BioMarin Reports Positive Voxzogo Trial Results
BioMarin Pharmaceutical (NASDAQ:BMRN) on March 12 reported positive clinical and real-world results for Voxzogo (vosoritide) in children with achondroplasia, showing sustained multi-year height gains and improved body proportionality, especially when treatment begins before age two. On March 16 Wells Fargo kept an Overweight rating with a $75 target despite discontinuing three Phase 2 Voxzogo studies due to slipped-capital-femoral-epiphysis; data will be presented at ACMG 2026.
Key Points
- 1Reports positive multi‑year Voxzogo growth and proportionality improvements in achondroplasia, strongest when treatment begins before age two.
- 2Highlights safety signals that led to discontinuation of three Phase 2 Voxzogo studies due to slipped-capital-femoral-epiphysis.
- 3Wells Fargo maintains Overweight $75 target and views shares as buying opportunity amid manageable indication-specific risk.
Scoring Rationale
Solid company update with positive clinical and real-world data and analyst backing. Scored moderate for novelty and scope (rare-disease niche) with high credibility from company and Wells Fargo; reduced slightly for limited technical detail and promotional content.
Sources
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