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FDA Accepts BMRN's sNDA Seeking Full Approval for Voxzogo
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Key Takeaways
BMRN's sNDA seeking full Voxzogo approval in achondroplasia was accepted for FDA review.
BMRN received accelerated FDA approval for Voxzogo in 2021 and now seeks traditional approval.
FDA decision is due on Feb. 28, 2027; Voxzogo may become the first achondroplasia drug to get traditional nod.
BioMarin Pharmaceutical (BMRN - Free Report) announced that the FDA has accepted the supplemental new drug application (sNDA) seeking full approval for Voxzogo (vosoritide) in children with achondroplasia, the most common form of dwarfism.
With the FDA accepting the sNDA for review, a decision from the regulatory body is expected on Feb. 28, 2027.
Voxzogo received an accelerated approval from the FDA in 2021 to treat children of all ages with achondroplasia. The drug is approved for similar indications in Europe, Japan and Australia.
The latest sNDA, which seeks to convert Voxzogo’s accelerated approval into full/traditional approval, is based on long-term safety and efficacy data from three ongoing studies, comprising adult height and additional clinical outcomes beyond linear growth, including body proportionality and arm span, evaluated over long-term follow-up.
Per management, if approved, Voxzogo would become the first therapy for achondroplasia to convert from accelerated to traditional approval based on comprehensive clinical data, including adult height outcomes and other measures assessed over an extended follow-up period.
BMRN’s Stock Performance
Year to date, shares of BioMarin have lost 0.4% against the industry’s increase of 4.1%.
Image Source: Zacks Investment Research
BMRN Banks on Voxzogo Amid Growing Rivalry
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.
BioMarin is also evaluating Voxzogo in the phase III CANOPY-HCH-3 study for a potential second indication, hypochondroplasia, which is generally considered to be a milder form of achondroplasia. In May 2026, the company reported positive data from this study, which achieved its primary and key secondary endpoints. Based on these results, BioMarin expects to submit a regulatory filing with the FDA in the third quarter of 2026.
If approved, the label expansion could broaden the drug’s addressable market opportunity.
However, 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 the approval of ASND’s Yuviwel, Voxzogo was the only FDA-approved therapy for the condition.
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. If approved, BBIO expects a potential launch for infigratinib in early to mid-2027.
Amid increasing competition, the successful development and potential label expansion of Voxzogo into additional indications could meaningfully strengthen BioMarin's long-term growth prospects.
BMRN’s Zacks Rank
BioMarin currently carries a Zacks Rank #4 (Sell).
View the Zacks #1 Rank List
Image: Bigstock
FDA Accepts BMRN's sNDA Seeking Full Approval for Voxzogo
Key Takeaways
BioMarin Pharmaceutical (BMRN - Free Report) announced that the FDA has accepted the supplemental new drug application (sNDA) seeking full approval for Voxzogo (vosoritide) in children with achondroplasia, the most common form of dwarfism.
With the FDA accepting the sNDA for review, a decision from the regulatory body is expected on Feb. 28, 2027.
Voxzogo received an accelerated approval from the FDA in 2021 to treat children of all ages with achondroplasia. The drug is approved for similar indications in Europe, Japan and Australia.
The latest sNDA, which seeks to convert Voxzogo’s accelerated approval into full/traditional approval, is based on long-term safety and efficacy data from three ongoing studies, comprising adult height and additional clinical outcomes beyond linear growth, including body proportionality and arm span, evaluated over long-term follow-up.
Per management, if approved, Voxzogo would become the first therapy for achondroplasia to convert from accelerated to traditional approval based on comprehensive clinical data, including adult height outcomes and other measures assessed over an extended follow-up period.
BMRN’s Stock Performance
Year to date, shares of BioMarin have lost 0.4% against the industry’s increase of 4.1%.
Image Source: Zacks Investment Research
BMRN Banks on Voxzogo Amid Growing Rivalry
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.
BioMarin is also evaluating Voxzogo in the phase III CANOPY-HCH-3 study for a potential second indication, hypochondroplasia, which is generally considered to be a milder form of achondroplasia. In May 2026, the company reported positive data from this study, which achieved its primary and key secondary endpoints. Based on these results, BioMarin expects to submit a regulatory filing with the FDA in the third quarter of 2026.
If approved, the label expansion could broaden the drug’s addressable market opportunity.
However, 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 the approval of ASND’s Yuviwel, Voxzogo was the only FDA-approved therapy for the condition.
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. If approved, BBIO expects a potential launch for infigratinib in early to mid-2027.
Amid increasing competition, the successful development and potential label expansion of Voxzogo into additional indications could meaningfully strengthen BioMarin's long-term growth prospects.
BMRN’s Zacks Rank
BioMarin currently carries a Zacks Rank #4 (Sell).
You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.