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BioMarin Stops Voxzogo Development for Noonan Syndrome
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Key Takeaways
BMRN stops Voxzogo development for Noonan syndrome, citing study feasibility and the treatment landscape.
Voxzogo generated $472 million in first-half 2026 sales, up 9% year over year on strong demand.
BMRN expects $1.00-$1.05 billion in 2026 Voxzogo sales, with hypochondroplasia approval targeted for 2027.
BioMarin Pharmaceutical (BMRN - Free Report) announced that it is discontinuing the development of Voxzogo (vosoritide) for children with Noonan syndrome, a rare genetic condition that can affect growth and development in multiple parts of the body.
This announcement came via an SEC filing on Tuesday, which attributed the decision to the study’s feasibility and the current treatment landscape. The company clarified that the decision was not related to the safety or efficacy of Voxzogo.
In March, BioMarin ended development of the drug for three other short stature indications, Turner Syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency. Unlike the Noonan syndrome, these studies were stopped due to the occurrence of an adverse event called slipped capital femoral epiphysis (SCFE) in study participants across two investigator-sponsored studies. At the time, BioMarin stated that it would continue studying Voxzogo for Noonan syndrome.
Voxzogo was approved by the FDA in 2021 as the first approved treatment for achondroplasia, the most common form of dwarfism. The drug is a C-type natriuretic peptide (CNP) analog administered once daily as a subcutaneous injection. CNP is a naturally occurring signaling molecule that plays a role in regulating bone growth.
BMRN Stock Performance
Year to date, shares of the company have gained 8.5% compared with the industry’s 5.5% growth.
Image Source: Zacks Investment Research
Voxzogo: A Key Driver of BioMarin’s Topline
Voxzogo has seen rapid uptake since launch. The drug has generated $472 million in sales during the first half of 2026, up 9% year over year, driven by strong prescription demand. Voxzogo is currently BioMarin's largest product by net product revenues.
Management expects sales in the second half of the year to exceed those in the first half. It expects to generate $1.00-$1.05 billion from Voxzogo sales in 2026, putting the drug on track to achieve blockbuster status.
Despite the discontinuation of development for Noonan syndrome, Voxzogo retains other potential label-expansion opportunities. BioMarin submitted a regulatory filing seeking label expansion for hypochondroplasia, which is generally considered to be a milder form of achondroplasia. This filing is supported by data from the phase III CANOPY-HCH-3 study, which met its primary and key secondary endpoints. If approved, BioMarin expects a potential launch in 2027.
BioMarin is also advancing Voxzogo in a label expansion study for another short-stature pathway condition called idiopathic short stature (without ACAN-deficiency).
BioMarin Confronts a Changing Competitive Landscape for Voxzogo
Voxzogo faces increasing competition from Ascendis Pharma (ASND - Free Report) , whose Yuviwel (navepegritide) received FDA approval for achondroplasia in February 2026. Like Voxzogo, Yuviwel is a CNP therapy but is administered once weekly. The ASND drug is designed to provide continuous exposure to active CNP, potentially offering a more convenient dosing profile than the BMRN drug.
BioMarin had previously alleged that Yuviwel infringed its intellectual property covering CNP technology, triggering patent disputes across the United States, Brazil, Denmark, Germany and South Korea, including before the U.S. International Trade Commission. Recently, the two companies entered into a settlement that meaningfully changed the economics of competition.
Per the settlement terms, BioMarin will grant Ascendis a non-exclusive, worldwide license to certain patents covering Yuviwel and related products, allowing Ascendis to research, develop, manufacture and commercialize the drug across current and potential indications.
In return, Ascendis will pay BioMarin royalties equal to 20% of annual net sales in the United States and 18% in the European Union, Brazil and South Korea, from the first commercial sale in each territory through May 20, 2030. The U.S. royalty is also applied retroactively to Yuviwel's first commercial sale. All pending lawsuits between the two companies will also be dismissed.
The settlement provides BioMarin with a meaningful royalty stream from a product that competes directly with Voxzogo. However, the royalties do not eliminate the competitive risk. If Yuviwel takes substantial market share from Voxzogo, BioMarin could lose Voxzogo revenues while gaining royalty income on Ascendis' sales. The net economic impact will therefore depend on the pace of Yuviwel adoption, the extent to which it expands the overall treated population and how much of its sales come at Voxzogo's expense.
BioMarin’s Voxzogo could have another potential competitor as early as next year. Recently, BridgeBio Pharma (BBIO - Free Report) submitted a regulatory filing with the FDA for infigratinib, an FGFR3 inhibitor that could become the first approved oral therapy for achondroplasia. The company expects a U.S. launch in mid-2027, subject to approval.
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BioMarin Stops Voxzogo Development for Noonan Syndrome
Key Takeaways
BioMarin Pharmaceutical (BMRN - Free Report) announced that it is discontinuing the development of Voxzogo (vosoritide) for children with Noonan syndrome, a rare genetic condition that can affect growth and development in multiple parts of the body.
This announcement came via an SEC filing on Tuesday, which attributed the decision to the study’s feasibility and the current treatment landscape. The company clarified that the decision was not related to the safety or efficacy of Voxzogo.
In March, BioMarin ended development of the drug for three other short stature indications, Turner Syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency. Unlike the Noonan syndrome, these studies were stopped due to the occurrence of an adverse event called slipped capital femoral epiphysis (SCFE) in study participants across two investigator-sponsored studies. At the time, BioMarin stated that it would continue studying Voxzogo for Noonan syndrome.
Voxzogo was approved by the FDA in 2021 as the first approved treatment for achondroplasia, the most common form of dwarfism. The drug is a C-type natriuretic peptide (CNP) analog administered once daily as a subcutaneous injection. CNP is a naturally occurring signaling molecule that plays a role in regulating bone growth.
BMRN Stock Performance
Year to date, shares of the company have gained 8.5% compared with the industry’s 5.5% growth.
Image Source: Zacks Investment Research
Voxzogo: A Key Driver of BioMarin’s Topline
Voxzogo has seen rapid uptake since launch. The drug has generated $472 million in sales during the first half of 2026, up 9% year over year, driven by strong prescription demand. Voxzogo is currently BioMarin's largest product by net product revenues.
Management expects sales in the second half of the year to exceed those in the first half. It expects to generate $1.00-$1.05 billion from Voxzogo sales in 2026, putting the drug on track to achieve blockbuster status.
Despite the discontinuation of development for Noonan syndrome, Voxzogo retains other potential label-expansion opportunities. BioMarin submitted a regulatory filing seeking label expansion for hypochondroplasia, which is generally considered to be a milder form of achondroplasia. This filing is supported by data from the phase III CANOPY-HCH-3 study, which met its primary and key secondary endpoints. If approved, BioMarin expects a potential launch in 2027.
BioMarin is also advancing Voxzogo in a label expansion study for another short-stature pathway condition called idiopathic short stature (without ACAN-deficiency).
BioMarin Confronts a Changing Competitive Landscape for Voxzogo
Voxzogo faces increasing competition from Ascendis Pharma (ASND - Free Report) , whose Yuviwel (navepegritide) received FDA approval for achondroplasia in February 2026. Like Voxzogo, Yuviwel is a CNP therapy but is administered once weekly. The ASND drug is designed to provide continuous exposure to active CNP, potentially offering a more convenient dosing profile than the BMRN drug.
BioMarin had previously alleged that Yuviwel infringed its intellectual property covering CNP technology, triggering patent disputes across the United States, Brazil, Denmark, Germany and South Korea, including before the U.S. International Trade Commission. Recently, the two companies entered into a settlement that meaningfully changed the economics of competition.
Per the settlement terms, BioMarin will grant Ascendis a non-exclusive, worldwide license to certain patents covering Yuviwel and related products, allowing Ascendis to research, develop, manufacture and commercialize the drug across current and potential indications.
In return, Ascendis will pay BioMarin royalties equal to 20% of annual net sales in the United States and 18% in the European Union, Brazil and South Korea, from the first commercial sale in each territory through May 20, 2030. The U.S. royalty is also applied retroactively to Yuviwel's first commercial sale. All pending lawsuits between the two companies will also be dismissed.
The settlement provides BioMarin with a meaningful royalty stream from a product that competes directly with Voxzogo. However, the royalties do not eliminate the competitive risk. If Yuviwel takes substantial market share from Voxzogo, BioMarin could lose Voxzogo revenues while gaining royalty income on Ascendis' sales. The net economic impact will therefore depend on the pace of Yuviwel adoption, the extent to which it expands the overall treated population and how much of its sales come at Voxzogo's expense.
BioMarin’s Voxzogo could have another potential competitor as early as next year. Recently, BridgeBio Pharma (BBIO - Free Report) submitted a regulatory filing with the FDA for infigratinib, an FGFR3 inhibitor that could become the first approved oral therapy for achondroplasia. The company expects a U.S. launch in mid-2027, subject to approval.
BioMarin Pharmaceutical Inc. Price
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BMRN’s Zacks Rank
BioMarin currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.