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New Data for BioMarin's VOXZOGO® (vosoritide) for Multiple Growth-Related Conditions in Children Presented at Pediatric Endocrine Society (PES) Annual Meeting
Investigator-Sponsored Phase 2 Study Demonstrated Positive Results in Children with Noonan Syndrome, Idiopathic Short Stature and Other Growth-Related
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"Investigator-Sponsored Phase 2 Study Demonstrated Positive Results in Children with Noonan Syndrome, Idiopathic Short Stature and Other Growth-Related Conditions\nBioMarin-Sponsored Phase 3 Data Show Notable Mean Annualized Growth Velocity Increases in Children with Achondroplasia Who Started Treatment During Adolescence\nSAN RAFAEL, Calif., May 4, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that positive new data supporting the safety and efficacy of VOXZOGO® (vosoritide) in children with achondroplasia, as well as positive data for investigational uses in growth-related conditions, including idiopathic short stature (ISS) and Noonan syndrome, were presented at the 2024 Pediatric Endocrine Society (PES) Annual Meeting in Chicago, May 2-5, 2024. Researchers also presented results from additional studies highlighting the medicine's efficacy and impact on health-related quality of life (HRQoL) in children with achondroplasia.\n\n \n \n \n \n \n \n\n \nNew Results in ISS and Noonan Syndrome \nPositive results were presented from an investigator-sponsored Phase 2 study of VOXZOGO in children 3-11 years old with several genetic growth-related conditions, including Noonan syndrome and genetic mutations associated with ISS, such as aggrecan (ACAN) deficiency, heterozygous NPR2 mutations and neurofibromatosis 1 (NF1). Results demonstrated marked improvement in annualized growth velocity (AGV) and height standard deviation (SD) across all the conditions studied. For the eight children who completed 12 months of treatment, mean AGV increased from a baseline of 3.7 cm/year to 8.5 cm/year and mean height SD changed from -3.6 SD to -2.9 SD. Of these eight children, three with NPR2 mutations had increases of 3.3, 4.8 and 9.3 cm/year; three with Noonan syndrome had increases of 3.0, 4.0 and 5.8 cm/year; and two with ACAN mutations had increases of 3.2 and 5.4 cm/year in their AGVs over baseline. Safety results were consistent with the well-characterized safety profile of VOXZOGO.\n\"For the first time, we are seeing evidence that VOXZOGO could positively impact growth in several different skeletal dysplasias and growth-related conditions beyond achondroplasia, including in children with Noonan syndrome and ACAN deficiency that was presented today, as well as in hypochondroplasia that was recently shared at ACMG,\...