The company’s data includes post-hoc analysis suggesting that VOXZOGO (vosoritide) positively influences the growth of the foramen magnum and craniocervical junction. According to Greg Friberg, M.D., BioMarin’s executive vice president and chief research officer, these results emphasize the clinical value of early intervention for conditions where skull anatomy often leads to serious complications like cervicomedullary compression.
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BioMarin unveils new clinical data on VOXZOGO for skeletal dysplasias
BioMarin Pharmaceutical is presenting findings from 13 studies at the American Society for Bone and Mineral Research annual meeting in Boston, highlighting the impact of its drug VOXZOGO on skull growth in children with achondroplasia and its potential to become the first targeted therapy for hypochondroplasia.
Beyond current applications, BioMarin is actively expanding its footprint in rare skeletal disorders. The firm has submitted a supplemental New Drug Application to the FDA for the treatment of hypochondroplasia, aiming for a potential market launch by 2027. The research presentations in Boston also cover broader pipeline developments, including the investigational long-acting medicine BMN 333 and Phase 3 trial results for BMN 401 in patients with ENPP1 deficiency. These efforts represent a push to address unmet needs in skeletal dysplasias through both existing therapies and next-generation candidates.
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