BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), a pharmaceutical company with exceptional financial health and a perfect Piotroski Score according to InvestingPro, revealed new data indicating that VOXZOGO (vosoritide) may have a significant impact on tibial bowing in children with achondroplasia, a common cause of pain and impaired function in this population. These findings were presented at recent endocrinology meetings in Copenhagen and Maryland.
An analysis from the VOXZOGO Phase 2 CANOPY clinical studies showed that children with achondroplasia who received VOXZOGO experienced a significant reduction in tibial bowing compared to those given a placebo. With an impressive gross profit margin of 81% and strong revenue growth of 19.4% over the last twelve months, BioMarin continues to demonstrate robust commercial execution. Notably, these improvements were sustained over several years of treatment. Michael Hughes of Little People of America emphasized the importance of addressing outcomes beyond height, such as tibial bowing, which can require surgery for some children.
Dr. Klane White, a pediatric orthopedic surgeon, noted the meaningful improvements in proportionality, quality of life, and tibial bowing observed in children treated with VOXZOGO. Additionally, new modeling data suggest that early and continuous treatment with VOXZOGO could lead to an increase in final adult height by up to 26.4 cm in boys and 21.7 cm in girls compared to untreated children.
Furthermore, research on hypochondroplasia, a form of skeletal dysplasia, showed that individuals with the condition had higher rates of comorbidities, surgeries, and doctor visits. BioMarin’s Dr. Greg Friberg highlighted the significance of early diagnosis and intervention.
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