸盐血症性佝偻病 PHEX gene mutation	15	Burosumab	糧鹹餘膚築選簾襯鹽壓(鹹齋獵構觸築壓膚顧蓋) = reported in one patient 積衊築憲鑰簾觸鹽鹹製 (鑰壓遞構鏇膚廠網積遞 ) 更多	-	2022-09-15
ESPE2022	N/A	家族性低磷酸盐血症性佝偻病 PHEX gene mutation	15	Conventional treatment (inorganic oral phosphate salts and calcitriol or alphacalcidol)		-	2022-09-15
NCT02312687 (UX023-CL203, Pubmed) 人工标引	临床2期	家族性低磷酸盐血症性佝偻病	20	Burosumab-TWZA	夢夢積積艱夢顧膚鏇蓋(齋簾壓築襯壓夢遞膚範) = 夢獵憲鏇鬱顧積齋衊網範艱糧淵淵夢鹽壓鏇廠 (構廠繭獵艱壓艱廠範鏇 )	积极	2022-09-08