BioMarin Presents New Data Advancing Understanding of Achondroplasia and Hypochondroplasia Treatment at the American Society for Bone and Mineral Research Annual Meeting
PR Newswire•09/10/2026•09:05 ET
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Key Highlights
- ➤BioMarin will present data from 13 studies at ASBMR 2026.
- ➤VOXZOGO analysis evaluates foramen magnum and craniocervical junction growth in children with achondroplasia.
- ➤BioMarin submitted an FDA sNDA seeking VOXZOGO approval for hypochondroplasia.
- ➤Potential 2027 launch would make VOXZOGO hypochondroplasia’s first targeted therapy if approved.
- ➤FDA PDUFA target date for VOXZOGO achondroplasia sNDA is Feb. 28, 2027.
Expert Statements
Greg Friberg, Executive Vice President and Chief Research & Development Officer at BioMarin
“These findings provide important new insight into the potential effect of VOXZOGO on growth-related dimensions of the foramen magnum and craniocervical junction, regions of the skull implicated in cervicomedullary compression and serious complications in children with achondroplasia.”
Greg Friberg, Executive Vice President and Chief Research & Development Officer at BioMarin
“The results also underscore the potential VOXZOGO treatment may offer when started as early as possible, when it can have the greatest impact.”
Greg Friberg, Executive Vice President and Chief Research & Development Officer at BioMarin
“Together with the additional data we’re presenting on bone strength, limb alignment, and our emerging work in hypochondroplasia and next-generation medicines such as BMN 333, these findings reflect the depth and breadth of our commitment to understanding and improving outcomes for children living with skeletal dysplasias.”
BioMarin Presents New Data Advancing Understanding of Achondroplasia and Hypochondroplasia Treatment at the American Society for Bone and Mineral Research Annual Meeting PR Newswire
New analysis showcases impact of VOXZOGO(®) (vosoritide) in foramen magnum and craniocervical junction growth in children with achondroplasia, including infants
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