BioMarin Pharmaceuticals Inc. (NASDAQ:BMRN) stock is trading higher on Thursday as the company is celebrating positive results from its Phase 3 CANOPY-HCH-3 study for VOXZOGO (vosoritide) in children with hypochondroplasia.
Hypochondroplasia is a form of genetic, short-limbed dwarfism.
The study met its primary endpoint, showing a statistically significant improvement in annualized growth velocity compared to placebo, which has excited investors and analysts alike.
VOXZOGO Study Meets Primary Endpoint
The CANOPY-HCH-3 study demonstrated a 2.33 cm/year increase in annualized growth velocity at week 52, significantly exceeding expectations.
With plans for a supplemental New Drug Application submission to the FDA in the third quarter of 2026, these results could pave the way for new treatment options in a market currently lacking approved therapies for hypochondroplasia.
Additionally, significant improvements in arm span were demonstrated (p=0.004), a key prespecified secondary endpoint in the study.
“For the first time, a pivotal Phase 3 study in hypochondroplasia has demonstrated impressive gains in growth, including improvements in arm span, highlighting VOXZOGO’s potential to fundamentally change the treatment landscape for this condition,” said Greg Friberg, EVP and Chief Research & Development Officer at BioMarin
Analyst Consensus & Recent Actions: The stock carries a Buy rating with an average price target of $87.47. Recent analyst moves include:
- B of A Securities: Buy (Lowers Target to $80.00) (May 19)
- Citigroup: Initiated with Buy (Target $75.00) (May 19)
- HC Wainwright & Co.: Neutral (Lowers Target to $50.00) (May 18)
How BioMarin Ranks On Value And Momentum
Below is the Benzinga Edge scorecard for BioMarin Pharmaceuticals, highlighting its strengths and weaknesses compared to the broader market:
- Value: 67.93 — The stock is performing moderately well in terms of value metrics.
- Momentum: 12.86 — Stock is underperforming the broader market.
The Verdict: BioMarin Pharmaceuticals’ Benzinga Edge signal reveals a mixed profile, with solid value metrics but weak momentum indicators. This suggests that while the company has a fair valuation, its current momentum may not support a sustained upward trend in the near term.
BMRN Price Action: BioMarin Pharmaceutical shares were up 8.05% at $54.24 at the time of publication on Thursday, according to Benzinga Pro data.
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