BioMarin's ENPP1 Deficiency Study Results Show Mixed Outcomes
Written by Emily J. Thompson, Senior Investment Analyst
Updated: May 18 2026
0mins
Source: Newsfilter
- Clinical Trial Results: BioMarin's ENERGY 3 trial demonstrated that BMN 401 significantly increased plasma inorganic pyrophosphate (PPi) levels through week 52, meeting one co-primary endpoint; however, it failed to improve Radiographic Global Impression of Change (RGI-C) scores, indicating limited treatment efficacy.
- Patient Recruitment: The trial enrolled 27 pediatric participants aged 1 to 12 to evaluate the safety and efficacy of BMN 401, yet no positive trends were observed across secondary endpoints, suggesting that the treatment did not meet expectations.
- Company Response: BioMarin's R&D head, Greg Friberg, expressed disappointment over the results, emphasizing the urgent need for effective treatments for ENPP1 deficiency, highlighting the company's commitment to addressing patient needs.
- Future Plans: BioMarin is actively evaluating the data to determine next steps and plans to present detailed results at an upcoming medical meeting, indicating the company's ongoing efforts to find solutions to this challenge.
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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: 52.740
Low
60.00
Averages
87.35
High
120.00
Current: 52.740
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.
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BioMarin's Voxzogo Phase 3 Data Shows Positive Results
- Clinical Trial Success: BioMarin Pharmaceutical's phase 3 trial for Voxzogo (vosoritide) in children with hypochondroplasia met its primary endpoint, demonstrating a statistically significant increase in annualized growth velocity compared to the placebo group.
- Significant Height Increases: Children treated with Voxzogo showed notable improvements in standing height and height Z-scores after one year, indicating the drug's effectiveness in promoting growth in pediatric patients.
- Accelerated Approval Context: Voxzogo received accelerated approval in November 2021, necessitating a post-market study to confirm its efficacy and ensure its continued availability in the market.
- Future Plans: BioMarin intends to submit a supplemental New Drug Application (sNDA) to the US FDA in Q3 to seek full approval for Voxzogo, further solidifying its market position.
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BioMarin's VOXZOGO Study Achieves Significant Growth Milestones
- Clinical Trial Success: BioMarin's VOXZOGO Phase 3 study for children with hypochondroplasia achieved a statistically significant annualized growth velocity (AGV) increase of 2.33 cm/year compared to placebo at week 52 (p<0.0001), providing new hope for treatment options.
- Height and Arm Span Improvements: Children receiving VOXZOGO also demonstrated significant increases in standing height and height Z-score (p<0.0001), which not only enhances their daily functioning but may also improve their independence.
- Consistent Safety Profile: The safety findings for VOXZOGO were consistent with its established profile in achondroplasia, with no new safety signals identified, thereby supporting the drug's further development and market introduction.
- Regulatory Submission Plans: BioMarin plans to submit a supplemental New Drug Application (sNDA) to the FDA in Q3 2026, followed by submissions to the EMA and other regional health authorities, indicating a strategic push into global markets.
See More
BioMarin's VOXZOGO Study Achieves Significant Growth Milestones
- Clinical Trial Success: BioMarin's VOXZOGO achieved a statistically significant increase in annualized growth velocity (AGV) of 2.33 cm/year compared to placebo in its Phase 3 study for children with hypochondroplasia, exceeding expectations and indicating the drug's potential to improve growth outcomes.
- Height and Arm Span Improvements: The study demonstrated significant increases in standing height and height Z-score for children receiving VOXZOGO, suggesting potential enhancements in daily activities and independence for these patients.
- Safety Profile Confirmed: The safety findings for VOXZOGO were consistent with the established profile in achondroplasia, with no new safety signals identified, thereby reinforcing confidence in its use for treating hypochondroplasia.
- Regulatory Submission Plans: BioMarin plans to submit a supplemental New Drug Application (sNDA) to the FDA in Q3 2026, followed by submissions to the EMA and other regional health authorities, marking a critical step towards market availability.
See More
BioMarin's BMN 401 Phase 3 Trial Results Mixed
- Trial Results: BioMarin's BMN 401 phase 3 trial for ENPP1 deficiency achieved only one of its two primary endpoints, with significant increases in plasma PPi levels over 52 weeks compared to the control group, but failed to meet the improvement in Radiographic Global Impression of Change (RGI-C), potentially impacting its market viability.
- Secondary Endpoints Missed: No positive trends were observed in secondary endpoints such as Rickets Severity Score (RSS) and growth Z-score, indicating limited efficacy of BMN 401, which may undermine investor confidence and future funding opportunities.
- Data Analysis Next Steps: BioMarin is currently analyzing the trial data to determine subsequent actions, a process that could influence its future research directions and resource allocation, particularly in the rare disease sector.
- Background on ENPP1 Deficiency: ENPP1 deficiency is a genetic condition leading to low pyrophosphate levels, and while BioMarin aims to address this medical gap, the complexity of the trial results may delay the product's market entry.
See More
BioMarin's ENERGY 3 Study Results on ENPP1 Deficiency
- Clinical Trial Results: BioMarin's ENERGY 3 trial demonstrated that treatment with BMN 401 led to statistically significant increases in plasma inorganic pyrophosphate (PPi) levels in children with ENPP1 deficiency over 52 weeks; however, no improvement was observed in Radiographic Global Impression of Change (RGI-C) scores, indicating limited treatment efficacy.
- Safety Assessment: BMN 401 was generally well-tolerated during the trial with no new safety signals reported, yet no positive trends were noted across secondary endpoints, including Rickets Severity Score and growth Z-score, highlighting the treatment's limitations.
- Significance of the Study: ENPP1 deficiency is a severe genetic condition with high mortality rates in infants, reaching up to 50%, thus the lack of significant clinical improvement from BMN 401 underscores the urgent need for new treatment options in this area.
- Next Steps: BioMarin is actively evaluating the trial data to determine the appropriate next steps for BMN 401, with plans to present detailed results at an upcoming medical meeting, continuing to focus on advancements in this critical research area.
See More
Morgan Stanley Bullish on Dividend Stocks
- Dividend Potential Analysis: Morgan Stanley highlights that companies initiating dividends outperform the market by an average of 650 basis points in the six months following the announcement and by 1,000 basis points in the subsequent 12 months, indicating a significant enhancement in investor returns, especially when dividends are reinvested for compounding benefits.
- Centene's Advantages: Centene boasts an 18% free cash flow yield and a net cash position exceeding 5% of its market cap, making it an ideal candidate for dividends; additionally, the company surpassed expectations for adjusted earnings per share and revenue in Q1, with its stock rising 44% this year.
- BioMarin's Acquisition Impact: BioMarin, with a net cash position of 7.6% of its market cap and a 10.4% free cash flow yield, successfully made the list after completing a $4.8 billion acquisition of Amicus Therapeutics, which is expected to significantly enhance its market share in rare metabolic diseases, although it slightly lowered its 2026 non-GAAP earnings per share guidance while raising its full-year revenue guidance to $3.825 to $3.925 billion.
- Deckers Outdoor's Growth Outlook: Deckers Outdoor posted better-than-expected results for its fiscal Q1, with a free cash flow yield of 6.7%, and analysts have rated the stock as a buy, anticipating that its portfolio of category-defining brands will drive future growth, with shares gaining nearly 10% this year.
See More
BioMarin's Voxzogo Phase 3 Data Shows Positive Results
- Clinical Trial Success: BioMarin Pharmaceutical's phase 3 trial for Voxzogo (vosoritide) in children with hypochondroplasia met its primary endpoint, demonstrating a statistically significant increase in annualized growth velocity compared to the placebo group.
- Significant Height Increases: Children treated with Voxzogo showed notable improvements in standing height and height Z-scores after one year, indicating the drug's effectiveness in promoting growth in pediatric patients.
- Accelerated Approval Context: Voxzogo received accelerated approval in November 2021, necessitating a post-market study to confirm its efficacy and ensure its continued availability in the market.
- Future Plans: BioMarin intends to submit a supplemental New Drug Application (sNDA) to the US FDA in Q3 to seek full approval for Voxzogo, further solidifying its market position.
See More
BioMarin's VOXZOGO Study Achieves Significant Growth Milestones
- Clinical Trial Success: BioMarin's VOXZOGO Phase 3 study for children with hypochondroplasia achieved a statistically significant annualized growth velocity (AGV) increase of 2.33 cm/year compared to placebo at week 52 (p<0.0001), providing new hope for treatment options.
- Height and Arm Span Improvements: Children receiving VOXZOGO also demonstrated significant increases in standing height and height Z-score (p<0.0001), which not only enhances their daily functioning but may also improve their independence.
- Consistent Safety Profile: The safety findings for VOXZOGO were consistent with its established profile in achondroplasia, with no new safety signals identified, thereby supporting the drug's further development and market introduction.
- Regulatory Submission Plans: BioMarin plans to submit a supplemental New Drug Application (sNDA) to the FDA in Q3 2026, followed by submissions to the EMA and other regional health authorities, indicating a strategic push into global markets.
See More
BioMarin's VOXZOGO Study Achieves Significant Growth Milestones
- Clinical Trial Success: BioMarin's VOXZOGO achieved a statistically significant increase in annualized growth velocity (AGV) of 2.33 cm/year compared to placebo in its Phase 3 study for children with hypochondroplasia, exceeding expectations and indicating the drug's potential to improve growth outcomes.
- Height and Arm Span Improvements: The study demonstrated significant increases in standing height and height Z-score for children receiving VOXZOGO, suggesting potential enhancements in daily activities and independence for these patients.
- Safety Profile Confirmed: The safety findings for VOXZOGO were consistent with the established profile in achondroplasia, with no new safety signals identified, thereby reinforcing confidence in its use for treating hypochondroplasia.
- Regulatory Submission Plans: BioMarin plans to submit a supplemental New Drug Application (sNDA) to the FDA in Q3 2026, followed by submissions to the EMA and other regional health authorities, marking a critical step towards market availability.
See More
BioMarin's BMN 401 Phase 3 Trial Results Mixed
- Trial Results: BioMarin's BMN 401 phase 3 trial for ENPP1 deficiency achieved only one of its two primary endpoints, with significant increases in plasma PPi levels over 52 weeks compared to the control group, but failed to meet the improvement in Radiographic Global Impression of Change (RGI-C), potentially impacting its market viability.
- Secondary Endpoints Missed: No positive trends were observed in secondary endpoints such as Rickets Severity Score (RSS) and growth Z-score, indicating limited efficacy of BMN 401, which may undermine investor confidence and future funding opportunities.
- Data Analysis Next Steps: BioMarin is currently analyzing the trial data to determine subsequent actions, a process that could influence its future research directions and resource allocation, particularly in the rare disease sector.
- Background on ENPP1 Deficiency: ENPP1 deficiency is a genetic condition leading to low pyrophosphate levels, and while BioMarin aims to address this medical gap, the complexity of the trial results may delay the product's market entry.
See More
BioMarin's ENERGY 3 Study Results on ENPP1 Deficiency
- Clinical Trial Results: BioMarin's ENERGY 3 trial demonstrated that treatment with BMN 401 led to statistically significant increases in plasma inorganic pyrophosphate (PPi) levels in children with ENPP1 deficiency over 52 weeks; however, no improvement was observed in Radiographic Global Impression of Change (RGI-C) scores, indicating limited treatment efficacy.
- Safety Assessment: BMN 401 was generally well-tolerated during the trial with no new safety signals reported, yet no positive trends were noted across secondary endpoints, including Rickets Severity Score and growth Z-score, highlighting the treatment's limitations.
- Significance of the Study: ENPP1 deficiency is a severe genetic condition with high mortality rates in infants, reaching up to 50%, thus the lack of significant clinical improvement from BMN 401 underscores the urgent need for new treatment options in this area.
- Next Steps: BioMarin is actively evaluating the trial data to determine the appropriate next steps for BMN 401, with plans to present detailed results at an upcoming medical meeting, continuing to focus on advancements in this critical research area.
See More
