ESPEYB16 5. Bone, Growth Plate and Mineral Metabolism Clinical Advances in Treatment (2 abstracts)
5.8. Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial
Imel EA , Glorieux FH , Whyte MP , Munns CF , Ward LM , Nilsson O , Simmons JH , Padidela R , Namba N , Cheong HI , Pitukcheewanont P , Sochett E , Högler W , Muroya K , Tanaka H , Gottesman GS , Biggin A , Perwad F , Mao M , Chen CY , Skrinar A , San Martin J & Portale AA
Department of Medicine and Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA
Abstract: Lancet. 2019 May 16.
In brief: In a randomised, active-controlled, open-label, phase 3 trial, burosumab (an anti-FGF23 antibody) demonstrated significantly greater clinical improvements in rickets severity, growth, and biochemistries among children with X-linked hypophosphataemia compared with continuation of conventional therapy with oral phosphate and active vitamin D analogues.
Comment: Burosumab, a fully human monoclonal antibody against FGF23, received approval from the US Food and Drug Administration and from Health Canada, and conditional marketing approval by the European Medicines Agency in 2018 for the treatment of X-linked hypophosphataemia. Previously, two paediatric, single-arm, phase 2 clinical trials had shown that inhibition of FGF23 with burosumab restored phosphate homeostasis, and improved rickets, growth, and mobility in affected 1–12 year old children.
This international, randomised, active-controlled, open-label, phase 3 trial is the first to compare the efficacy and safety of switching to burosumab versus continuing conventional therapy among 1–12 year old children with X-linked hypophosphataemia. 64-weeks of burosumab treatment resulted in greater radiographic improvement of rickets and lower extremity bowing, larger decreases in serum alkaline phosphatase activity, and greater increases in serum phosphorus, growth, and mobility than continuation of conventional therapy.
This phase 3 trial presents the first comparison of conventional therapy with burosumab in children with X-linked hypophosphataemia and showed the superiority of burosumab over continuation of conventional therapy for several clinical outcomes, including the correction of renal phosphate wasting. By improving rickets, long bone deformities, and linear growth, burosumab confirms the promising results from the phase 2 trials and demonstrate that this new treatment approach for children with X-linked hypophosphataemia clearly is superior to optimised conventional treatment.
Volume 16
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ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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