BioMarin's VOXZOGO Shows Positive Results in Real-World Studies

A report highlights BioMarin Pharmaceutical's promising results for VOXZOGO in treating children with achondroplasia, showing significant height increases over 12 and 24-month periods, potentially impacting investor sentiment positively.

Date: 17-11-2024

AI Rating: 7

BioMarin Pharmaceutical Inc. (BMRN) provided comprehensive insights into the efficacy of its drug VOXZOGO, particularly in the treatment of achondroplasia among children. The report highlighted several key findings:

Adequate Height Increase: Children aged around 6.12 years, treated with VOXZOGO demonstrated a significant increase in height over 12 months with an average increase of 6.36 cm, and a further average increase of 11.86 cm over 24 months among those treated for the longer period.
Consistency with Prior Studies: The longitudinal data from the European CrescNet registry was consistent with previously reported clinical trial results, reinforcing the drug's credibility and effectiveness.
Improved Z-scores: The height Z-scores improved significantly, indicating that treated children are experiencing growth comparable to or exceeding that of untreated peers.
Safety and Effectiveness Monitoring: The report mentions no discontinuations from the study, indicating a favorable safety profile alongside efficacy.

Overall, the results from the studies indicate a clear positive trend in the efficacy of VOXZOGO, specifically in enhancing growth in children with achondroplasia. Such results can enhance BioMarin's reputation and may lead to increased investor confidence, potentially upwardly influencing the stock prices.

Stocks: BMRN