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BioMarin's Voxzogo Meets Goal in Late-Stage Study for Hypochondroplasia
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Key Takeaways
BioMarin's Voxzogo boosted annualized growth velocity by 2.33 cm/yr versus placebo.
BMRN plans a U.S. filing in Q3 2026 to expand Voxzogo into hypochondroplasia.
Voxzogo faces new achondroplasia rivals as BioMarin targets a new growth opportunity.
Shares of BioMarin Pharmaceutical (BMRN - Free Report) were up 3% in pre-market trading today after the company reported positive top-line results from the phase III CANOPY-HCH-3 study evaluating Voxzogo for label expansion in hypochondroplasia.
The study met its primary endpoint, with patients treated with the drug for 52 weeks showing a statistically significant increase in annualized growth velocity (AGV) of 2.33 cm/yr compared to placebo. Voxzogo also showed significant improvement across several key secondary pre-specified endpoints, including standing height, height Z-score and arm span.
Voxzogo was approved by the FDA in 2021 to treat children of all ages with achondroplasia, the most common form of dwarfism. It is approved for similar indications in Europe, Japan and Australia.
Hypochondroplasia is a rare genetic skeletal growth disorder marked by short stature and impaired bone growth. The condition is generally considered a milder form of achondroplasia. Per BioMarin, there are currently no therapies approved by the FDA or the EMA for hypochondroplasia, highlighting a significant unmet medical need.
Data from the CANOPY-HCH-3 study will support regulatory submissions seeking label expansion for Voxzogo in hypochondroplasia. BioMarin expects to submit a regulatory filing in the United States in the third quarter of 2026, followed by submissions to the EMA and other global regulatory authorities. If approved, the label expansion could broaden the drug’s addressable market opportunity.
Since its launch, Voxzogo has seen rapid uptake, driven by strong prescription demand. BioMarin expects to generate $0.98-$1.03 billion from Voxzogo sales in 2026.
BMRN’s Stock Performance
Year to date, shares of BioMarin have lost nearly 16% compared with the industry’s 3% fall.
Image Source: Zacks Investment Research
BMRN’s Voxzogo Faces an Evolving Competitive Landscape
The achondroplasia treatment market is becoming increasingly competitive. In February 2026, the FDA approved Ascendis Pharma’s (ASND - Free Report) Yuviwel for achondroplasia, marking the first direct competitor to Voxzogo. Before this approval, Voxzogo was the only FDA-approved therapy for the condition. The approval of ASND’s Yuviwel was also a key factor behind BioMarin withdrawing its prior 2027 revenue guidance of approximately $4 billion.
Meanwhile, BridgeBio Pharma (BBIO - Free Report) is preparing to submit a regulatory filing to the FDA in the third quarter of 2026 for its investigational achondroplasia candidate, infigratinib.
Against this increasingly competitive backdrop, potential expansion of Voxzogo into hypochondroplasia could provide BioMarin with an additional growth avenue and help offset future competitive pressure in the achondroplasia market. With no FDA- or EMA-approved therapies currently available for hypochondroplasia, the company may also benefit from a potential first-mover advantage in the indication.
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BioMarin's Voxzogo Meets Goal in Late-Stage Study for Hypochondroplasia
Key Takeaways
Shares of BioMarin Pharmaceutical (BMRN - Free Report) were up 3% in pre-market trading today after the company reported positive top-line results from the phase III CANOPY-HCH-3 study evaluating Voxzogo for label expansion in hypochondroplasia.
The study met its primary endpoint, with patients treated with the drug for 52 weeks showing a statistically significant increase in annualized growth velocity (AGV) of 2.33 cm/yr compared to placebo. Voxzogo also showed significant improvement across several key secondary pre-specified endpoints, including standing height, height Z-score and arm span.
Voxzogo was approved by the FDA in 2021 to treat children of all ages with achondroplasia, the most common form of dwarfism. It is approved for similar indications in Europe, Japan and Australia.
Hypochondroplasia is a rare genetic skeletal growth disorder marked by short stature and impaired bone growth. The condition is generally considered a milder form of achondroplasia. Per BioMarin, there are currently no therapies approved by the FDA or the EMA for hypochondroplasia, highlighting a significant unmet medical need.
Data from the CANOPY-HCH-3 study will support regulatory submissions seeking label expansion for Voxzogo in hypochondroplasia. BioMarin expects to submit a regulatory filing in the United States in the third quarter of 2026, followed by submissions to the EMA and other global regulatory authorities. If approved, the label expansion could broaden the drug’s addressable market opportunity.
Since its launch, Voxzogo has seen rapid uptake, driven by strong prescription demand. BioMarin expects to generate $0.98-$1.03 billion from Voxzogo sales in 2026.
BMRN’s Stock Performance
Year to date, shares of BioMarin have lost nearly 16% compared with the industry’s 3% fall.
Image Source: Zacks Investment Research
BMRN’s Voxzogo Faces an Evolving Competitive Landscape
The achondroplasia treatment market is becoming increasingly competitive. In February 2026, the FDA approved Ascendis Pharma’s (ASND - Free Report) Yuviwel for achondroplasia, marking the first direct competitor to Voxzogo. Before this approval, Voxzogo was the only FDA-approved therapy for the condition. The approval of ASND’s Yuviwel was also a key factor behind BioMarin withdrawing its prior 2027 revenue guidance of approximately $4 billion.
Meanwhile, BridgeBio Pharma (BBIO - Free Report) is preparing to submit a regulatory filing to the FDA in the third quarter of 2026 for its investigational achondroplasia candidate, infigratinib.
Against this increasingly competitive backdrop, potential expansion of Voxzogo into hypochondroplasia could provide BioMarin with an additional growth avenue and help offset future competitive pressure in the achondroplasia market. With no FDA- or EMA-approved therapies currently available for hypochondroplasia, the company may also benefit from a potential first-mover advantage in the indication.
BioMarin Pharmaceutical Inc. Price
BioMarin Pharmaceutical Inc. price | BioMarin Pharmaceutical Inc. Quote
BMRN’s Zacks Rank
The stock currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.