BioMarin Presents Groundbreaking Data on Bone Health Improvement at ASBMR 2025
BioMarin Pharmaceutical Inc. made significant strides in bone health research by presenting new findings at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting 2025 in Seattle. The company showcased data from 14 studies which emphasized the benefits of its treatment, VOXZOGO (vosoritide), for children suffering from achondroplasia and conditions related to it.
In one of the pivotal studies, a Phase 2 clinical trial, VOXZOGO demonstrated noticeable improvements in spinal morphology among young children afflicted with achondroplasia. Notably, children aged 5 and under who received the treatment for 52 weeks showed considerable anatomical benefits compared to those who were administered a placebo. Key metrics included an increase in interpedicular distance and spinal canal width across all five vertebrae in the lower back (L1 to L5). Furthermore, a greater percentage of these children, 57%, exhibited a reduction in thoracolumbar kyphosis, a condition characterized by an excessive curvature of the spine, in contrast to just 33% in the placebo group.
Medical professionals recognize that achondroplasia not only affects physical stature but can also lead to serious complications such as spinal stenosis, which can manifest severe symptoms including pain, mobility challenges, and other debilitating issues. Speaking on the recent findings, Dr. Greg Friberg, BioMarin's Executive Vice President and Chief Research Development Officer, underscored that VOXZOGO is the first FDA-approved medication backed by data that shows a positive impact on spinal morphology. This could lead to significant clinical improvements for children affected by achaondoplasia and the accompanying conditions.
Importantly, the benefits of VOXZOGO were not confined to younger patients. Results from the open-label, Phase 3 CANOPY trial extension indicated that adolescents who began treatment after entering puberty continued to experience growth benefits. For instance, male participants experienced a growth of 24.62 cm from the age of puberty onset until they reached 18 years, compared to only 17.07 cm for those untreated. Female participants similarly showed an increase in height from their mean age of puberty onset, illustrating VOXZOGO's efficacy across different developmental stages.
Often, the challenges associated with achondroplasia do not stop in childhood. Many symptoms can persist or worsen into adulthood, necessitating ongoing care and innovative treatments. BioMarin's research underscores the critical nature of addressing these unmet needs, particularly through their exploration of conditions like ENPP1 deficiency—a rare genetic condition leading to dire health consequences. BioMarin plans to introduce BMN 401, an enzyme therapy, to address this deficiency, with pivotal data anticipated in early 2026.
The presentation of these findings is a part of BioMarin's broader commitment to addressing rare genetic disorders through transformative therapies. As the first approved treatment aimed at improving both growth and spinal health in children with achondroplasia, VOXZOGO is set to pave the way for future advancements in pediatric bone health management.
In conclusion, BioMarin's presentations at ASBMR 2025 deepen the understanding of achondroplasia and its ramifications, while also propelling forward the conversation on treatment options that can greatly enhance the quality of life for affected children. For parents considering VOXZOGO, it’s essential to discuss treatment plans with healthcare providers and understand the comprehensive support offered by BioMarin’s RareConnections program to ensure optimal outcomes.
In one of the pivotal studies, a Phase 2 clinical trial, VOXZOGO demonstrated noticeable improvements in spinal morphology among young children afflicted with achondroplasia. Notably, children aged 5 and under who received the treatment for 52 weeks showed considerable anatomical benefits compared to those who were administered a placebo. Key metrics included an increase in interpedicular distance and spinal canal width across all five vertebrae in the lower back (L1 to L5). Furthermore, a greater percentage of these children, 57%, exhibited a reduction in thoracolumbar kyphosis, a condition characterized by an excessive curvature of the spine, in contrast to just 33% in the placebo group.
Medical professionals recognize that achondroplasia not only affects physical stature but can also lead to serious complications such as spinal stenosis, which can manifest severe symptoms including pain, mobility challenges, and other debilitating issues. Speaking on the recent findings, Dr. Greg Friberg, BioMarin's Executive Vice President and Chief Research Development Officer, underscored that VOXZOGO is the first FDA-approved medication backed by data that shows a positive impact on spinal morphology. This could lead to significant clinical improvements for children affected by achaondoplasia and the accompanying conditions.
Importantly, the benefits of VOXZOGO were not confined to younger patients. Results from the open-label, Phase 3 CANOPY trial extension indicated that adolescents who began treatment after entering puberty continued to experience growth benefits. For instance, male participants experienced a growth of 24.62 cm from the age of puberty onset until they reached 18 years, compared to only 17.07 cm for those untreated. Female participants similarly showed an increase in height from their mean age of puberty onset, illustrating VOXZOGO's efficacy across different developmental stages.
Often, the challenges associated with achondroplasia do not stop in childhood. Many symptoms can persist or worsen into adulthood, necessitating ongoing care and innovative treatments. BioMarin's research underscores the critical nature of addressing these unmet needs, particularly through their exploration of conditions like ENPP1 deficiency—a rare genetic condition leading to dire health consequences. BioMarin plans to introduce BMN 401, an enzyme therapy, to address this deficiency, with pivotal data anticipated in early 2026.
The presentation of these findings is a part of BioMarin's broader commitment to addressing rare genetic disorders through transformative therapies. As the first approved treatment aimed at improving both growth and spinal health in children with achondroplasia, VOXZOGO is set to pave the way for future advancements in pediatric bone health management.
In conclusion, BioMarin's presentations at ASBMR 2025 deepen the understanding of achondroplasia and its ramifications, while also propelling forward the conversation on treatment options that can greatly enhance the quality of life for affected children. For parents considering VOXZOGO, it’s essential to discuss treatment plans with healthcare providers and understand the comprehensive support offered by BioMarin’s RareConnections program to ensure optimal outcomes.
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