BioMarin Discontinues Phase 2 Trials for Voxzogo
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Mar 16 2026
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Should l Buy BMRN?
BioMarin announced its decision to discontinue dosing and enrollment in its Phase 2 trials for Voxzogo in Turner syndrome, SHOX-deficiency and aggrecan-deficiency following the occurrence of several slipped capital femoral epiphysis events in two ongoing investigator-sponsored trials. "SCFE events have not been observed in the Phase 2 BioMarin trials in these same conditions, nor have any cases been observed in the more than 5,000 infants and children who have received Voxzogo for achondroplasia, which includes 10 years of clinical research and more than 10,000 patient-years of safety data in clinical studies and post-marketing surveillance," the company added. There have also been no observed cases in BioMarin's clinical trials for hypochondroplasia. The Phase 2 CANOPY trials of Voxzogo in children with Noonan syndrome, as well as those living with idiopathic short stature without ACAN-deficiency, which represents approximately 95% of children enrolled in the ISS trial, will continue as planned, the company added. Shares of BioMarin are down 3% to $56.91 in premarket trading.
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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: 54.880
Low
60.00
Averages
87.35
High
120.00
Current: 54.880
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.
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- Experienced Leadership: Ian Clark, who joined BioMarin's board in 2025, previously served as CEO of Genentech from 2010 to 2016, bringing extensive biopharmaceutical industry experience that could benefit the company's future growth.
- Current Chair's Retirement: Current Chair Richard Meier will retire after nearly 20 years on the board, having joined in 2006 and served as Chair since 2023, signaling a new direction in the company's leadership.
- Stock Price Fluctuation: BioMarin's stock closed down 0.47% at $54.62 on Nasdaq, but traded up 1.24% in after-hours at $55.30, reflecting a relatively stable market reaction to the new chair appointment.
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- New Chair Background: Ian T. Clark joined BioMarin's Board in 2025 and previously served as CEO of Genentech, bringing extensive governance and leadership experience in the biopharmaceutical industry, which is anticipated to provide strategic insights as the company expands in 80 countries worldwide.
- Former Chair Contributions: Richard A. Meier has been on the Board since 2006, during which time the company saw meaningful improvements in profitability, with significant growth in non-GAAP operating margins and earnings per share, highlighting his crucial role in the company's evolution.
- Future Outlook: Under the new leadership, BioMarin plans to continue executing its global strategy, leveraging recent acquisitions to unlock significant value for all stakeholders, particularly patients, showcasing the company's strong potential in the rare disease treatment landscape.
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- Rich Leadership Experience: Clark joined BioMarin's Board in 2025 and previously served as CEO of Genentech, bringing extensive governance and executive experience in the biopharmaceutical industry, which is expected to provide strategic guidance for the company's future growth.
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- Contributions of Former Chair: During Meier's tenure as Chair, he significantly improved the company's profitability, with notable growth in non-GAAP operating margin and earnings per share, indicating that the company will continue to benefit from its strong foundation and strategic clarity moving forward.
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- Audience Engagement: The CNBC Cures newsletter has surpassed 20,000 subscribers, making it one of the fastest-growing newsletters on the network, indicating strong viewer interest and support for rare disease topics.
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- Safety Profile Maintained: Although no SCFE events were observed in BioMarin's other phase 2 trials and none in over 5,000 children treated with Voxzogo, the occurrence of these events may still undermine investor confidence in the company's products, potentially affecting stock performance.
- Continued Trials: BioMarin stated that the Canopy phase 2 trials for children with Noonan syndrome and idiopathic short stature (ISS) without ACAN deficiency will proceed as planned, suggesting that the company still sees potential in Voxzogo for certain indications, which may support future market performance.
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See More
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- 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.
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See More
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- Board Chair Transition: BioMarin Pharmaceutical Inc. announced the appointment of Ian Clark as Chair of the Board, with the election set for June 2 at the Annual Meeting, indicating a significant governance shift for the company.
- Experienced Leadership: Ian Clark, who joined BioMarin's board in 2025, previously served as CEO of Genentech from 2010 to 2016, bringing extensive biopharmaceutical industry experience that could benefit the company's future growth.
- Current Chair's Retirement: Current Chair Richard Meier will retire after nearly 20 years on the board, having joined in 2006 and served as Chair since 2023, signaling a new direction in the company's leadership.
- Stock Price Fluctuation: BioMarin's stock closed down 0.47% at $54.62 on Nasdaq, but traded up 1.24% in after-hours at $55.30, reflecting a relatively stable market reaction to the new chair appointment.
See More
BioMarin Appoints New Chair of the Board
- Board Leadership Change: BioMarin Pharmaceutical Inc. has appointed Ian T. Clark as Chair of the Board, succeeding Richard A. Meier, who has served for 20 years, marking a significant governance shift that is expected to drive further growth in the rare disease sector.
- New Chair Background: Ian T. Clark joined BioMarin's Board in 2025 and previously served as CEO of Genentech, bringing extensive governance and leadership experience in the biopharmaceutical industry, which is anticipated to provide strategic insights as the company expands in 80 countries worldwide.
- Former Chair Contributions: Richard A. Meier has been on the Board since 2006, during which time the company saw meaningful improvements in profitability, with significant growth in non-GAAP operating margins and earnings per share, highlighting his crucial role in the company's evolution.
- Future Outlook: Under the new leadership, BioMarin plans to continue executing its global strategy, leveraging recent acquisitions to unlock significant value for all stakeholders, particularly patients, showcasing the company's strong potential in the rare disease treatment landscape.
See More
BioMarin Elects Ian T. Clark as Board Chair
- New Board Chair: BioMarin has appointed Ian T. Clark as Chair of the Board, effective after the Annual Meeting of Stockholders on June 2, 2026, succeeding Richard A. Meier, who has served on the Board for 20 years, marking a significant leadership transition for the company.
- Rich Leadership Experience: Clark joined BioMarin's Board in 2025 and previously served as CEO of Genentech, bringing extensive governance and executive experience in the biopharmaceutical industry, which is expected to provide strategic guidance for the company's future growth.
- Strategic Growth Potential: Clark stated that BioMarin has the potential to unlock significant value for all stakeholders through two recent acquisitions, which not only include employees and shareholders but most importantly patients, highlighting the company's growth potential in the rare disease sector.
- Contributions of Former Chair: During Meier's tenure as Chair, he significantly improved the company's profitability, with notable growth in non-GAAP operating margin and earnings per share, indicating that the company will continue to benefit from its strong foundation and strategic clarity moving forward.
See More
CNBC Launches Advocacy Platform for Rare Diseases
- Platform Launch: CNBC launched the CNBC Cures platform on January 8, aimed at giving a voice to the 30 million Americans living with rare diseases, connecting them with existing advocacy groups to drive attention and support from the medical community.
- Audience Engagement: The CNBC Cures newsletter has surpassed 20,000 subscribers, making it one of the fastest-growing newsletters on the network, indicating strong viewer interest and support for rare disease topics.
- Social Media Impact: Cures-related videos have garnered 1 million views across Facebook, TikTok, Instagram, and LinkedIn, demonstrating significant outreach and enhancing public awareness of rare diseases.
- Successful Summit: The inaugural CNBC Cures Summit attracted notable figures like Warren Buffett and sold out shortly after registration opened, with over 8,000 views for the livestream, reflecting heightened societal attention and support for rare disease issues.
See More
BioMarin Discontinues Voxzogo Trials Amid Safety Concerns
- Trial Discontinuation: BioMarin announced the discontinuation of dosing and enrollment in its phase 2 trials for Voxzogo in Turner syndrome, SHOX deficiency, and ACAN deficiency due to multiple slipped capital femoral epiphysis (SCFE) events, indicating potential safety concerns that could impact future drug development strategies.
- Safety Profile Maintained: Although no SCFE events were observed in BioMarin's other phase 2 trials and none in over 5,000 children treated with Voxzogo, the occurrence of these events may still undermine investor confidence in the company's products, potentially affecting stock performance.
- Continued Trials: BioMarin stated that the Canopy phase 2 trials for children with Noonan syndrome and idiopathic short stature (ISS) without ACAN deficiency will proceed as planned, suggesting that the company still sees potential in Voxzogo for certain indications, which may support future market performance.
- Stock Price Reaction: Following this announcement, BioMarin's shares fell 4.6% to $55.80 in premarket trading, reflecting the market's negative reaction to the trial discontinuation, which could impact its future financing and R&D capabilities.
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
