IMPE2023 Free Communications Growth and Syndromes (4 abstracts)
Persistent and stable growth promoting effects of vosoritide in children with achondroplasia for up to 3.5 years: results from an ongoing Phase 3 extension study
Lynda E. Polgreen 1 , Ravi Savarirayan 2 , Louise Tofts 3 , Melita Irving 4 , William Wilcox 5 , Carlos A. Bacino 6 , Julie Hoover-Fong 7 , Rosendo Ullot Font Ullot Font 8 , Paul Harmatz 9 , Frank Rutsch 10 , Michael B. Bober 11 , Ignacio Ginebreda 12 , Klaus Mohnike 13 , Joel Charrow 14 , Daniel Hoernschemeyer 15 , Keiichi Ozono 16 , Yasemin Alanay 17 , Paul Arundel 18 , Shoji Kagami 19 , Natsuo Yasui 19 , Klane White 20 , Howard M. Saal 21 , Antonio Leiva-Gea 22 , Felipe Luna-González 23 , Hiroshi Mochizuki 24 , Donald Basel 25 , Dania M. Porco 26 , Kala Jayaram 26 , Elena Fisheleva 27 , Sue Lawrinson 27 & Jonathan Day 27
1Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, USA. 2Murdoch Children’s Research Institute, Royal Children’s Hospital, and University of Melbourne, Parkville, Australia. 3Kids Rehab, The Children's Hospital at Westmead, Westmead, Australia. 4Guy’s and St. Thomas’ NHS Foundation Trust, Evelina Children's Hospital, London, United Kingdom. 5Emory University, Atlanta, USA. 6Baylor College of Medicine, Houston, USA. 7Johns Hopkins University School of Medicine, Baltimore, USA. 8Hospital Sant Joan de Déu, Barcelona, Spain. 9UCSF Benioff Children’s Hospital Oakland, Oakland, USA. 10Department of General Pediatrics, Muenster University Children’s Hospital, Münster, Germany. 11Nemours /Alfred I. du Pont Hospital for Children, Wilmington, USA. 12Hospital Universitario Quirón Dexeus, Barcelona, Spain. 13Otto-von-Guericke Universität, Universitätskinderklinik, Magdeburg, Germany. 14Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, USA. 15University of Missouri-Columbia, Columbia, USA. 16Osaka University Hospital, Osaka, Japan. 17Acibadem Mehmet Ali Aydiniar University, School of Medicine, Istanbul, Turkey. 18Sheffield Children's NHS Foundation Trust, Sheffield Children's Hospital, Sheffield, United Kingdom. 19Tokushima University Hospital, Tokushima, Japan. 20Seattle Children's Hospital, Seattle, USA. 21Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, USA. 22Hospital Universitario Virgen de la Victoria, Málaga, Spain. 23Hospitalario de Málaga, Málaga, Spain. 24Saitama Children’s Hospital, Saitama, Japan. 25Medical College of Wisconsin, Milwaukee, USA. 26BioMarin Pharmaceutical Inc., Novato, USA. 27BioMarin (U.K.) Limited, London, United Kingdom
Objectives: Vosoritide is a potent stimulator of endochondral bone growth and is in development for the treatment of achondroplasia, the most common form of disproportionate short stature. We previously reported on a 52-week, phase 3, pivotal study that demonstrated a significant improvement in annualized growth velocity (AGV) when vosoritide was compared to placebo in children with achondroplasia aged 5-18 years (Savarirayan et al, Lancet, 2020). This is an analysis of available long-term data in the ongoing phase 3 extension study.
Methods: After completion of the phase 3 placebo-controlled study, 119 children were enrolled into the extension study, where they all receive open label 15 μg/kg/day vosoritide. Fifty-eight continued treatment with vosoritide and 61 switched from placebo to vosoritide. AGV and body proportion ratio were analyzed to assess efficacy of vosoritide.
Results: In children randomized to receive vosoritide, the mean (standard deviation [SD]) cumulative AGV improved from 4.26 (1.54) cm/year at baseline (n=58) to 5.66 (0.82) cm/year at Week 104 (n=48) and was maintained at Week 156 (n=52) at 5.57 (0.76) cm/year. In children with available follow up at Week 182 (n=34) AGV was maintained at 5.45 (0.79) cm/year and Mean (SD) upper-to-lower body segment ratio improved with a change from baseline of -0.03 (0.11) at Week 52 to 0.13 (0.21) after 3.5 years on treatment. In children who switched from placebo to vosoritide after 52 weeks, cumulative AGV improved from 3.77 (1.29) cm/year at baseline (n=61) to 5.45 (1.35) cm/year at Week 104 (n=58) and was maintained at 5.43 (1.22) cm/year at Week 130 (n=42). Most adverse events were mild, and no discontinuations occurred due to AEs. One SAE of genu valgum was reported as related to vosoritide. There was no evidence of worsening body proportions, bone mineral density, bone age or worsening of spinal angles.
Conclusions: The effect of vosoritide administration on growth continues to be maintained up to 3.5 years in children with achondroplasia aged 5 to 18 years. The long-term safety profile remains favourable without any treatment-limiting adverse events.
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