Crysvita (burosumab-twza) / Ultragenyx, Kyowa Kirin - LARVOL DELTA

Use of burosumab in McCune Albright syndrome: case report and review of literature in mosaic disorders with FGF23 overproduction. (PubMed, Front Endocrinol (Lausanne)) - "At the age of 5 years and 6 months, intravenous neridronate was started every 3 months with a partial improvement of bone pain and bone deformities. Nonetheless, a partial progression of FD was documented on periodic X-rays. Burosumab showed beneficial effects on bone tissue metabolisms in our patient without significant adverse effects but did not change FD course."

Journal • Review • Genetic Disorders • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Pain • Pediatrics • Renal Disease • FGF23

Sustained Improvements in Patient-Reported Outcomes Across Age Groups in Burosumab-Treated X-linked Hypophosphatemia (XLH): Results From the XLH Disease Monitoring Program (ENDO 2025) - P | "Adults with XLH also showed significant improvements at DMP Y1 and 3 in all WOMAC domains. Despite small sample sizes, this analysis provides real-world data on the positive impact of burosumab treatment on PROs."

Clinical • Patient reported outcomes • Fatigue • Immunology • Osteoarthritis • Pain • Renal Disease • Rheumatology • FGF23

Real-World Outcomes in Late Adolescence Among Individuals With X-linked Hypophosphatemia (XLH): Results From the XLH Disease Monitoring Program (ENDO 2025) - P | "ConclusionBiochemical parameters and patient-reported outcomes were generally stable for older adolescents treated with burosumab, suggesting that consistent disease management, as anticipated in the DMP, was in place at a time when many persons may otherwise be lost to follow-up. As the DMP evolves, further analyses will be performed to incorporate additional pts and longer follow-up times to enhance the understanding of outcomes during late adolescence."

Clinical • Real-world • Real-world evidence • Fatigue • Immunology • Osteoarthritis • Pain • Renal Disease • Rheumatology

Tumor Induced Osteomalacia Caused by Metastatic Thyroid Cancer (ENDO 2025) - "Cabozantinib, a tyrosine kinase inhibitor, and palliative radiation therapy were initiated along with zoledronate... TIO is a rare paraneoplastic disorder often associated with phosphaturic mesenchymal tumors. The patient's clinical presentation of severe FGF23 mediated hypophosphatemia and diffuse skeletal fragility was initially misattributed to metastatic poorly differentiated thyroid cancer. Despite extensive evaluation, no secondary mesenchymal tumor was identified on imaging."

Metastases • Endocrine Cancer • Endocrine Disorders • Fatigue • Musculoskeletal Diseases • Oncology • Pain • Renal Disease • Solid Tumor • Thyroid Gland Carcinoma • Thyroid Gland Papillary Carcinoma • FGF23

Impact of Treatment with Burosumab versus Conventional Therapy on Patient-Important Outcomes in Adult X-Linked Hypophosphatemia: A Retrospective and Prospective Cohort Study (ENDO 2025) - "Patients on burosumab had lower BPI pain, WOMAC physical function, and total WOMAC scores than those on CT, suggesting less pain, stiffness, and functional limitation. Lower WOMAC physical function scores for patients on burosumab indicate fewer difficulties completing daily tasks such as walking, sitting, and climbing stairs compared to CT. However, this was not statistically significant."

Retrospective data • Fatigue • Genetic Disorders • Obesity • Pain • Renal Disease

Three-Year Evolution of X-Linked Hypophosphatemia in Children Treated with Burosumab: Predictive Factors for Dosing and Long-Term Outcomes (ENDO 2025) - "Over 3 years, burosumab treatment led to expected sustained biochemical and skeletal improvements in children with XLH. The required burosumab dose was influenced by baseline height SDS and BMI SDS, suggesting that these factors may be useful for individualized dose adjustments. Additionally, long-term complications, such as nephrocalcinosis and hearing loss, showed a decreasing trend, suggesting the potential protective effects of burosumab."

Biomarker • Clinical • Metabolic Disorders • Nephrology • Orthopedics • Otorhinolaryngology • Pediatrics • Renal Disease • FGF23

Tumor-Induced Osteomalacia Complicated with Post-operative Hungry-Bone Syndrome and Delay Reduction of Fibroblast Growth Factor 23 (ENDO 2025) - "It is known to interfere with the measurement of FGF23 levels leading to anomalously high C-terminal fragment results and has a prolonged elimination half-life of 19 days. Persistent elevation in FGF23 levels should be expected if patients with TIO have received Burosumab preoperatively."

Endocrine Disorders • Fatigue • Musculoskeletal Pain • Myositis • Oncology • Orthopedics • Osteoporosis • Pain • Renal Disease • Rheumatology • FGF23

X linked hypophosphatemia XLH Follow up experience in Argentina (ENDO 2025) - "At one year of follow-up, patients under treatment with burosumab presented better levels of phosphataemia, FAL and PTH and all were in stable work. 4/6 patients under conventional treatment are unemployed. Longer-term studies are needed to evaluate the impact of both therapies."

Orthopedics • Renal Disease • FGF23

Challenges in the management of pregnancy in a woman with XLH undergoing burosumab treatment (ENDO 2025) - "This is the first report of pregnancy in a XLH woman undergoing burosumab therapy. Burosumab appears to be safe during conception. Real-world data are necessary to evaluate its long-term safety and confirm this finding."

Clinical • Genetic Disorders • Musculoskeletal Pain • Pain • Renal Disease • FGF23

Toward lean body weight- and ECV-based dosing of burosumab: eliminating apparent sex differences in XLH therapy. (PubMed, Pediatr Nephrol) - No abstract available

Journal

Examining Burden of Disease and Access to Care in Adults with X-Linked Hypophosphatemia in Brazil (ISPOR 2025) - "Despite generally comparable measures of XLH disease burden, far fewer Brazilian adults received burosumab (5/50; 10%) versus the overall DMP population (332/481; 69%). This highlights the unmet need for access to adequate treatment for Brazilian adults with XLH, which likely stems from the lack of reimbursement in this country despite marketing authorization of burosumab 5 years ago."

Clinical • HEOR • Genetic Disorders • Pain • Renal Disease • FGF23

Burosumab treatment of tumour-induced osteomalacia from a rib lesion. (PubMed, BMJ Case Rep) - "The patient was started on burosumab, a human monoclonal antibody against FGF-23, which normalised her serum phosphate and led to marked clinical improvement. She is now awaiting curative surgery for the primary rib lesion."

Journal • Oncology • Pain • Renal Disease • Sarcoma • Solid Tumor • Spindle Cell Sarcoma • FGF23

Real-world characteristics & disease history of patients with X-linked hypophosphatemia before treatment with burosumab. (PubMed, Arch Osteoporos) - "At initiation of burosumab, over half of patients with XLH were < 18 years of age. Claims indicated a high prevalence of XLH-related morbidities, which began at a young age and increased over time."

Journal • Real-world evidence • Retrospective data • Cardiovascular • CNS Disorders • Depression • Hypertension • Immunology • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Osteoarthritis • Pain • Psychiatry • Renal Disease • Rheumatology

Oncogenic rickets diagnosed at age 8 and the risk of persistent rickets: a rare case of pediatric-onset tumor-induced osteomalacia. (PubMed, Osteoporos Int) - "Initially misdiagnosed with X-linked hypophosphatemia (XLH) and treated with burosumab, genetic testing ruled out hereditary hypophosphatemic disorders...Pediatric-onset TIO is an underrecognized entity that can lead to severe disability if not diagnosed early. This case underscores the importance of early tumor identification."

Journal • Musculoskeletal Diseases • Oncology • Orthopedics • Pain • Pediatrics • Renal Disease • FGF23

Short-term outcome of a novel treatment with burosumab in Polish patients with XLH (ESPE-ESE 2025) - "Burosumab as a recombinant antibody against FGF23 has become an effective therapeutic option for patients with XLH. Their short-term follow-up revealed a reduction of lower limb deformities and marked biochemical and radiological improvement."

Clinical • Late-breaking abstract • Orthopedics • Pediatrics • Renal Disease • FGF23

The management of X-Linked Hypophosphatemia: Oral phosphate and calcitriol supplementation vs. Burosumab. A case report (ESPE-ESE 2025) - "In severe XLH, conventional therapy often fails to correct hypophosphatemia and skeletal deformities. This case underscores Burosumab’s superior efficacy, significantly improving phosphate homeostasis, bone health, and overall quality of life."

Case report • Clinical • Genetic Disorders • Metabolic Disorders • Nephrology • Renal Disease • FGF23

Is Burosumab a Good Choice?: Experience in Adult X-Linked Hypophosphatemic Rickets in a Turkish Patient (ESPE-ESE 2025) - "There were no side affects releated to Burosumab treatment.At the followup 3 monts after the treatmet the patient report improvements in pain, mobility,physical function, energy , fatigue and mental wellbeing also there is improvement in laboratory values (f.e phosphorus levels increased from 2.1 to 3 )(shown in Table 1 and Table2). Conclusion In our case we reported that a positive laboratory and clinical response with Burosumab treatment in the diagnosis of X-linked hypophosphatemic rickets, and observed no side effects developed in the short term.The lack of a significant improvement in 25-OH vitamin D and ALP values may be due to the early period.However,since Burosumab treatment is a new and promising option,its effectiveness and safety are unknown.We emphasize that there is a need for studies with more case reports and a larger number of patients to monitor long term effects."

Clinical • Endocrine Disorders • Fatigue • Metabolic Disorders • Nephrology • Orthopedics • Pain • Renal Disease • Urinary Incontinence • Urology • ERBB3 • FGF23

Analysis of the therapeutic effect of Burosumab in the treatment of 6 children with X-Linked Hypophosphatemia (ESPE-ESE 2025) - "One patients showed nephrocalcinosis, while the other patients did not experience any adverse drug reactions such as nephrocalcinosis, local skin injection reaction, hyperphosphatemia, or vitamin D deficiency were observed. Conclusion Treatment with brosuzumab can improve clinical symptoms in children with XLH, and no adverse drug reactions have been observed."

Clinical • Metabolic Disorders • Nephrology • Pediatrics • Renal Disease

Clinical and Molecular profiles of 14 new patients with X-linked hypophosphatemia (XLH) in Greece (ESPE-ESE 2025) - "Conclusion This is the first study describing the detailed molecular and clinical profiles of X-linked hypophosphatemic patients in Greece. Most of them have, recently, started receiving burosumab, a fully humanized monoclonal antibody that neutralizes circulating FGF23, and their response to treatment is being recorded."

Clinical • Immunology • Metabolic Disorders • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Osteoarthritis • Pain • Renal Disease • Rheumatology • FGF23

X-linked Hypophosphatemic Rickets in children and adolescents - single centre expertise in Romania (ESPE-ESE 2025) - "There are two main therapeutic options: conventional therapy (phosphate supplements and active vitamin D) and biological therapy (Burosumab, a specific antibody against FGF23, EMA approved since 2018)...Conclusions. Although a rare disease, XLH can be easily identified, evaluated, and treated by multidisciplinary teams from specialised bone disorders children’s centers."

Clinical • Endocrine Disorders • Musculoskeletal Diseases • Nephrology • Orthopedics • Pediatrics • Rare Diseases • Renal Calculi • Renal Disease • FGF23

A Rare Case of Genetically Confirmed X-linked Dominant Hypophosphatemic Rickets in a 7-year-old Girl from Kazakhstan (ESPE-ESE 2025) - "The patient was diagnosed with X-linked dominant hypophosphatemic rickets based on clinical, biochemical, genetic, and imaging findings. Targeted therapy with burosumab (Crivit) was initiated at a dose of 0.8 mg/kg subcutaneously every two weeks. This case highlights the importance of early genetic testing, comprehensive metabolic evaluation, and targeted treatment in improving outcomes for patients with XLH."

Clinical • Dental Disorders • Endocrine Disorders • Genetic Disorders • Musculoskeletal Pain • Pain • Pediatrics • Renal Disease • FGF23

Hereditary and Acquired Phosphopenic Rickets: From Childhood to Adulthood (ESPE-ESE 2025) - "Diagnosis included cisplatin toxicity (CT)(n=1, Pearson syndrome n=1, cystinosis n=2, and RTD n=3. Elucidation of etiology will enable early and correct diagnosis but delay causes severe morbidity. Burosumab, an FGF23 inhibitor, targeted therapy for X-linked hypophosphatemia improves skeletal deformities and growth in children, enhances fracture healing, reduces pain, and boosts physical activity in adults."

Clinical • Hepatology • Liver Failure • Metabolic Disorders • Musculoskeletal Diseases • Nephrology • Orthopedics • Pain • Pediatrics • Renal Disease • FGF23

Successful Treatment of Autosomal Dominant Hypophosphatemic Rickets with Burosumab: A Case Report (ESPE-ESE 2025) - "Burosumab is potentially an effective treatment for ADHR, demonstrating rapid symptom relief, improved BMD, and enhanced fracture healing. While burosumab was well-tolerated, careful management of concurrent iron therapy is essential to avoid hyperphosphatemia. Further studies with larger cohorts are needed."

Case report • Clinical • Genetic Disorders • Hematological Disorders • Metabolic Disorders • Musculoskeletal Diseases • Musculoskeletal Pain • Nephrology • Orthopedics • Pain • Renal Disease • FGF23

Non-interventional Post-Authorisation Safety Study of Burosumab in the Treatment of Children and Adolescents with X-linked Hypophosphataemia: Second Interim Analysis (ESPE-ESE 2025) - P | "†Nephrocalcinosis. AE, adverse event; CKD chronic kidney disease; CSAF, cohort SAF; GFR, glomerular filtration rate; SAF, safety analysis set."

Clinical • Chronic Kidney Disease • Metabolic Disorders • Musculoskeletal Pain • Nephrology • Orthopedics • Pain • Pediatrics • Renal Disease • FGF23

Real-world efficacy and safety of burosumab in tumor-induced osteomalacia: case series from an early access program. (PubMed, JBMR Plus) - "These preliminary findings following burosumab EAP for patients with TIO in France support benefits in terms of efficacy, safety, and ease of treatment. Burosumab appears to be a promising option for patients who are ineligible or refractory to surgery."

Journal • Real-world evidence • Oncology • Pain • Renal Disease • FGF23