New Research on VOXZOGO in Children with Achondroplasia
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 1 hour ago
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Should l Buy BMRN?
Source: Newsfilter
- Significant Long-Term Effects: VOXZOGO's long-term clinical trials in children with achondroplasia show that children who started treatment after age 5 achieved a mean height difference of 10.60 cm after six years and 13.59 cm after eight years, indicating the positive impact of early intervention on child health.
- Sustained Bone Health Improvement: In a study of 119 children treated with VOXZOGO, bone mineral content and density showed significant increases over six years, with stable bone density Z-scores, demonstrating the treatment's effectiveness in maintaining bone health and enhancing parental confidence in the therapy.
- New Research Data Presentation: BioMarin will present new research data on VOXZOGO for achondroplasia and hypochondroplasia at the 2026 Pediatric Endocrine Society Annual Meeting, which is expected to provide critical insights for future clinical applications.
- Registration Trial Progress: BioMarin plans to release pivotal Phase 3 trial results for hypochondroplasia in the first half of 2026, and if results are positive, will submit applications to regulatory authorities in the second half of 2026 to expand VOXZOGO's indications.
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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: 53.910
Low
60.00
Averages
87.35
High
120.00
Current: 53.910
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 a portfolio of 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 Galafold (migalastat) is the first oral treatment for Fabry disease, and Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat), is a two-component therapy for Pompe disease.
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.
BioMarin Pharmaceutical Q1 Earnings Announcement Scheduled
- Earnings Release Date: BioMarin Pharmaceutical is set to announce its Q1 2023 earnings on May 4th after market close, with consensus EPS estimate at $0.91, reflecting a 19.5% year-over-year decline, which could negatively impact investor sentiment.
- Revenue Expectations: The anticipated revenue for Q1 is $751.68 million, representing a modest 0.9% year-over-year growth, indicating the company's stability in the rare disease sector despite limited growth potential.
- Forecast Changes: Over the past three months, there have been no upward revisions to EPS estimates and 12 downward revisions, highlighting analysts' concerns regarding the company's profitability, which may exert downward pressure on the stock price.
- Historical Performance Review: BioMarin has beaten EPS and revenue estimates 75% of the time over the last two years, although the recent trend of downward revisions could affect future performance.
See More
New Research on VOXZOGO in Children with Achondroplasia
- Significant Long-Term Effects: VOXZOGO's long-term clinical trials in children with achondroplasia show that children who started treatment after age 5 achieved a mean height difference of 10.60 cm after six years and 13.59 cm after eight years, indicating the positive impact of early intervention on child health.
- Sustained Bone Health Improvement: In a study of 119 children treated with VOXZOGO, bone mineral content and density showed significant increases over six years, with stable bone density Z-scores, demonstrating the treatment's effectiveness in maintaining bone health and enhancing parental confidence in the therapy.
- New Research Data Presentation: BioMarin will present new research data on VOXZOGO for achondroplasia and hypochondroplasia at the 2026 Pediatric Endocrine Society Annual Meeting, which is expected to provide critical insights for future clinical applications.
- Registration Trial Progress: BioMarin plans to release pivotal Phase 3 trial results for hypochondroplasia in the first half of 2026, and if results are positive, will submit applications to regulatory authorities in the second half of 2026 to expand VOXZOGO's indications.
See More
VOXZOGO Shows Positive Long-Term Effects on Children's Bone Health
- Significant Long-Term Effects: VOXZOGO demonstrates a positive impact on children's bone health, with treated children showing an average height increase of 10.60 cm after six years and 13.59 cm after eight years, significantly outperforming untreated cohorts, highlighting the importance of early intervention for child health.
- Sustained Bone Density Improvement: In a study of 119 children receiving VOXZOGO, bone mineral content (BMC) increased annually over six years while bone mineral density (BMD) Z-scores remained stable, indicating that long-term treatment effectively maintains bone health and boosts parental confidence in the therapy.
- New Research Findings: BioMarin will present new data on VOXZOGO's effects in hypochondroplasia at the 2026 Pediatric Endocrine Society Annual Meeting, including significant improvements in BMD and BMC after 12 months, which are expected to support approval for this new indication.
- Global Impact Expansion: Since its approval in 2021, VOXZOGO has assisted over 5,000 children, and BioMarin is committed to enhancing global awareness and treatment outcomes for children's bone health through ongoing clinical research and robust patient support services.
See More
BioMarin Acquires Amicus for $4.8 Billion to Enhance Rare Disease Portfolio
- Acquisition Completed: BioMarin has finalized its acquisition of Amicus Therapeutics for $14.50 per share, totaling approximately $4.8 billion, significantly enhancing BioMarin's market position in the rare disease sector.
- New Drugs Added: This acquisition adds Galafold® for Fabry disease and Pombiliti® + Opfolda® for Pompe disease to BioMarin's commercial portfolio, which is expected to generate new revenue streams and expand market coverage.
- Future Guidance: BioMarin plans to provide updated FY 2026 guidance during its first quarter earnings call, with expectations that this acquisition will drive future revenue growth and product line diversification.
- Strategic Importance: The CEO of BioMarin stated that this acquisition not only strengthens the company's growth strategy but also leverages its global scale and advanced manufacturing capabilities to further enhance treatment options for patients with rare diseases.
See More
Intellia's Gene Editing Therapy Trial Achieves Success
- Clinical Trial Success: Intellia Therapeutics' gene editing therapy achieved an 87% reduction in attacks during late-stage trials, significantly surpassing the placebo group, marking a crucial milestone in gene editing and paving the way for FDA approval.
- Positive Patient Outcomes: Six months post-treatment, 62% of patients were attack-free and not using other therapies, demonstrating the therapy's efficacy and durability, which could transform treatment paradigms for hereditary angioedema patients.
- Favorable Safety Profile: Despite a patient death in a separate trial due to liver toxicity, Intellia reported a favorable safety and tolerability profile for its therapy, with common side effects being infusion-related reactions, headaches, and fatigue, bolstering market confidence.
- Market Competition Outlook: If approved by the FDA, Intellia's lonvoguran ziclumeran will compete with other chronic drugs, and while the commercial success of gene therapies has been mixed, the potential for a one-time treatment continues to attract investor interest.
See More
BioMarin to Host Q1 2026 Financial Results Call on May 4
- Conference Call Announcement: BioMarin Pharmaceutical Inc. will host a conference call on May 4, 2026, at 4:30 p.m. ET, where CEO Alexander Hardy will discuss Q1 2026 financial results and provide a business update, which is expected to deliver crucial financial and strategic insights to investors.
- Dial-in Information Released: The call will feature dial-in numbers including 800-715-9871 for U.S./Canada and 646-307-1963 for international participants, ensuring global investors can easily join this significant financial communication, thereby enhancing transparency and investor confidence.
- Live Webcast Access: Interested parties can access a live audio webcast of the conference call via the investor section of BioMarin's website, further improving information accessibility and ensuring all stakeholders stay informed about the company's latest developments.
- Replay Service Offered: Following the call, BioMarin will provide a replay service for one week, with dial-in numbers of 800-770-2030 for U.S./Canada and 609-800-9909 for international, ensuring that investors who cannot participate live can still obtain key information, thus strengthening communication between the company and its investors.
See More
BioMarin Pharmaceutical Q1 Earnings Announcement Scheduled
- Earnings Release Date: BioMarin Pharmaceutical is set to announce its Q1 2023 earnings on May 4th after market close, with consensus EPS estimate at $0.91, reflecting a 19.5% year-over-year decline, which could negatively impact investor sentiment.
- Revenue Expectations: The anticipated revenue for Q1 is $751.68 million, representing a modest 0.9% year-over-year growth, indicating the company's stability in the rare disease sector despite limited growth potential.
- Forecast Changes: Over the past three months, there have been no upward revisions to EPS estimates and 12 downward revisions, highlighting analysts' concerns regarding the company's profitability, which may exert downward pressure on the stock price.
- Historical Performance Review: BioMarin has beaten EPS and revenue estimates 75% of the time over the last two years, although the recent trend of downward revisions could affect future performance.
See More
New Research on VOXZOGO in Children with Achondroplasia
- Significant Long-Term Effects: VOXZOGO's long-term clinical trials in children with achondroplasia show that children who started treatment after age 5 achieved a mean height difference of 10.60 cm after six years and 13.59 cm after eight years, indicating the positive impact of early intervention on child health.
- Sustained Bone Health Improvement: In a study of 119 children treated with VOXZOGO, bone mineral content and density showed significant increases over six years, with stable bone density Z-scores, demonstrating the treatment's effectiveness in maintaining bone health and enhancing parental confidence in the therapy.
- New Research Data Presentation: BioMarin will present new research data on VOXZOGO for achondroplasia and hypochondroplasia at the 2026 Pediatric Endocrine Society Annual Meeting, which is expected to provide critical insights for future clinical applications.
- Registration Trial Progress: BioMarin plans to release pivotal Phase 3 trial results for hypochondroplasia in the first half of 2026, and if results are positive, will submit applications to regulatory authorities in the second half of 2026 to expand VOXZOGO's indications.
See More
VOXZOGO Shows Positive Long-Term Effects on Children's Bone Health
- Significant Long-Term Effects: VOXZOGO demonstrates a positive impact on children's bone health, with treated children showing an average height increase of 10.60 cm after six years and 13.59 cm after eight years, significantly outperforming untreated cohorts, highlighting the importance of early intervention for child health.
- Sustained Bone Density Improvement: In a study of 119 children receiving VOXZOGO, bone mineral content (BMC) increased annually over six years while bone mineral density (BMD) Z-scores remained stable, indicating that long-term treatment effectively maintains bone health and boosts parental confidence in the therapy.
- New Research Findings: BioMarin will present new data on VOXZOGO's effects in hypochondroplasia at the 2026 Pediatric Endocrine Society Annual Meeting, including significant improvements in BMD and BMC after 12 months, which are expected to support approval for this new indication.
- Global Impact Expansion: Since its approval in 2021, VOXZOGO has assisted over 5,000 children, and BioMarin is committed to enhancing global awareness and treatment outcomes for children's bone health through ongoing clinical research and robust patient support services.
See More
BioMarin Acquires Amicus for $4.8 Billion to Enhance Rare Disease Portfolio
- Acquisition Completed: BioMarin has finalized its acquisition of Amicus Therapeutics for $14.50 per share, totaling approximately $4.8 billion, significantly enhancing BioMarin's market position in the rare disease sector.
- New Drugs Added: This acquisition adds Galafold® for Fabry disease and Pombiliti® + Opfolda® for Pompe disease to BioMarin's commercial portfolio, which is expected to generate new revenue streams and expand market coverage.
- Future Guidance: BioMarin plans to provide updated FY 2026 guidance during its first quarter earnings call, with expectations that this acquisition will drive future revenue growth and product line diversification.
- Strategic Importance: The CEO of BioMarin stated that this acquisition not only strengthens the company's growth strategy but also leverages its global scale and advanced manufacturing capabilities to further enhance treatment options for patients with rare diseases.
See More
Intellia's Gene Editing Therapy Trial Achieves Success
- Clinical Trial Success: Intellia Therapeutics' gene editing therapy achieved an 87% reduction in attacks during late-stage trials, significantly surpassing the placebo group, marking a crucial milestone in gene editing and paving the way for FDA approval.
- Positive Patient Outcomes: Six months post-treatment, 62% of patients were attack-free and not using other therapies, demonstrating the therapy's efficacy and durability, which could transform treatment paradigms for hereditary angioedema patients.
- Favorable Safety Profile: Despite a patient death in a separate trial due to liver toxicity, Intellia reported a favorable safety and tolerability profile for its therapy, with common side effects being infusion-related reactions, headaches, and fatigue, bolstering market confidence.
- Market Competition Outlook: If approved by the FDA, Intellia's lonvoguran ziclumeran will compete with other chronic drugs, and while the commercial success of gene therapies has been mixed, the potential for a one-time treatment continues to attract investor interest.
See More
BioMarin to Host Q1 2026 Financial Results Call on May 4
- Conference Call Announcement: BioMarin Pharmaceutical Inc. will host a conference call on May 4, 2026, at 4:30 p.m. ET, where CEO Alexander Hardy will discuss Q1 2026 financial results and provide a business update, which is expected to deliver crucial financial and strategic insights to investors.
- Dial-in Information Released: The call will feature dial-in numbers including 800-715-9871 for U.S./Canada and 646-307-1963 for international participants, ensuring global investors can easily join this significant financial communication, thereby enhancing transparency and investor confidence.
- Live Webcast Access: Interested parties can access a live audio webcast of the conference call via the investor section of BioMarin's website, further improving information accessibility and ensuring all stakeholders stay informed about the company's latest developments.
- Replay Service Offered: Following the call, BioMarin will provide a replay service for one week, with dial-in numbers of 800-770-2030 for U.S./Canada and 609-800-9909 for international, ensuring that investors who cannot participate live can still obtain key information, thus strengthening communication between the company and its investors.
See More
