VOXZOGO Shows Significant Efficacy in Children
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 6 hours ago
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Should l Buy BMRN?
Source: Newsfilter
- Early Treatment Impact: New data indicates that children starting VOXZOGO treatment before age 2 experienced an average height increase of 4.7 cm over four years (p=0.0002), underscoring the significant impact of early intervention on skeletal growth and reinforcing VOXZOGO's status as the only approved treatment.
- Proportionality Improvement: Among children treated with VOXZOGO, the upper-to-lower body segment ratio (ULBR) improved by 0.33 (p=0.0005) in the first year and reached 0.53 (p<0.0001) by year four, indicating that early treatment enhances not only height but also overall body proportionality, which is crucial for health.
- Global Evidence Support: More than 5,000 children across over 50 countries have received VOXZOGO, with Japanese children showing an average height increase of 9.91 cm at 12 months and 15.62 cm at 24 months, further validating the efficacy and safety of VOXZOGO.
- Clinical Research Findings: BioMarin will present these research results at the 2026 American College of Medical Genetics and Genomics Annual Meeting, emphasizing the importance of VOXZOGO in children's skeletal development and facilitating communication between families and healthcare providers.
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Analyst Views on BMRN
Wall Street analysts forecast BMRN stock price to rise
19 Analyst Rating
14 Buy
5 Hold
0 Sell
Moderate Buy
Current: 60.320
Low
60.00
Averages
87.35
High
120.00
Current: 60.320
Low
60.00
Averages
87.35
High
120.00
About BMRN
BioMarin Pharmaceutical Inc. is a global biotechnology company engaged in the development of genetic discovery into medicines that make an impact on the life of each patient. The Company has eight commercial therapies and a clinical and preclinical pipeline. Its commercial products include Vimizim (elosulfase alpha), Naglazyme (galsulfase), Palynziq (pegvaliase-pqpz), Brineura (cerliponase alfa), Aldurazyme (laronidase), Roctavian (valoctocogene roxaparvovec), Kuvan (sapropterin dihydrochloride), and Voxzogo (vosoritide). Vimizim is an enzyme replacement therapy for the treatment of MPS IVA, a lysosomal storage disorder. The Roctavian is an adeno-associated virus (AAV5) vector gene therapy designed to restore factor VIII plasma concentrations in patients with severe hemophilia A. Its key clinical programs include Vosoritide, BMN 333, BMN 390, BMN 351, BMN 349, and INZ-701. Its late-stage enzyme replacement therapy, INZ-701, is being developed for the treatment of ENPP1 deficiency.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
VOXZOGO Treatment Shows Significant Improvements in Children's Skeletal Growth
- Early Treatment Impact: New data indicates that children starting VOXZOGO treatment before age 2 experienced an average height increase of 4.7 cm (p=0.0002) after four years, underscoring the critical importance of early intervention for skeletal growth.
- Proportionality Improvements: Among children treated with VOXZOGO, the upper-to-lower body segment ratio (ULBR) improved by 0.33 (p=0.0005) in the first year and further improved to 0.53 (p<0.0001) by year four, demonstrating the drug's effectiveness in enhancing body proportionality.
- Global Clinical Evidence: More than 5,000 children across over 50 countries have received VOXZOGO, with Japanese children showing an average height increase of 9.91 cm at 12 months and 15.62 cm at 24 months, further validating the drug's efficacy and safety.
- Long-Term Safety Studies: BioMarin will present findings on VOXZOGO's long-term safety and growth outcomes at the 2026 ACMG Annual Clinical Genetics Meeting, emphasizing its significant role in children's skeletal development and aiding communication between physicians and families.
See More
VOXZOGO Shows Significant Efficacy in Children
- Early Treatment Impact: New data indicates that children starting VOXZOGO treatment before age 2 experienced an average height increase of 4.7 cm over four years (p=0.0002), underscoring the significant impact of early intervention on skeletal growth and reinforcing VOXZOGO's status as the only approved treatment.
- Proportionality Improvement: Among children treated with VOXZOGO, the upper-to-lower body segment ratio (ULBR) improved by 0.33 (p=0.0005) in the first year and reached 0.53 (p<0.0001) by year four, indicating that early treatment enhances not only height but also overall body proportionality, which is crucial for health.
- Global Evidence Support: More than 5,000 children across over 50 countries have received VOXZOGO, with Japanese children showing an average height increase of 9.91 cm at 12 months and 15.62 cm at 24 months, further validating the efficacy and safety of VOXZOGO.
- Clinical Research Findings: BioMarin will present these research results at the 2026 American College of Medical Genetics and Genomics Annual Meeting, emphasizing the importance of VOXZOGO in children's skeletal development and facilitating communication between families and healthcare providers.
See More
BridgeBio's Promising Biotech Pipeline Highlighted by William Blair
- Unique Market Position: Analyst John Boyle highlights BridgeBio's unique position with the accelerating launch of Attruby and near-term NDA submissions, setting the stage for late-2026/early-2027 launches in the rare genetic disease treatment market.
- Product Competitive Edge: Market surveys indicate that prescribers view both Attruby and Amvuttra as superior to Pfizer's Tafamidis, which has approximately $6 billion in annual sales, suggesting that BridgeBio's products are gaining traction in clinical applications.
- Sales Forecast and Risks: The analyst models U.S. sales of Attruby reaching $3.4 billion by 2035, while acknowledging potential generic entry risks in late 2028; however, Attruby's strong TTR stabilization and simple twice-daily dosing could justify premium pricing.
- Other Market Opportunities: BridgeBio also sees significant market opportunities with other rare disease drugs, particularly infigratinib for achondroplasia and encaleret for autosomal dominant hypocalcemia type 1, with the latter achieving its primary endpoint in the CALIBRATE Phase 3 study in October 2025, indicating strong future prospects.
See More
BioMarin Pharmaceutical Outlines Strategic Refresh and Market Outlook
- Strategic Update: BioMarin Pharmaceutical emphasized its strategic refresh at the TD Cowen conference, particularly through the Inozyme deal and the pending Amicus acquisition, aiming to expand enzyme therapy scale across approximately 80 countries, thereby enhancing its competitive position in the rare disease market.
- Revenue Outlook: Despite anticipating a roughly 3% revenue headwind in 2026 due to the removal of ROCTAVIAN and Kuvan revenue declines, BioMarin expects core enzyme and skeletal businesses to grow at mid-to-high single digits, targeting a 40% operating margin by 2026.
- Competitive Dynamics: BioMarin anticipates new FDA-approved competition for Voxzogo; however, management believes that patient switching will be gradual due to its five-year market lead and strong patient adherence, which will help maintain market share.
- Clinical Catalysts: BioMarin is advancing multiple clinical trials, including a Phase 3 study for hypochondroplasia and a pediatric Phase 3 study for BMN 401, which are expected to provide significant growth drivers for the company, particularly in the global rare disease treatment landscape.
See More
FDA Grants Approvals for Multiple New Therapies
- Hernexeos Approval: On February 26, 2026, the FDA granted accelerated approval to Boehringer Ingelheim's Hernexeos as a first-line treatment for adult patients with HER2-mutant advanced non-small cell lung cancer, representing a significant milestone that could improve patient prognosis and reduce the risk of brain metastases for the 2-4% of NSCLC cases affected.
- Yuviwel Launch: Ascendis Pharma secured FDA approval on February 27, 2026, for Yuviwel, the first and only therapy providing continuous systemic exposure to C-type natriuretic peptide (CNP) for children aged 2 and older with achondroplasia, which is expected to enhance treatment adherence compared to daily alternatives.
- Palynziq Indication Expansion: BioMarin received FDA approval on February 27, 2026, to expand Palynziq's indication to include pediatric patients aged 12 and older with phenylketonuria, which is anticipated to further drive sales growth, having generated $433 million in revenue in 2025.
- Sogroya New Indications: Novo Nordisk obtained FDA approval on February 27, 2026, for Sogroya's expanded indications for various growth disorders, offering a once-weekly growth hormone alternative that is expected to improve treatment adherence and alleviate the burden of daily injections for families.
See More
UniQure's Gene Therapy Faces FDA Challenges
- FDA Study Requirement: The U.S. FDA has mandated that UniQure conduct a placebo-controlled trial to validate the efficacy of its gene therapy for Huntington's disease, despite the company's claims that such a trial is unethical due to the need for prolonged general anesthesia.
- Clinical Trial Controversy: An FDA official indicated that UniQure's clinical trial data does not meet approval criteria, suggesting that the company may be aware of its earlier trial failures and has not pursued necessary clinical studies.
- Stock Price Fluctuation: Despite facing challenges from the FDA, UniQure's stock rose over 10% on Thursday, although it has fallen 58% this year, reflecting market concerns regarding the therapy's prospects.
- Regulatory Tensions: The relationship between UniQure and the FDA is increasingly strained, with the agency facing criticism for recent drug application rejections, and UniQure accusing the FDA of reversing its acceptance standards for the company's clinical trial data.
See More
VOXZOGO Treatment Shows Significant Improvements in Children's Skeletal Growth
- Early Treatment Impact: New data indicates that children starting VOXZOGO treatment before age 2 experienced an average height increase of 4.7 cm (p=0.0002) after four years, underscoring the critical importance of early intervention for skeletal growth.
- Proportionality Improvements: Among children treated with VOXZOGO, the upper-to-lower body segment ratio (ULBR) improved by 0.33 (p=0.0005) in the first year and further improved to 0.53 (p<0.0001) by year four, demonstrating the drug's effectiveness in enhancing body proportionality.
- Global Clinical Evidence: More than 5,000 children across over 50 countries have received VOXZOGO, with Japanese children showing an average height increase of 9.91 cm at 12 months and 15.62 cm at 24 months, further validating the drug's efficacy and safety.
- Long-Term Safety Studies: BioMarin will present findings on VOXZOGO's long-term safety and growth outcomes at the 2026 ACMG Annual Clinical Genetics Meeting, emphasizing its significant role in children's skeletal development and aiding communication between physicians and families.
See More
VOXZOGO Shows Significant Efficacy in Children
- Early Treatment Impact: New data indicates that children starting VOXZOGO treatment before age 2 experienced an average height increase of 4.7 cm over four years (p=0.0002), underscoring the significant impact of early intervention on skeletal growth and reinforcing VOXZOGO's status as the only approved treatment.
- Proportionality Improvement: Among children treated with VOXZOGO, the upper-to-lower body segment ratio (ULBR) improved by 0.33 (p=0.0005) in the first year and reached 0.53 (p<0.0001) by year four, indicating that early treatment enhances not only height but also overall body proportionality, which is crucial for health.
- Global Evidence Support: More than 5,000 children across over 50 countries have received VOXZOGO, with Japanese children showing an average height increase of 9.91 cm at 12 months and 15.62 cm at 24 months, further validating the efficacy and safety of VOXZOGO.
- Clinical Research Findings: BioMarin will present these research results at the 2026 American College of Medical Genetics and Genomics Annual Meeting, emphasizing the importance of VOXZOGO in children's skeletal development and facilitating communication between families and healthcare providers.
See More
BridgeBio's Promising Biotech Pipeline Highlighted by William Blair
- Unique Market Position: Analyst John Boyle highlights BridgeBio's unique position with the accelerating launch of Attruby and near-term NDA submissions, setting the stage for late-2026/early-2027 launches in the rare genetic disease treatment market.
- Product Competitive Edge: Market surveys indicate that prescribers view both Attruby and Amvuttra as superior to Pfizer's Tafamidis, which has approximately $6 billion in annual sales, suggesting that BridgeBio's products are gaining traction in clinical applications.
- Sales Forecast and Risks: The analyst models U.S. sales of Attruby reaching $3.4 billion by 2035, while acknowledging potential generic entry risks in late 2028; however, Attruby's strong TTR stabilization and simple twice-daily dosing could justify premium pricing.
- Other Market Opportunities: BridgeBio also sees significant market opportunities with other rare disease drugs, particularly infigratinib for achondroplasia and encaleret for autosomal dominant hypocalcemia type 1, with the latter achieving its primary endpoint in the CALIBRATE Phase 3 study in October 2025, indicating strong future prospects.
See More
BioMarin Pharmaceutical Outlines Strategic Refresh and Market Outlook
- Strategic Update: BioMarin Pharmaceutical emphasized its strategic refresh at the TD Cowen conference, particularly through the Inozyme deal and the pending Amicus acquisition, aiming to expand enzyme therapy scale across approximately 80 countries, thereby enhancing its competitive position in the rare disease market.
- Revenue Outlook: Despite anticipating a roughly 3% revenue headwind in 2026 due to the removal of ROCTAVIAN and Kuvan revenue declines, BioMarin expects core enzyme and skeletal businesses to grow at mid-to-high single digits, targeting a 40% operating margin by 2026.
- Competitive Dynamics: BioMarin anticipates new FDA-approved competition for Voxzogo; however, management believes that patient switching will be gradual due to its five-year market lead and strong patient adherence, which will help maintain market share.
- Clinical Catalysts: BioMarin is advancing multiple clinical trials, including a Phase 3 study for hypochondroplasia and a pediatric Phase 3 study for BMN 401, which are expected to provide significant growth drivers for the company, particularly in the global rare disease treatment landscape.
See More
FDA Grants Approvals for Multiple New Therapies
- Hernexeos Approval: On February 26, 2026, the FDA granted accelerated approval to Boehringer Ingelheim's Hernexeos as a first-line treatment for adult patients with HER2-mutant advanced non-small cell lung cancer, representing a significant milestone that could improve patient prognosis and reduce the risk of brain metastases for the 2-4% of NSCLC cases affected.
- Yuviwel Launch: Ascendis Pharma secured FDA approval on February 27, 2026, for Yuviwel, the first and only therapy providing continuous systemic exposure to C-type natriuretic peptide (CNP) for children aged 2 and older with achondroplasia, which is expected to enhance treatment adherence compared to daily alternatives.
- Palynziq Indication Expansion: BioMarin received FDA approval on February 27, 2026, to expand Palynziq's indication to include pediatric patients aged 12 and older with phenylketonuria, which is anticipated to further drive sales growth, having generated $433 million in revenue in 2025.
- Sogroya New Indications: Novo Nordisk obtained FDA approval on February 27, 2026, for Sogroya's expanded indications for various growth disorders, offering a once-weekly growth hormone alternative that is expected to improve treatment adherence and alleviate the burden of daily injections for families.
See More
UniQure's Gene Therapy Faces FDA Challenges
- FDA Study Requirement: The U.S. FDA has mandated that UniQure conduct a placebo-controlled trial to validate the efficacy of its gene therapy for Huntington's disease, despite the company's claims that such a trial is unethical due to the need for prolonged general anesthesia.
- Clinical Trial Controversy: An FDA official indicated that UniQure's clinical trial data does not meet approval criteria, suggesting that the company may be aware of its earlier trial failures and has not pursued necessary clinical studies.
- Stock Price Fluctuation: Despite facing challenges from the FDA, UniQure's stock rose over 10% on Thursday, although it has fallen 58% this year, reflecting market concerns regarding the therapy's prospects.
- Regulatory Tensions: The relationship between UniQure and the FDA is increasingly strained, with the agency facing criticism for recent drug application rejections, and UniQure accusing the FDA of reversing its acceptance standards for the company's clinical trial data.
See More
