RegulationConditional marketing approval (CN), Overseas New Drugs Urgently Needed in Clinical Settings (CN), Orphan Drug (JP), Fast Track (US), Priority Review (AU), Breakthrough Therapy (US)

Gene Sequence

Sequence Code 10038962H

Source: *****

Sequence Code 10038967L

Source: *****

Clinical Trials associated with Burosumab-TWZA

NCT06202027 / RecruitingNot Applicable

Post Marketing Surveillance Study to Observe Safety and Effectiveness of CRYSVITA® in S. Korean Patients

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

Start Date11 Aug 2023

Sponsor / Collaborator

Kyowa Kirin Korea Co., Ltd.

NCT05509595 / Active, not recruitingPhase 2IIT

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.

Start Date07 Dec 2022

Sponsor / Collaborator

National Institute of Dental & Craniofacial Research

NCT05357573 / CompletedPhase 4

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

Start Date07 Sep 2022

Sponsor / Collaborator

Kyowa Kirin Co., Ltd.

100 Clinical Results associated with Burosumab-TWZA

100 Translational Medicine associated with Burosumab-TWZA

100 Patents (Medical) associated with Burosumab-TWZA

259

Literatures (Medical) associated with Burosumab-TWZA

01 Jan 2025·Bone

Tertiary hyperparathyroidism in two paediatric patients with X-linked hypophosphatemia during Burosumab treatment

Article

Author: Rayner, Lauren ; Boot, Annemieke M ; Boot, Annemieke M. ; Padidela, Raja

INTRODUCTIONAlthough secondary hyperparathyroidism is known in X-linked hypophosphatemia (XLH) patients receiving treatment, tertiary hyperparathyroidism with hypercalcemia is rare, especially in children. We report two paediatric XLH patients treated with Burosumab who developed this rare complication.CASE DESCRIPTIONS1: A female patient with XLH on conventional therapy (phosphate and active vitamin D) since one year of age was switched to Burosumab at 10 years. At 14 years of age, she developed tertiary hyperparathyroidism with hypercalcaemia. Burosumab was continued. Post-parathyroidectomy her hypercalcaemia resolved and 4 years post-surgery her calcium levels continue to remain normal. 2: A female patient with XLH on conventional therapy since 4 months of age was switched to Burosumab at 4 years of age. At 7 years of age, she developed secondary hyperparathyroidism and within 6 months she developed tertiary hyperparathyroidism. Burosumab was discontinued at 7.5 years of age, and she was commenced on Cinacalcet but, hypercalcaemia failed to resolve. Post-parathyroidectomy her tertiary hyperparathyroidism resolved. However, within 2 weeks, PTH increased which normalised with Cinacalcet. Burosumab has been recommenced and she continues cinacalcet.DISCUSSIONThe cause of tertiary hyperparathyroidism is not clear in these patients. Higher post-dose phosphate levels or a direct effect of PHEX mutation on the parathyroid gland could have triggered PTH secretion.CONCLUSIONXLH patients treated with Burosumab can develop hyperparathyroidism and should be regularly monitored for the development of secondary and tertiary hyperparathyroidism.

01 Dec 2024·Clinical Pharmacology & Therapeutics

Model‐Informed Approach to Recommend Burosumab Dosing Regimens for Pediatric and Adult Patients With the Ultrarare Disease Tumor‐Induced Osteomalacia

Article

Author: Lee, Sun Ku ; Mascelli, Mary Ann ; Jan de Beur, Suzanne M. ; Marsteller, Douglas ; Mehta, Krina ; Sid‐Otmane, Lamia ; Hruska, Matthew W. ; Quattrocchi, Emilia ; Gosselin, Nathalie H.

Burosumab is approved for the treatment of hypophosphatemia in persistent tumor‐induced osteomalacia. This work exemplifies a model‐informed drug development approach that evaluated burosumab pharmacokinetics and pharmacokinetic/pharmacodynamics in the ultrarare tumor‐induced osteomalacia population to support adult and pediatric dosing. Data from tumor‐induced osteomalacia participants were combined with data from X‐linked hypophosphatemia to understand pharmacokinetic and pharmacokinetic/pharmacodynamic characteristics and covariates specific to tumor‐induced osteomalacia. Pharmacokinetic and pharmacokinetic/pharmacodynamic simulations were performed using final models to support dosing recommendations for adults and extrapolation to pediatric patients. Burosumab pharmacokinetics were described using a one‐compartment model with first‐order absorption and body weight as a significant covariate. Pharmacokinetic/pharmacodynamic relationships were described using a sigmoidal Emax model with significant covariates of baseline fibroblast growth factor 23 on baseline fasting serum phosphate and potency of burosumab response and tumor‐induced osteomalacia disease state resulting in a steep slope of response; however, the covariates are not clinically meaningful. Simulations demonstrated that, in pediatric patients, starting doses of burosumab 0.3 and 0.4 mg/kg every 2 weeks at steady state would achieve normal serum phosphate levels in ≥ 30% of patients with relatively low risk of hyperphosphatemia (< 3%). In adults, burosumab 0.3 and 0.5 mg/kg every 4 weeks achieves similar percentages of responders and a relative low risk of hyperphosphatemia (< 7%). Serum phosphate titration‐based burosumab dosing increased the probability of achieving normal serum phosphate levels. The models supported a model‐informed drug development approach for global approvals of titration‐based burosumab dosing, guided by monitoring fasting serum phosphate levels.

01 Nov 2024·Osteoporosis International

Longitudinal assessment of physical function in adults with X-linked hypophosphatemia following initiation of burosumab therapy

Article

Author: Orlando, Giorgio ; Roy, Matthew ; Keen, Richard ; Bubbear, Judith ; Ireland, Alex ; Javaid, Muhammad Kassim ; Clarke, Shane

We assessed multiple components of muscle function in ten adults with X-linked hypophosphatemia (XLH) receiving burosumab treatment. Lower limb power (+ 9%), short physical performance battery (SPPB) score (+ 1.2 points), and physical activity (+ 65%) increased following 6 months of treatment, and hand grip increased (+ 10%) between 6 and 12 months of treatment.PURPOSEX-linked hypophosphatemia (XLH) is a rare genetic disorder of phosphate metabolism. Burosumab is a monoclonal antibody treatment shown to improve phosphate homeostasis and improve symptoms as well as fracture healing when used as a therapy for XLH in adults. However, little is known about its effects on the large deficits in multiple components of physical function previously reported in XLH.METHODSTen adults (6 females, age 41.1 ± 15.7 y) were recruited from specialist centres in London and Bristol. During clinical visits for initial burosumab treatment and at 6-month and 12-month follow-up, physical function, and physical activity (PA) assessments were performed. In detail, lower limb power was assessed by mechanography via a countermovement jump, mobility by short physical performance battery (SPPB), functional capacity by 6-min walk test (6MWT), upper limb strength by hand grip dynamometry, and PA via an International Physical Activity Questionnaire (IPAQ). Differences between baseline and 6-month follow-up, and in a subset of 5 patients between 6- and 12-month follow-up, were assessed.RESULTSLower limb power increased by 9% (P = 0.049) from baseline to 6 months, as did SPPB score (+ 1.2 points, P = 0.033) and total PA (+ 65%, P = 0.046) although hand grip and 6MWT did not differ. Only for hand grip was a significant improvement (+ 10%, P = 0.023) seen between 6 and 12 months.CONCLUSIONSBurosumab treatment is associated with improved lower limb function and mobility at 6 months, with improvement in hand grip strength at 12 months. Future studies should explore the underlying mechanisms and describe on function and other patient outcomes.

News (Medical) associated with Burosumab-TWZA

19 Sep 2024

·www.prnewswire.com

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

Phase 3Clinical Result

10 Sep 2024

·www.businesswire.com

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].

Clinical ResultClinical Study

07 Aug 2024

·www.businesswire.com

Kyowa Kirin International and NewBridge Pharmaceuticals Enter Into Agreement to Improve Access to Medicines for Rare Disease Patients Across Middle East and North Africa

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 signing of a Promotion and Distribution Agreement with NewBridge Pharmaceuticals for the distribution of its existing rare disease portfolio across the Middle East and North Africa (MENA). Under the terms of the agreement, NewBridge will commercialise CRYSVITA ® (burosumab) for the treatment of X-Linked Hypophosphataemia (XLH) and Tumour-Induced Osteomalacia (TIO), as well as POTELIGEO ® (mogamulizumab) for two subtypes of cutaneous T-cell lymphoma (CTCL), a rare non-Hodgkin’s lymphoma. As part of the agreement, NewBridge will exclusively distribute the two products across MENA countries of Algeria, Iraq, Libya and Jordan. KKI and NewBridge also hope to collaboratively address a number of challenges facing the rare disease community in the region by supporting disease awareness, improving time to diagnosis, and improving access to innovative treatments indicated for rare and orphan diseases. Jeremy Morgan, President of KKI, commented: “At Kyowa Kirin our purpose is to deliver life-changing medicines and make people smile. We are pleased to be entering into this agreement with NewBridge Pharmaceuticals who have a clear mission to transform the lives of MENA patients and their families. With a commitment to delivering life changing value for people impacted by under-diagnosed and under-served diseases, this agreement is key to ensuring patients living with XLH, TIO and CTCL are able to access the medicines they need, and is another positive step on our journey.” "Given the high prevalence of rare diseases in the MENA region, I am personally inspired and proud that we at NewBridge are building on our existing partnership with Kyowa Kirin with the addition of their rare disease products to our region,” added Joe Henein, President & CEO of NewBridge Pharmaceuticals. “This collaboration totally aligns with our mission to provide access to new therapies for patients, aiming to alleviate their suffering and enhancing their quality of life." About CRYSVITA (burosumab) CRYSVITA (burosumab) is a recombinant human monoclonal antibody (mAb) that binds to the protein fibroblast growth factor 23 (FGF23). This has the impact of inhibiting the action of FGF23, allowing phosphate regulation in the body to be restored. 1 About POTELIGEO (mogamulizumab) Mogamulizumab is a first-in-class humanised mAb directed against CC chemokine receptor 4 (CCR4), a protein consistently expressed in both mycosis fungoides and Sézary syndrome 2–4 once mogamulizumab binds to CCR4, it increases attraction of immune cells from the immune system to destroy the cancerous cells. 5 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 About NewBridge Pharmaceuticals NewBridge Pharmaceuticals is a regional specialty company with a comprehensive pharmaceutical platform of services and expertise, established to bridge the access gap and partner with global pharma and biotech companies to in-license and commercialise innovative therapeutics that address unmet medical needs into the Middle East and North Africa (MENA) regions. For more information, please visit www.nbpharma.com References 1 Electronic Medicines Compendium. Crysvita 10 mg solution for injection. Available from: https://www.medicines.org.uk/emc/product/9779/smpc#gref . Last accessed: November 2023. 2 Ferenczi K, et al. Increased CCR4 expression in cutaneous T cell lymphoma. J Invest Dermatol. 2002;119:1405–10. 3 Yoshie O, et al. Frequent Expression of CCR4 in Adult T-Cell Leukemia and Human T-cell Leukemia Virus Type 1-transformed T cells. Blood. 2002;99(5):1505–11. 4 Ishida T, et al. Clinical Significance of CCR4 Expression in Adult T-cell Leukemia/Lymphoma: Its Close Association With Skin Involvement and Unfavorable Outcome. Clin Cancer Res. 2003;9:3625–34. 5 Duvic M, et al. Mogamulizumab for the treatment of cutaneous T-cell lymphoma: recent advances and clinical potential. Ther Adv Hematol. 2016;7(3):171–174. KKI/INT/KKI/0959 Date of preparation: July 2024

License out/in

100 Deals associated with Burosumab-TWZA

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KEGG	Wiki	ATC	Drug Bank
-	Burosumab-TWZA	M05BX05	DB14012

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
FGF23 positive Hypophosphatemia	KR	Kyowa Kirin Korea Co., Ltd.	17 Sep 2020
Oncogenic Osteomalacia	US	Kyowa Kirin, Inc. /Sancuso/	18 Jun 2020
Rickets, Hypophosphatemic	JP	Kyowa Kirin Co., Ltd.	20 Sep 2019
Osteomalacia	CA	Kyowa Kirin Co., Ltd.	28 Jan 2019
Familial Hypophosphatemic Rickets	NO	Kyowa Kirin Holdings BV	19 Feb 2018
Familial Hypophosphatemic Rickets	EU	Kyowa Kirin Holdings BV	19 Feb 2018
Familial Hypophosphatemic Rickets	IS	Kyowa Kirin Holdings BV	19 Feb 2018
Familial Hypophosphatemic Rickets	LI	Kyowa Kirin Holdings BV	19 Feb 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Osteomalacia	Phase 1	US	Kyowa Kirin, Inc. /Sancuso/	23 Dec 2015
Osteomalacia	Phase 1	JP	Kyowa Kirin, Inc. /Sancuso/	23 Dec 2015
Osteomalacia	Phase 1	US	Kyowa Kirin Co., Ltd.	23 Dec 2015
Osteomalacia	Phase 1	FR	Kyowa Kirin, Inc. /Sancuso/	23 Dec 2015
Familial Hypophosphatemic Rickets	Phase 1	IT	Kyowa Kirin, Inc. /Sancuso/	22 Oct 2015
Familial Hypophosphatemic Rickets	Phase 1	KR	Kyowa Kirin, Inc. /Sancuso/	22 Oct 2015
Familial Hypophosphatemic Rickets	Phase 1	GB	Kyowa Kirin, Inc. /Sancuso/	22 Oct 2015
Familial Hypophosphatemic Rickets	Phase 1	IE	Kyowa Kirin, Inc. /Sancuso/	22 Oct 2015
Familial Hypophosphatemic Rickets	Phase 1	FR	Kyowa Kirin, Inc. /Sancuso/	22 Oct 2015
Familial Hypophosphatemic Rickets	Phase 1	JP	Kyowa Kirin, Inc. /Sancuso/	22 Oct 2015

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ESPE2023	Not Applicable	Familial Hypophosphatemic Rickets	37	Burosumab	bopofarnwt(zaehpqudhs) = Four subjects of 37 (10.8 %) developed secondary HPTH with mean±SD PTH levels equal to 110±17.9 ng/l fabnfbwuqd (qgsalarbbx ) View more	Negative	21 Sep 2023
ESPE2023	Not Applicable	Puberty, Precocious \| Familial Hypophosphatemic Rickets	-	Leuprolide acetate	vttrhtfsam(qypuesgmee) = rymcqummon arqezffglv (bslhfrioji ) View more	Positive	21 Sep 2023
ESPE2023	Not Applicable	Puberty, Precocious \| Familial Hypophosphatemic Rickets	-	Burosumab	vttrhtfsam(qypuesgmee) = lawzvjeiss arqezffglv (bslhfrioji ) View more	Positive	21 Sep 2023
ESPE2023	Not Applicable	Familial Hypophosphatemic Rickets PHEX mutation \| FGF-23	2	Burosumab	(mtacgprsil) = uoppjrmpjj epbxrklafk (fpyrftmjps ) View more	Positive	21 Sep 2023
ESPE2023	Not Applicable	Familial Hypophosphatemic Rickets PHEX mutation \| FGF-23	2	Burosumab	(lqzagceisw) = lpcqrsblhe njrtuakeix (tcdqdlzphc )	Positive	21 Sep 2023
NCT04146935 (CTgov)	Phase 4	Familial Hypophosphatemic Rickets	10	Burosumab Injection [Crysvita]	hxywkkmmrt(raepyetxgw) = dqqwkjmrsb euxsmeozsd (bvpkcnsusd, bqxbjpanwn - cbupwvfaza) View more	-	24 Aug 2023
EHA2023	Not Applicable	Bacterial Infections \| Virus Diseases	-	I-FGF23 PEPTIDE (Viral infection)	oaqsbuhiti(ofhbmokngk) = xutpxpdaja wnoedylpzd (msikngzggs ) View more	-	08 Jun 2023
ASN2022	Not Applicable	Familial Hypophosphatemic Rickets	-	Burosumab 60 mg q4 weeks	(fxpvypvcms) = 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. tqkmvcqfiy (awhnzrpgvh )	-	05 Nov 2022
ASN2022	Not Applicable	Familial Hypophosphatemic Rickets	-	Burosumab 40 mg monthly		-	05 Nov 2022
ESPE2022	Not Applicable	Familial Hypophosphatemic Rickets PHEX gene mutation	15	Burosumab	(dcvpikxmof) = reported in one patient hvjgczokpl (gtdeooemlg ) View more	-	15 Sep 2022
ESPE2022	Not Applicable	Familial Hypophosphatemic Rickets PHEX gene mutation	15	Conventional treatment (inorganic oral phosphate salts and calcitriol or alphacalcidol)		-	15 Sep 2022
German XLH Registry (ESPE2022)	Not Applicable	Familial Hypophosphatemic Rickets serum ALP levels \| serum phosphate levels \| renal tubular reabsorption of phosphate per glomerular filtration rate (TmP/GFR) ... View more	77	Burosumab treatment	(eicoxblguf) = movmiltbrg ywhfpyayrg (fclneeprvp )	Positive	15 Sep 2022
NCT02312687 (UX023-CL203, Pubmed) Manual	Phase 2	Familial Hypophosphatemic Rickets	20	Burosumab-TWZA	fimxtlwaef(pxtflmlgmo) = zyzfkbjguo ggkrezkxtf (ydzwldanbt )	Positive	08 Sep 2022
NCT02537431 (phase 3, Pubmed)	Phase 3	Familial Hypophosphatemic Rickets \| Osteomalacia	14	Burosumab	pzeutbiwnj(mbvoappumk) = ceodinhirf xrkgkzijfp (jqjawadntx )	Positive	01 Jul 2022

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