BioMarin Pharmaceutical (BMRN)
Market Price (9/25/2026): $61.6 | Market Cap: $11.9 BilSector: Health Care | Industry: Biotechnology
BioMarin Pharmaceutical (BMRN)
Market Price (9/25/2026): $61.6Market Cap: $11.9 BilSector: Health CareIndustry: Biotechnology
Investment Highlights Why It Matters Detailed financial logic regarding cash flow yields vs trend-riding momentum.
Attractive cash flow generationCFO/Rev LTMCash Flow from Operations / Revenue (Sales), Last Twelve Months (LTM) is 25%, FCF/Rev LTMFree Cash Flow / Revenue (Sales), Last Twelve Months (LTM) is 21% Attractive yieldFCF Yield is 6.1% Low stock price volatilityVol 12M is 35% Megatrend and thematic driversMegatrends include Biotechnology & Genomics, and Precision Medicine. Themes include Gene Editing & Therapy, Targeted Therapies, Show more. | Weak multi-year price returns2Y Excs Rtn is -49%, 3Y Excs Rtn is -104% | Expensive valuation multiplesP/EBITPrice/EBIT or Price/(Operating Income) ratio is 51x, P/EPrice/Earnings or Price/(Net Income) is 163x Yield minus risk free rate is negativeERPEquity Risk Premium (ERP) = Total Yield - Risk Free Rate, Reflects the premium above risk free assets offered by the investment. is -3.3% Key risksBMRN key risks include [1] intensifying competition for its flagship product Voxzogo, Show more. |
| Attractive cash flow generationCFO/Rev LTMCash Flow from Operations / Revenue (Sales), Last Twelve Months (LTM) is 25%, FCF/Rev LTMFree Cash Flow / Revenue (Sales), Last Twelve Months (LTM) is 21% |
| Attractive yieldFCF Yield is 6.1% |
| Low stock price volatilityVol 12M is 35% |
| Megatrend and thematic driversMegatrends include Biotechnology & Genomics, and Precision Medicine. Themes include Gene Editing & Therapy, Targeted Therapies, Show more. |
| Weak multi-year price returns2Y Excs Rtn is -49%, 3Y Excs Rtn is -104% |
| Expensive valuation multiplesP/EBITPrice/EBIT or Price/(Operating Income) ratio is 51x, P/EPrice/Earnings or Price/(Net Income) is 163x |
| Yield minus risk free rate is negativeERPEquity Risk Premium (ERP) = Total Yield - Risk Free Rate, Reflects the premium above risk free assets offered by the investment. is -3.3% |
| Key risksBMRN key risks include [1] intensifying competition for its flagship product Voxzogo, Show more. |
Qualitative Assessment
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Updated on 9/24/2026
BioMarin Pharmaceutical (BMRN) stock has gained about 5% since 5/31/2026 because of the following key factors:
1. Exceptional Fiscal Q2 2026 Financial Results and Increased Full-Year Outlook.
BioMarin Pharmaceutical reported strong financial performance for the second quarter ended June 30, 2026. The company posted adjusted earnings per share (EPS) of $1.20, significantly exceeding the consensus estimate of $0.95. Total revenues for the quarter increased 20% year-over-year to $990 million, surpassing analyst expectations of approximately $932 million. Following these results, BioMarin raised its full-year fiscal year 2026 guidance for total revenues and non-GAAP diluted EPS.
2. Strategic Contributions from Amicus Therapeutics Acquisition and Realized Synergies.
The acquisition of Amicus Therapeutics, which closed on April 27, 2026, played a pivotal role in BioMarin's growth during the period. The newly acquired products, GALAFOLD® and POMBILITI® + OPFOLDA®, contributed to a 25% year-over-year growth in Metabolic Conditions revenue during fiscal Q2 2026. BioMarin projects approximately $220 million in annual non-GAAP cost reductions from this integration, expected to be fully realized by fiscal year 2028, along with an accelerated timeline for achieving its gross leverage target.
Show more
BioMarin Pharmaceutical (BMRN) stock has gained about 5% since 5/31/2026 because of the following key factors:
1. Exceptional Fiscal Q2 2026 Financial Results and Increased Full-Year Outlook.
BioMarin Pharmaceutical reported strong financial performance for the second quarter ended June 30, 2026. The company posted adjusted earnings per share (EPS) of $1.20, significantly exceeding the consensus estimate of $0.95. Total revenues for the quarter increased 20% year-over-year to $990 million, surpassing analyst expectations of approximately $932 million. Following these results, BioMarin raised its full-year fiscal year 2026 guidance for total revenues and non-GAAP diluted EPS.
2. Strategic Contributions from Amicus Therapeutics Acquisition and Realized Synergies.
The acquisition of Amicus Therapeutics, which closed on April 27, 2026, played a pivotal role in BioMarin's growth during the period. The newly acquired products, GALAFOLD® and POMBILITI® + OPFOLDA®, contributed to a 25% year-over-year growth in Metabolic Conditions revenue during fiscal Q2 2026. BioMarin projects approximately $220 million in annual non-GAAP cost reductions from this integration, expected to be fully realized by fiscal year 2028, along with an accelerated timeline for achieving its gross leverage target.
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Updated on 9/24/2026
BioMarin Pharmaceutical (BMRN) stock has gained about 5% since 5/31/2026 because of the following key factors:
1. Exceptional Fiscal Q2 2026 Financial Results and Increased Full-Year Outlook.
BioMarin Pharmaceutical reported strong financial performance for the second quarter ended June 30, 2026. The company posted adjusted earnings per share (EPS) of $1.20, significantly exceeding the consensus estimate of $0.95. Total revenues for the quarter increased 20% year-over-year to $990 million, surpassing analyst expectations of approximately $932 million. Following these results, BioMarin raised its full-year fiscal year 2026 guidance for total revenues and non-GAAP diluted EPS.
2. Strategic Contributions from Amicus Therapeutics Acquisition and Realized Synergies.
The acquisition of Amicus Therapeutics, which closed on April 27, 2026, played a pivotal role in BioMarin's growth during the period. The newly acquired products, GALAFOLD® and POMBILITI® + OPFOLDA®, contributed to a 25% year-over-year growth in Metabolic Conditions revenue during fiscal Q2 2026. BioMarin projects approximately $220 million in annual non-GAAP cost reductions from this integration, expected to be fully realized by fiscal year 2028, along with an accelerated timeline for achieving its gross leverage target.
3. Advancements and Expanded Potential for VOXZOGO®.
BioMarin achieved significant progress with its key product, VOXZOGO® (vosoritide). The company increased its full-year fiscal year 2026 revenue guidance for VOXZOGO to at least $1 billion, driven by strong global demand. Furthermore, positive Phase 3 CANOPY-HCH-3 study data for VOXZOGO in hypochondroplasia, demonstrating a statistically significant improvement in annualized growth velocity of 2.33 cm/year versus placebo, was published in September 2026. A supplemental New Drug Application (sNDA) for this indication was submitted to the FDA in fiscal Q3 2026, targeting a potential 2027 launch. Additionally, the FDA accepted BioMarin’s sNDA for full approval of VOXZOGO in children with achondroplasia, setting a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.
4. Strengthened Pipeline and Intellectual Property Position.
BioMarin enhanced its pipeline through the acquisition of Alesta Therapeutics in September 2026, adding ALE1 for Hypophosphatasia as an important clinical program. The company also reached a global settlement with Ascendis Pharma on August 31, 2026, which includes royalty payments to BioMarin on future Yuviwel sales in key markets. This settlement is expected to mitigate competitive risk for VOXZOGO and secure a revenue stream through 2030.
Show less
BioMarin Pharmaceutical (BMRN) stock has gained about 5% since 5/31/2026 because of the following key factors:
1. Exceptional Fiscal Q2 2026 Financial Results and Increased Full-Year Outlook.
BioMarin Pharmaceutical reported strong financial performance for the second quarter ended June 30, 2026. The company posted adjusted earnings per share (EPS) of $1.20, significantly exceeding the consensus estimate of $0.95. Total revenues for the quarter increased 20% year-over-year to $990 million, surpassing analyst expectations of approximately $932 million. Following these results, BioMarin raised its full-year fiscal year 2026 guidance for total revenues and non-GAAP diluted EPS.
2. Strategic Contributions from Amicus Therapeutics Acquisition and Realized Synergies.
The acquisition of Amicus Therapeutics, which closed on April 27, 2026, played a pivotal role in BioMarin's growth during the period. The newly acquired products, GALAFOLD® and POMBILITI® + OPFOLDA®, contributed to a 25% year-over-year growth in Metabolic Conditions revenue during fiscal Q2 2026. BioMarin projects approximately $220 million in annual non-GAAP cost reductions from this integration, expected to be fully realized by fiscal year 2028, along with an accelerated timeline for achieving its gross leverage target.
3. Advancements and Expanded Potential for VOXZOGO®.
BioMarin achieved significant progress with its key product, VOXZOGO® (vosoritide). The company increased its full-year fiscal year 2026 revenue guidance for VOXZOGO to at least $1 billion, driven by strong global demand. Furthermore, positive Phase 3 CANOPY-HCH-3 study data for VOXZOGO in hypochondroplasia, demonstrating a statistically significant improvement in annualized growth velocity of 2.33 cm/year versus placebo, was published in September 2026. A supplemental New Drug Application (sNDA) for this indication was submitted to the FDA in fiscal Q3 2026, targeting a potential 2027 launch. Additionally, the FDA accepted BioMarin’s sNDA for full approval of VOXZOGO in children with achondroplasia, setting a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.
4. Strengthened Pipeline and Intellectual Property Position.
BioMarin enhanced its pipeline through the acquisition of Alesta Therapeutics in September 2026, adding ALE1 for Hypophosphatasia as an important clinical program. The company also reached a global settlement with Ascendis Pharma on August 31, 2026, which includes royalty payments to BioMarin on future Yuviwel sales in key markets. This settlement is expected to mitigate competitive risk for VOXZOGO and secure a revenue stream through 2030.
Show less
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Stock Movement Drivers
Fundamental Drivers
The 7.1% change in BMRN stock from 5/31/2026 to 9/24/2026 was primarily driven by a 296.3% change in the company's P/E Multiple.| (LTM values as of) | 5312026 | 9242026 | Change |
|---|---|---|---|
| Stock Price ($) | 57.29 | 61.35 | 7.1% |
| Change Contribution By: | |||
| Total Revenues ($ Mil) | 3,242 | 3,407 | 5.1% |
| Net Income Margin (%) | 8.3% | 2.1% | -74.2% |
| P/E Multiple | 41.0 | 162.6 | 296.3% |
| Shares Outstanding (Mil) | 192 | 193 | -0.5% |
| Cumulative Contribution | 7.1% |
Market Drivers
5/31/2026 to 9/24/2026| Return | Correlation | |
|---|---|---|
| BMRN | 7.1% | |
| Market (SPY) | 1.7% | -1.6% |
| Sector (XLV) | 14.1% | 63.8% |
Fundamental Drivers
The -0.6% change in BMRN stock from 2/28/2026 to 9/24/2026 was primarily driven by a -80.2% change in the company's Net Income Margin (%).| (LTM values as of) | 2282026 | 9242026 | Change |
|---|---|---|---|
| Stock Price ($) | 61.73 | 61.35 | -0.6% |
| Change Contribution By: | |||
| Total Revenues ($ Mil) | 3,221 | 3,407 | 5.8% |
| Net Income Margin (%) | 10.8% | 2.1% | -80.2% |
| P/E Multiple | 34.0 | 162.6 | 378.1% |
| Shares Outstanding (Mil) | 192 | 193 | -0.6% |
| Cumulative Contribution | -0.6% |
Market Drivers
2/28/2026 to 9/24/2026| Return | Correlation | |
|---|---|---|
| BMRN | -0.6% | |
| Market (SPY) | 12.4% | 12.8% |
| Sector (XLV) | 6.9% | 48.5% |
Fundamental Drivers
The 5.3% change in BMRN stock from 8/31/2025 to 9/24/2026 was primarily driven by a 855.8% change in the company's P/E Multiple.| (LTM values as of) | 8312025 | 9242026 | Change |
|---|---|---|---|
| Stock Price ($) | 58.27 | 61.35 | 5.3% |
| Change Contribution By: | |||
| Total Revenues ($ Mil) | 3,064 | 3,407 | 11.2% |
| Net Income Margin (%) | 21.5% | 2.1% | -90.0% |
| P/E Multiple | 17.0 | 162.6 | 855.8% |
| Shares Outstanding (Mil) | 192 | 193 | -0.8% |
| Cumulative Contribution | 5.3% |
Market Drivers
8/31/2025 to 9/24/2026| Return | Correlation | |
|---|---|---|
| BMRN | 5.3% | |
| Market (SPY) | 20.3% | 15.5% |
| Sector (XLV) | 25.8% | 40.8% |
Fundamental Drivers
The -32.9% change in BMRN stock from 8/31/2023 to 9/24/2026 was primarily driven by a -52.1% change in the company's Net Income Margin (%).| (LTM values as of) | 8312023 | 9242026 | Change |
|---|---|---|---|
| Stock Price ($) | 91.38 | 61.35 | -32.9% |
| Change Contribution By: | |||
| Total Revenues ($ Mil) | 2,235 | 3,407 | 52.5% |
| Net Income Margin (%) | 4.5% | 2.1% | -52.1% |
| P/E Multiple | 171.8 | 162.6 | -5.3% |
| Shares Outstanding (Mil) | 188 | 193 | -2.8% |
| Cumulative Contribution | -32.9% |
Market Drivers
8/31/2023 to 9/24/2026| Return | Correlation | |
|---|---|---|
| BMRN | -32.9% | |
| Market (SPY) | 76.8% | 28.6% |
| Sector (XLV) | 34.1% | 43.8% |
Price Returns Compared
| 2021 | 2022 | 2023 | 2024 | 2025 | 2026 | Total [1] | |
|---|---|---|---|---|---|---|---|
| Returns | |||||||
| BMRN Return | 1% | 17% | -7% | -32% | -10% | 5% | -29% |
| Peers Return | 2% | 30% | 6% | 4% | 8% | -1% | 56% |
| S&P 500 Return | 27% | -19% | 24% | 23% | 16% | 13% | 105% |
Monthly Win Rates [3] | |||||||
| BMRN Win Rate | 50% | 67% | 42% | 42% | 42% | 44% | |
| Peers Win Rate | 48% | 63% | 55% | 48% | 57% | 44% | |
| S&P 500 Win Rate | 75% | 42% | 67% | 75% | 67% | 56% | |
Max Drawdowns [4] | |||||||
| BMRN Max Drawdown | -21% | -23% | -35% | -37% | -29% | -22% | |
| Peers Max Drawdown | -31% | -24% | -29% | -30% | -39% | -29% | |
| S&P 500 Max Drawdown | -5% | -25% | -10% | -8% | -19% | -9% | |
[1] Cumulative total returns since the beginning of 2021
[2] Peers: VRTX, SRPT, ALNY, REGN, AMGN. See BMRN Returns vs. Peers.
[3] Win Rate = % of calendar months in which monthly returns were positive
[4] Max drawdown represents maximum peak-to-trough decline within a year
[5] 2026 data is for the year up to 9/24/2026 (YTD)
How Low Can It Go
| Event | BMRN | S&P 500 |
|---|---|---|
| 2025 US Tariff Shock | ||
| % Loss | -15.8% | -18.8% |
| % Gain to Breakeven | 18.7% | 23.1% |
| Time to Breakeven | 431 days | 79 days |
| Summer-Fall 2023 Five Percent Yield Shock | ||
| % Loss | -13.2% | -9.5% |
| % Gain to Breakeven | 15.2% | 10.5% |
| Time to Breakeven | 15 days | 24 days |
| 2022 Inflation Shock & Fed Tightening | ||
| % Loss | -20.0% | -24.5% |
| % Gain to Breakeven | 25.0% | 32.4% |
| Time to Breakeven | 51 days | 427 days |
| 2020 COVID-19 Crash | ||
| % Loss | -20.6% | -33.7% |
| % Gain to Breakeven | 25.9% | 50.9% |
| Time to Breakeven | 28 days | 140 days |
| Q4 2018 Fed Policy Error / Growth Scare | ||
| % Loss | -19.6% | -19.2% |
| % Gain to Breakeven | 24.4% | 23.8% |
| Time to Breakeven | 519 days | 105 days |
| 2016-2017 Trump Reflation Bond Selloff | ||
| % Loss | -18.1% | -3.7% |
| % Gain to Breakeven | 22.1% | 3.9% |
| Time to Breakeven | 174 days | 6 days |
In The Past
BioMarin Pharmaceutical's stock fell -15.8% during the 2025 US Tariff Shock. Such a loss loss requires a 18.7% gain to breakeven.
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| Event | BMRN | S&P 500 |
|---|---|---|
| 2022 Inflation Shock & Fed Tightening | ||
| % Loss | -20.0% | -24.5% |
| % Gain to Breakeven | 25.0% | 32.4% |
| Time to Breakeven | 51 days | 427 days |
| 2020 COVID-19 Crash | ||
| % Loss | -20.6% | -33.7% |
| % Gain to Breakeven | 25.9% | 50.9% |
| Time to Breakeven | 28 days | 140 days |
| 2011 US Debt Ceiling Crisis & European Contagion | ||
| % Loss | -21.8% | -17.9% |
| % Gain to Breakeven | 27.9% | 21.8% |
| Time to Breakeven | 37 days | 123 days |
| 2010 Eurozone Sovereign Debt Crisis / Flash Crash | ||
| % Loss | -21.6% | -15.4% |
| % Gain to Breakeven | 27.6% | 18.2% |
| Time to Breakeven | 108 days | 125 days |
| 2008-2009 Global Financial Crisis | ||
| % Loss | -72.3% | -53.4% |
| % Gain to Breakeven | 260.7% | 114.4% |
| Time to Breakeven | 1033 days | 1085 days |
In The Past
BioMarin Pharmaceutical's stock fell -15.8% during the 2025 US Tariff Shock. Such a loss loss requires a 18.7% gain to breakeven.
Preserve Wealth
Limiting losses and compounding gains is essential to preserving wealth.
Asset Allocation
Actively managed asset allocation strategies protect wealth. Learn more.
About BioMarin Pharmaceutical (BMRN)
BioMarin Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing innovative therapies for individuals living with serious and life-threatening rare diseases and medical conditions. The company's core mission is to address critical unmet medical needs within specialized patient populations by bringing advanced scientific research and product development to market.
The company's commercial portfolio includes several key products designed to treat a range of rare disorders. These therapies include Vimizim and Naglazyme, both enzyme replacement therapies for different types of mucopolysaccharidosis (MPS); Kuvan and Palynziq, which address phenylketonuria (PKU); Brineura, used for a form of Batten disease; Voxzogo, for achondroplasia; and Aldurazyme for MPS I. These products often represent highly specialized treatments for complex genetic and metabolic conditions.
In addition to its marketed products, BioMarin has a robust development pipeline, featuring gene therapies such as valoctocogene roxaparvovec for severe hemophilia A and BMN 307 for PKU, as well as BMN 255 for primary hyperoxaluria. BioMarin serves a global customer base, distributing its specialized therapies to specialty pharmacies, hospitals, and government agencies, along with pharmaceutical wholesalers and distributors across the United States, Europe, Latin America, and other international markets.
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Like Vertex Pharmaceuticals, but focused on a broader spectrum of rare genetic and metabolic diseases.
Amgen, but exclusively dedicated to developing therapies for rare genetic and metabolic disorders.
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- Vimizim: An enzyme replacement therapy used to treat mucopolysaccharidosis (MPS) IV type A.
- Naglazyme: A recombinant enzyme for patients with mucopolysaccharidosis (MPS) VI.
- Kuvan: A proprietary synthetic oral form of 6R-BH4 used to treat patients with phenylketonuria (PKU).
- Palynziq: A PEGylated recombinant enzyme delivered via subcutaneous injection to reduce blood phenylalanine concentrations.
- Brineura: A recombinant human enzyme for the treatment of patients with ceroid lipofuscinosis type 2 (Batten disease).
- Voxzogo: A once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia.
- Aldurazyme: A purified protein designed to be identical to a naturally occurring human enzyme alpha-L-iduronidase.
- Valoctocogene Roxaparvovec: An adeno-associated virus vector gene therapy in Phase III clinical trial for severe hemophilia A.
- BMN 307: An AAV5 mediated gene therapy in Phase 1/2 clinical trial for normalizing blood phenylalanine concentration levels in patients with PKU.
- BMN 255: A therapy in Phase 1/2 clinical trial for treating primary hyperoxaluria.
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Major Customers of BioMarin Pharmaceutical (BMRN)
BioMarin Pharmaceutical primarily sells its therapies to various organizations and commercial entities that then distribute or administer the products to patients. Its major customers include:
- Specialty pharmacies
- Hospitals
- Non-U.S. government agencies
- Distributors
- Pharmaceutical wholesalers
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Alexander Hardy, President and Chief Executive Officer
Alexander Hardy joined BioMarin as President and Chief Executive Officer in December 2023. Before joining BioMarin, he served as the Chief Executive Officer of Genentech, Roche’s research and development arm, for four and a half years.
Brian R. Mueller, Executive Vice President and Chief Financial Officer
Brian R. Mueller joined BioMarin in December 2002 and currently serves as Executive Vice President and Chief Financial Officer, overseeing all Finance and Information Management teams. His previous roles at BioMarin include Senior Vice President, Finance and Chief Accounting Officer, and Group Vice President, Corporate Controller. Prior to BioMarin, Mr. Mueller worked for KPMG as a senior manager in the firm's audit practice, and before that, spent seven years with Arthur Andersen LLP in their audit and business advisory services practice. From June 2014 to May 2020, he was a member of the board of directors and served as Chairman of the Audit Committee for Anthera Pharmaceuticals, Inc., a previously public biopharmaceutical company.
Greg Friberg, Executive Vice President, Chief Research & Development Officer
Greg Friberg assumed the role of Executive Vice President, Chief Research & Development Officer, effective September 30, 2024. He joined BioMarin from Amgen, Inc., where he spent nearly two decades in various leadership positions, most recently as Vice President of Global Medical Affairs for their rare disease drug portfolio. Dr. Friberg brings extensive experience in clinical development and lifecycle management, having been responsible for advancing numerous medicines from investigational new drug application filing through late-stage development.
Cristin Hubbard, Executive Vice President and Chief Commercial Officer
Cristin Hubbard joined BioMarin in May 2024 as Executive Vice President and Chief Commercial Officer, leading the company's global commercial operations and portfolio strategy. She has over 20 years of experience in the biopharmaceutical and diagnostics industries. Most recently, she was the Head of Global Product Strategy for Roche Pharma, where she was responsible for lifecycle management and accelerating the delivery of medicines from development to commercialization across five therapeutic areas. During her more than 16 years at Roche and Genentech, Ms. Hubbard held various leadership positions, including global head of Diagnostics Partnering for Roche Diagnostics, focusing on licensing deals and acquisitions. She began her career as a medicinal chemist at Theravance.
C. Greg Guyer, Executive Vice President and Chief Technical Officer
C. Greg Guyer, Ph.D., joined BioMarin in May 2020 and currently serves as Chief Technical Officer and Executive Vice President of Global Manufacturing and Technical Operations. In this role, he is responsible for manufacturing, process development, quality, supply chain, engineering, and analytical chemistry departments. From 2015 to 2019, Dr. Guyer held several increasingly responsible positions at Bristol Myers Squibb (BMS), primarily leading all operations and biologic development. Before BMS, he worked for Merck & Co., Inc. from 1994 to 2015, where he led various global organizations in biologic and pharmaceutical operations, quality, regulatory, emerging markets strategy, and enterprise systems. He started his career at the FDA in the lab and rose to Division Director for Generic Drugs over 10 years.
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The key risks for BioMarin Pharmaceutical (BMRN) include:
- Clinical Development and Regulatory Approval of Gene Therapies: BioMarin has made substantial investments in its gene therapy pipeline, notably with valoctocogene roxaparvovec, an adeno associated virus vector in Phase III clinical trials for severe hemophilia A, and BMN 307, an AAV5 mediated gene therapy in Phase I/II for phenylketonuria (PKU). The successful development, manufacturing, and obtaining regulatory approval for these complex and novel gene therapies are critical for the company's future growth. Failures in late-stage clinical trials, unexpected safety concerns, or significant delays in regulatory processes could severely impact BioMarin's financial performance and long-term prospects.
- Patent Expiry and Generic/Biosimilar Competition: BioMarin's revenue is largely derived from its portfolio of commercialized therapies for rare diseases. As these products mature, they become vulnerable to patent expiration, which can lead to the entry of generic or biosimilar competitors. For instance, Kuvan, a proprietary synthetic oral form of 6R-BH4 for PKU, is an older commercial product that could face increased competition as its market exclusivity periods conclude. The loss of patent protection for key revenue-generating products could result in substantial revenue erosion.
- Market Access, Pricing Pressure, and Competition for Commercialized Rare Disease Therapies: Despite operating in the rare disease space, BioMarin's existing commercial products, such as Vimizim, Naglazyme, Palynziq, Brineura, Voxzogo, and Aldurazyme, face ongoing challenges related to market access, reimbursement negotiations, and pricing scrutiny from healthcare payers globally. Additionally, while these therapies address niche patient populations, the rare disease market can still experience competition from new entrants, or the development of alternative or improved treatments could erode the market share and profitability of BioMarin's current product portfolio.
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BioMarin Pharmaceutical Inc. develops and commercializes therapies for serious and life-threatening rare diseases and medical conditions. The addressable markets for its main products and services are detailed below:
- Vimizim (mucopolysaccharidosis (MPS) IV type A): The global market for Morquio A syndrome therapies, for which Vimizim is the only FDA-approved biologic, is limited by the rarity of the disease. The estimated global Morquio A patient population is between 1,000 and 2,000 patients. The global Morquio Syndrome (MPS-IV) drug market size was US$ 1.9 million in 2025 and is expected to reach US$ 4.4 million by 2032.
- Naglazyme (MPS VI): The global market for Naglazyme is estimated at approximately $300 million annually. In 2023, Naglazyme sales were approximately $303 million.
- Kuvan and Palynziq (phenylketonuria (PKU)): The global phenylketonuria (PKU) market size was valued at USD 1.17 billion in 2023 and is estimated to reach USD 2.02 billion by 2032. Another source indicates the global Phenylketonuria Treatment Market size is estimated to be valued at USD 1.2 billion in 2025 and is expected to reach USD 2.1 billion by 2032. In 2023, the Phenylketonuria Market Size was approximately USD 700 million, with the United States accounting for about 60% of the total market size across the 7 Major Markets (7MM).
- Brineura (ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease): The global Batten disease drug pipeline market is projected to reach $60.65 million by 2025. The 7 major Batten disease markets are expected to exhibit a compound annual growth rate (CAGR) of 5.34%.
- Voxzogo (achondroplasia): The market size of achondroplasia in the 7MM is estimated to be around USD 300 million in 2024, with projections to grow to approximately USD 1.5 billion by 2034. Another report states the 7 major achondroplasia markets reached a value of USD 136.8 million in 2024 and are expected to reach USD 655.5 million by 2035. BioMarin estimates the potential market could encompass up to 420,000 patients across indications globally. For hypochondroplasia, an expansion for Voxzogo, BioMarin estimates a total addressable global population of about 14,000 diagnosed patients.
- Aldurazyme (MPS I): The Mucopolysaccharidosis I Market in the 7MM was valued at approximately USD 145 million in 2024 and is projected to grow at a CAGR of 8.0% from 2025 to 2034. In 2024, the MPS I market size in the U.S. was around USD 70 million, accounting for 48% of the total 7MM market.
- valoctocogene roxaparvovec (severe hemophilia A): Market size information for this specific product or the severe hemophilia A gene therapy market was not available in the search results.
- BMN 307 (PKU): This product is in clinical trials, and its addressable market is covered by the overall Phenylketonuria (PKU) market detailed above for Kuvan and Palynziq.
- BMN 255 (primary hyperoxaluria): The Primary Hyperoxaluria (PH) Treatment Market in the 7MM was approximately USD 316 million in 2023. The global primary hyperoxaluria treatment market reached US$ 168.96 million in 2023 and is expected to reach US$ 365.49 million by 2031. The United States accounted for approximately USD 281 million of the 7MM market in 2023, representing about 89% of that total.
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BioMarin Pharmaceutical Inc. (BMRN) expects its future revenue growth over the next 2-3 years to be driven by several key factors:
- Continued Growth of Voxzogo: Voxzogo, a treatment for achondroplasia, is anticipated to be a significant growth driver. The company expects sustained high patient demand and deeper market penetration, particularly in international markets. Voxzogo achieved 26% year-over-year revenue growth in 2025 and is projected to see high single-digit growth rates in 2026, with anticipated sales reaching $975 million to $1.025 billion. Further expansion is expected from potential approval for additional indications, such as hypochondroplasia, with Phase 3 data expected in the first half of 2026.
- Sustained Growth in Enzyme Therapies: BioMarin's portfolio of enzyme therapies, including products like Naglazyme, Vimizim, Palynziq, and Aldurazyme, consistently demonstrates strong performance. This segment grew by 9% in 2025, with Palynziq showing robust 22% growth. BioMarin forecasts enzyme therapies revenue to be between $2.225 billion and $2.275 billion in 2026, driven by high market penetration and patient adherence across its extensive global footprint.
- Acquisition of Amicus Therapeutics: The acquisition of Amicus Therapeutics, expected to close in the second quarter of 2026, is a key strategic move to bolster BioMarin's revenue. This acquisition will add high-growth products Galafold for Fabry disease and Pombiliti + Opfolda for Pompe disease to BioMarin's commercial portfolio. This is anticipated to provide a meaningful uplift to BioMarin's total revenue growth rate and become significantly accretive starting in 2027. An adolescent label expansion for Pombiliti + Opfolda is also anticipated in the second half of 2026.
- Advancement of Pipeline Candidates: BioMarin has several promising pipeline candidates that could contribute to future revenue growth. These include BMN 401 for ENPP1 deficiency, which is expected to have Phase 3 data read out in the first half of 2026, and BMN 333, a long-acting CNP for achondroplasia currently in a Phase 2/3 study. The company continues to invest in innovation and advance its clinical-stage assets, indicating potential for new product launches or expanded indications in the coming years.
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Share Repurchases
- BioMarin Pharmaceutical has not engaged in significant share repurchase programs over the last 3-5 years, with the latest available data indicating a negative or null share buyback ratio.
Share Issuance
- The number of BioMarin Pharmaceutical's common shares issued and outstanding saw a minor increase, reaching 192,300,091 shares by the end of 2025, up from 190,761,349 shares.
Outbound Investments
- In December 2025, BioMarin entered a definitive agreement to acquire Amicus Therapeutics for $4.8 billion in an all-cash transaction, expected to close in the second quarter of 2026. This acquisition is intended to accelerate and diversify BioMarin's revenue streams by adding therapies for Fabry and Pompe diseases.
- The company completed the acquisition of Inozyme Pharma in July 2025 for $270 million, which added BMN 401 for ENPP1 Deficiency to its pipeline.
Capital Expenditures
- BioMarin's annual capital expenditures were $119.2 million in 2021, $131.5 million in 2022, $107.6 million in 2023, and $97.4 million in 2024.
- Projected capital expenditures are $176 million for 2025, $193 million for 2026, and $211 million for 2027.
- These capital expenditures are primarily focused on supporting investment in innovation and future growth initiatives.
Peer Outperformance in Biotechnology
BMRN has outperformed 72% of its 377 Biotechnology peers over 5Y. Biotechnology ranks 10th of 10 industries in Health Care by median 5Y return. The sector’s gains have largely come from elsewhere.
Share of Biotechnology constituents that BMRN has outperformed, by holding period. Peers without a full price history over a given window are excluded from that window.
1Y
59%
of 468 industry peers · 14.1% return
3Y
48%
of 418 industry peers · -29.5% return
5Y
72%
of 377 industry peers · -21.9% return
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Median price return by industry across the Health Care sector, ranked by 5Y. Biotechnology is BMRN's own industry. Use this to see which corners of the sector have carried the returns.
| Industry | Names | 1Y med | 3Y med | 5Y med | Top 3 by 5Y |
|---|---|---|---|---|---|
| Health Care Distributors | 6 | 8.0% | 42.8% | 88.3% | CAH 385% · MCK 339% · COR 167% |
| Health Care Services | 20 | 18.6% | 31.5% | 3.3% | HIMS 261% · RDNT 144% · DGX 70% |
| Managed Health Care | 8 | 32.3% | -10.0% | 0.5% | OSCR 70% · HQY 34% · ELV 12% |
| Health Care Facilities | 24 | 1.9% | 6.9% | -4.8% | NHC 266% · THC 250% · ENSG 132% |
| Health Care Equipment | 50 | -3.5% | -2.2% | -19.2% | POCI 11838% · UFPT 380% · GKOS 206% |
| Health Care Supplies | 12 | 24.2% | -7.1% | -38.0% | LNTH 270% · HAE 53% · MMSI 21% |
| Pharmaceuticals | 63 | -8.1% | 6.9% | -41.8% | LQDA 2372% · INDV 1100% · ETON 1087% |
| Life Sciences Tools & Services | 113 | -0.4% | -10.9% | -67.1% | SNWV 1880% · NTRA 246% · MEDP 213% |
| Health Care Technology | 21 | -29.4% | -52.6% | -78.6% | SPOK 70% · HSTM 1% · VEEV -8% |
| Biotechnology ← | 378 | -8.8% | -22.0% | -79.4% | CELZ 1286% · DNTH 1114% · ELVN 975% |
Median of constituents with a full price history over each window. Top names are unfiltered by size in this render.
Latest Trefis Analyses
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Wealth Management
Peer Comparisons
| Peers to compare with: |
Financials
| Median | |
|---|---|
| Name | |
| Mkt Price | 327.81 |
| Mkt Cap | 57.6 |
| Rev LTM | 8,696 |
| Op Inc LTM | 2,587 |
| FCF LTM | 2,198 |
| FCF 3Y Avg | 1,250 |
| CFO LTM | 2,602 |
| CFO 3Y Avg | 1,525 |
Growth & Margins
| Median | |
|---|---|
| Name | |
| Rev Chg LTM | 9.8% |
| Rev Chg 3Y Avg | 14.0% |
| Rev Chg Q | 14.6% |
| QoQ Delta Rev Chg LTM | 3.6% |
| Op Inc Chg LTM | 22.8% |
| Op Inc Chg 3Y Avg | 46.3% |
| Op Mgn LTM | 23.8% |
| Op Mgn 3Y Avg | 18.8% |
| QoQ Delta Op Mgn LTM | 0.5% |
| CFO/Rev LTM | 27.6% |
| CFO/Rev 3Y Avg | 20.3% |
| FCF/Rev LTM | 22.1% |
| FCF/Rev 3Y Avg | 16.5% |
Valuation
| Median | |
|---|---|
| Name | |
| Mkt Cap | 57.6 |
| P/S | 5.5 |
| P/Op Inc | 23.7 |
| P/EBIT | 21.6 |
| P/E | 27.6 |
| P/CFO | 17.5 |
| Total Yield | 2.9% |
| Dividend Yield | 0.0% |
| FCF Yield 3Y Avg | 3.2% |
| D/E | 0.1 |
| Net D/E | 0.0 |
Returns
| Median | |
|---|---|
| Name | |
| 1M Rtn | -5.0% |
| 3M Rtn | 11.8% |
| 6M Rtn | 8.0% |
| 12M Rtn | 26.4% |
| 3Y Rtn | 21.2% |
| 1M Excs Rtn | -5.4% |
| 3M Excs Rtn | 7.1% |
| 6M Excs Rtn | -8.4% |
| 12M Excs Rtn | 10.5% |
| 3Y Excs Rtn | -54.3% |
Comparison Analyses
FDA Approved Drugs Data
Expand for More| Post-Approval Fwd Returns | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| FDA App # | Brand Name | Generic Name | Dosage Form | FDA Approval | 3M Rtn | 6M Rtn | 1Y Rtn | 2Y Rtn | Total Rtn |
| NDA214938 | VOXZOGO | vosoritide | powder | 11192021 | -7.2% | -12.8% | -3.5% | -4.3% | -32.9% |
| BLA761079 | PALYNZIQ | pegvaliase-pqpz | injectable | 5242018 | 12.2% | 6.0% | 0.9% | 10.5% | -30.5% |
| BLA761052 | BRINEURA | cerliponase alfa | injectable | 4272017 | -9.3% | -14.3% | -12.4% | -11.6% | -36.3% |
| BLA125460 | VIMIZIM | elosulfase alfa | injectable | 2142014 | -24.0% | -11.3% | 32.9% | -7.6% | -19.1% |
| NDA205065 | KUVAN | sapropterin dihydrochloride | for solution | 12192013 | 14.9% | -8.7% | 37.3% | 52.8% | -10.3% |
| NDA022181 | KUVAN | sapropterin dihydrochloride | tablet | 12132007 | 16.6% | 15.8% | -42.9% | -42.4% | 106.1% |
| Post-Approval Fwd Returns | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| FDA App # | Brand Name | Generic Name | Dosage Form | FDA Approval | 3M Rtn | 6M Rtn | 1Y Rtn | 2Y Rtn | Total Rtn |
| NDA214938 | VOXZOGO | vosoritide | powder | 11192021 | -7.2% | -12.8% | -3.5% | -4.3% | -32.9% |
| BLA761079 | PALYNZIQ | pegvaliase-pqpz | injectable | 5242018 | 12.2% | 6.0% | 0.9% | 10.5% | -30.5% |
| BLA761052 | BRINEURA | cerliponase alfa | injectable | 4272017 | -9.3% | -14.3% | -12.4% | -11.6% | -36.3% |
| BLA125460 | VIMIZIM | elosulfase alfa | injectable | 2142014 | -24.0% | -11.3% | 32.9% | -7.6% | -19.1% |
| NDA205065 | KUVAN | sapropterin dihydrochloride | for solution | 12192013 | 14.9% | -8.7% | 37.3% | 52.8% | -10.3% |
| NDA022181 | KUVAN | sapropterin dihydrochloride | tablet | 12132007 | 16.6% | 15.8% | -42.9% | -42.4% | 106.1% |
Clinical Pipeline
| Drug Candidate | Indication | Phase 3 Trials | Est. Primary Completion |
|---|---|---|---|
| BMN 333 | Achondroplasia | 1 | Jun 2029 |
| Vosoritide (BMN 111) | Achondroplasia and hypochondroplasia | 3 | Jun 2031 |
| Pegvaliase | Phenylketonuria | 1 | Jan 2025 |
| Valoctocogene roxaparvovec | Severe hemophilia A | 1 | Apr 2025 |
Active Phase 3 studies led by the company. Source: ClinicalTrials.gov · Updated 8/28/2026
Segment Financials
Revenue by Segment| $ Mil | 2025 | 2024 | 2023 | 2022 | 2021 |
|---|---|---|---|---|---|
| VOXZOGO | 927 | 735 | 470 | 169 | 6 |
| VIMIZIM | 792 | 740 | 701 | 664 | 623 |
| NAGLAZYME | 485 | 480 | 420 | 444 | 380 |
| PALYNZIQ | 433 | 355 | 304 | 255 | 237 |
| ALDURAZYME | 209 | 184 | 131 | 128 | 123 |
| BRINEURA | 186 | 169 | 162 | 154 | 128 |
| KUVAN | 100 | 121 | 181 | 228 | 286 |
| Royalty and other revenues | 53 | 44 | 47 | 54 | 63 |
| ROCTAVIAN | 36 | 26 | 3 | 0 | 0 |
| Total | 3,221 | 2,854 | 2,419 | 2,096 | 1,846 |
Price Behavior
Price Behavior
| Market Price | $61.35 | |
| Market Cap ($ Bil) | 11.9 | |
| First Trading Date | 07/26/1999 | |
| Distance from 52W High | -12.7% | |
| 50 Days | 200 Days | |
| DMA Price | $63.98 | $58.49 |
| DMA Trend | up | up |
| Distance from DMA | -4.1% | 4.9% |
| 3M | 1YR | |
| Volatility | 30.3% | 35.2% |
| Downside Capture | 4.83 | 40.76 |
| Upside Capture | 37.00 | 48.49 |
| Correlation (SPY) | 0.7% | 16.8% |
BMRN Betas & Captures as of 8/31/2026
[1] Upside and downside betas calculated using positive and negative benchmark daily returns respectively
| 1M | 2M | 3M | 6M | 1Y | 3Y | |
|---|---|---|---|---|---|---|
| Beta | 0.55 | 0.08 | -0.18 | 0.28 | 0.41 | 0.62 |
| Up Beta | -0.17 | -0.76 | -0.64 | 0.18 | 0.58 | 0.61 |
| Down Beta | 6.16 | 1.39 | 0.83 | 0.72 | 0.50 | 0.83 |
| Up Capture | 90% | 54% | -7% | 17% | 24% | 12% |
| Bmk +ve Days | 10 | 21 | 32 | 68 | 138 | 427 |
| Stock +ve Days | 10 | 21 | 32 | 60 | 120 | 372 |
| Down Capture | -71% | -53% | -88% | 14% | 32% | 84% |
| Bmk -ve Days | 11 | 21 | 32 | 59 | 113 | 324 |
| Stock -ve Days | 11 | 21 | 32 | 67 | 131 | 377 |
[1] Upside and downside betas calculated using positive and negative benchmark daily returns respectively
Based On 1-Year Data
| Annualized Return | Annualized Volatility | Sharpe Ratio | Correlation with BMRN | |
|---|---|---|---|---|
| BMRN | 14.9% | 35.1% | 0.44 | - |
| Sector ETF (XLV) | 25.8% | 16.2% | 1.23 | 40.2% |
| Equity (SPY) | 16.7% | 13.0% | 0.92 | 16.8% |
| Gold (GLD) | 13.1% | 29.3% | 0.42 | 10.6% |
| Commodities (DBC) | 45.1% | 20.7% | 1.71 | -7.2% |
| Real Estate (VNQ) | 3.7% | 13.7% | 0.01 | 11.0% |
| Bitcoin (BTCUSD) | -24.8% | 44.8% | -0.51 | 5.4% |
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Based On 5-Year Data
| Annualized Return | Annualized Volatility | Sharpe Ratio | Correlation with BMRN | |
|---|---|---|---|---|
| BMRN | -4.4% | 32.9% | -0.09 | - |
| Sector ETF (XLV) | 6.8% | 15.2% | 0.26 | 44.3% |
| Equity (SPY) | 12.9% | 17.2% | 0.57 | 35.9% |
| Gold (GLD) | 19.0% | 18.8% | 0.82 | 4.4% |
| Commodities (DBC) | 10.6% | 19.6% | 0.42 | -1.1% |
| Real Estate (VNQ) | 0.7% | 18.9% | -0.07 | 31.0% |
| Bitcoin (BTCUSD) | 12.0% | 52.6% | 0.41 | 17.6% |
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Based On 10-Year Data
| Annualized Return | Annualized Volatility | Sharpe Ratio | Correlation with BMRN | |
|---|---|---|---|---|
| BMRN | -4.4% | 35.0% | -0.03 | - |
| Sector ETF (XLV) | 10.9% | 16.7% | 0.53 | 49.1% |
| Equity (SPY) | 15.5% | 17.9% | 0.73 | 43.3% |
| Gold (GLD) | 12.0% | 16.4% | 0.60 | 6.6% |
| Commodities (DBC) | 8.5% | 18.1% | 0.39 | 9.8% |
| Real Estate (VNQ) | 4.8% | 20.7% | 0.19 | 32.4% |
| Bitcoin (BTCUSD) | 63.7% | 66.2% | 1.03 | 13.6% |
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Returns Analyses
Earnings Returns History
Updated 9/9/2026| Forward Returns | |||
|---|---|---|---|
| Earnings Date | 1D Returns | 5D Returns | 21D Returns |
| 8/6/2026 | 3.8% | 11.5% | 8.2% |
| 5/4/2026 | -4.0% | -5.2% | -1.4% |
| 2/23/2026 | -0.5% | -5.1% | -14.0% |
| 10/27/2025 | 1.6% | 0.3% | 6.9% |
| 8/4/2025 | 2.7% | -5.8% | -2.2% |
| 5/1/2025 | -1.1% | -4.6% | -9.0% |
| 2/19/2025 | 4.8% | 4.9% | 9.1% |
| 10/29/2024 | -4.6% | -5.5% | -6.4% |
| ... | |||
| SUMMARY STATS | |||
| # Positive | 12 | 10 | 11 |
| # Negative | 13 | 15 | 14 |
| Median Positive | 2.8% | 5.1% | 7.4% |
| Median Negative | -4.0% | -5.2% | -5.0% |
| Max Positive | 9.0% | 16.4% | 22.1% |
| Max Negative | -9.9% | -8.7% | -16.8% |
| Forward Returns | |||
|---|---|---|---|
| Earnings Date | 1D Returns | 5D Returns | 21D Returns |
| 8/6/2026 | 3.8% | 11.5% | 8.2% |
| 5/4/2026 | -4.0% | -5.2% | -1.4% |
| 2/23/2026 | -0.5% | -5.1% | -14.0% |
| 10/27/2025 | 1.6% | 0.3% | 6.9% |
| 8/4/2025 | 2.7% | -5.8% | -2.2% |
| 5/1/2025 | -1.1% | -4.6% | -9.0% |
| 2/19/2025 | 4.8% | 4.9% | 9.1% |
| 10/29/2024 | -4.6% | -5.5% | -6.4% |
| 8/5/2024 | 8.0% | 10.6% | 11.7% |
| 4/24/2024 | -9.9% | -8.6% | -16.8% |
| 2/22/2024 | 1.4% | -4.1% | -5.4% |
| 11/1/2023 | -5.2% | 1.8% | 10.0% |
| 7/31/2023 | 1.2% | -0.1% | 7.4% |
| 4/26/2023 | -0.6% | 0.6% | -4.6% |
| 2/27/2023 | -3.5% | -8.7% | -11.4% |
| 1/11/2023 | 2.2% | -1.8% | -1.5% |
| 10/26/2022 | -6.9% | -8.6% | 5.2% |
| 8/3/2022 | 5.2% | 8.6% | 2.8% |
| 4/27/2022 | 2.9% | 5.4% | -1.8% |
| 2/23/2022 | -6.3% | -5.3% | -2.0% |
| 10/27/2021 | 9.0% | 16.4% | 22.1% |
| 7/28/2021 | -0.3% | -2.9% | 0.1% |
| 4/29/2021 | -2.8% | -2.0% | -3.6% |
| 2/25/2021 | -5.3% | -6.6% | -7.7% |
| 11/5/2020 | 2.3% | 2.5% | 4.5% |
| SUMMARY STATS | |||
| # Positive | 12 | 10 | 11 |
| # Negative | 13 | 15 | 14 |
| Median Positive | 2.8% | 5.1% | 7.4% |
| Median Negative | -4.0% | -5.2% | -5.0% |
| Max Positive | 9.0% | 16.4% | 22.1% |
| Max Negative | -9.9% | -8.7% | -16.8% |
SEC Filings
Expand for More| Report Date | Filing Date | Filing |
|---|---|---|
| 06/30/2026 | 08/06/2026 | 10-Q |
| 03/31/2026 | 05/05/2026 | 10-Q |
| 12/31/2025 | 02/26/2026 | 10-K |
| 09/30/2025 | 10/28/2025 | 10-Q |
| 06/30/2025 | 08/05/2025 | 10-Q |
| 03/31/2025 | 05/02/2025 | 10-Q |
| 12/31/2024 | 02/24/2025 | 10-K |
| 09/30/2024 | 10/31/2024 | 10-Q |
| 06/30/2024 | 08/05/2024 | 10-Q |
| 03/31/2024 | 04/26/2024 | 10-Q |
| 12/31/2023 | 02/26/2024 | 10-K |
| 09/30/2023 | 11/02/2023 | 10-Q |
| 06/30/2023 | 08/02/2023 | 10-Q |
| 03/31/2023 | 04/28/2023 | 10-Q |
| 12/31/2022 | 02/27/2023 | 10-K |
| 09/30/2022 | 10/28/2022 | 10-Q |
| Report Date | Filing Date | Filing |
|---|---|---|
| 06/30/2026 | 08/06/2026 | 10-Q |
| 03/31/2026 | 05/05/2026 | 10-Q |
| 12/31/2025 | 02/26/2026 | 10-K |
| 09/30/2025 | 10/28/2025 | 10-Q |
| 06/30/2025 | 08/05/2025 | 10-Q |
| 03/31/2025 | 05/02/2025 | 10-Q |
| 12/31/2024 | 02/24/2025 | 10-K |
| 09/30/2024 | 10/31/2024 | 10-Q |
| 06/30/2024 | 08/05/2024 | 10-Q |
| 03/31/2024 | 04/26/2024 | 10-Q |
| 12/31/2023 | 02/26/2024 | 10-K |
| 09/30/2023 | 11/02/2023 | 10-Q |
| 06/30/2023 | 08/02/2023 | 10-Q |
| 03/31/2023 | 04/28/2023 | 10-Q |
| 12/31/2022 | 02/27/2023 | 10-K |
| 09/30/2022 | 10/28/2022 | 10-Q |
| 06/30/2022 | 08/04/2022 | 10-Q |
| 03/31/2022 | 04/29/2022 | 10-Q |
| 12/31/2021 | 02/25/2022 | 10-K |
| 09/30/2021 | 10/29/2021 | 10-Q |
| 06/30/2021 | 07/30/2021 | 10-Q |
| 03/31/2021 | 04/30/2021 | 10-Q |
| 12/31/2020 | 02/26/2021 | 10-K |
| 09/30/2020 | 11/06/2020 | 10-Q |
| 06/30/2020 | 08/05/2020 | 10-Q |
| 03/31/2020 | 05/01/2020 | 10-Q |
| 12/31/2019 | 02/27/2020 | 10-K |
| 09/30/2019 | 10/29/2019 | 10-Q |
Recent Forward Guidance
Updated 8/7/2026Latest: Q2 2026 Earnings Reported 8/6/2026
| Forward Guidance | Guidance Change | |||||||
|---|---|---|---|---|---|---|---|---|
| Metric | Source | Low | Mid | High | % Chg | % Delta | Change | Prior |
| 2026 Total Revenues | Reported | 3.88 Bil | 3.90 Bil | 3.92 Bil | 0.6% | Raised | Guidance: 3.88 Bil for 2026 | |
| 2026 Metabolic Conditions Revenue | Reported | 2.73 Bil | 2.75 Bil | 2.77 Bil | ||||
| 2026 VOXZOGO Revenue | Reported | 1.00 Bil | 1.02 Bil | 1.05 Bil | ||||
| 2026 Non-GAAP Diluted EPS | Reported | 4.9 | 5 | 5.1 | 1.0% | Raised | Guidance: 4.95 for 2026 | |
| 2035 GALAFOLD Peak Revenue | Reported | 1.40 Bil | ||||||
| 2035 POMBILITI + OPFOLDA Peak Revenue | Reported | 1.20 Bil | ||||||
| 2030 Non-GAAP Operating Margin | Reported | 60.0% | ||||||
Prior: Q1 2026 Earnings Reported 5/4/2026
| Forward Guidance | Guidance Change | |||||||
|---|---|---|---|---|---|---|---|---|
| Metric | Source | Low | Mid | High | % Chg | % Delta | Change | Prior |
| 2026 Total Revenues | Reported | 3.83 Bil | 3.88 Bil | 3.92 Bil | 14.8% | Raised | Guidance: 3.38 Bil for 2026 | |
| 2026 Enzyme Therapies Revenue | Reported | 2.73 Bil | 2.75 Bil | 2.77 Bil | 22.2% | Raised | Guidance: 2.25 Bil for 2026 | |
| 2026 Non-GAAP Diluted EPS | Reported | 4.85 | 4.95 | 5.05 | -2.0% | Lowered | Guidance: 5.05 for 2026 | |
Q4 2025 Earnings Reported 2/23/2026
| Forward Guidance | Guidance Change | |||||||
|---|---|---|---|---|---|---|---|---|
| Metric | Source | Low | Mid | High | % Chg | % Delta | Change | Prior |
| 2026 Revenue | Reported | 3.33 Bil | 3.38 Bil | 3.42 Bil | 6.3% | Higher New | Actual: 3.17 Bil for 2025 | |
| 2026 Enzyme Therapies Revenue | Reported | 2.23 Bil | 2.25 Bil | 2.27 Bil | ||||
| 2026 VOXZOGO Revenue | Reported | 975.00 Mil | 1.00 Bil | 1.02 Bil | 9.0% | Higher New | Actual: 917.50 Mil for 2025 | |
| 2026 Other Revenues | Reported | 100.00 Mil | 112.50 Mil | 125.00 Mil | ||||
| 2026 Non-GAAP Operating Margin | Reported | 40.0% | 13.5% | Higher New | Actual: 26.5% for 2025 | |||
| 2026 Non-GAAP Diluted EPS | Reported | 4.95 | 5.05 | 5.15 | 42.3% | Higher New | Actual: 3.55 for 2025 | |
Q3 2025 Earnings Reported 10/27/2025
| Forward Guidance | Guidance Change | |||||||
|---|---|---|---|---|---|---|---|---|
| Metric | Source | Low | Mid | High | % Chg | % Delta | Change | Prior |
| 2025 Total Revenues | Reported | 3.15 Bil | 3.17 Bil | 3.20 Bil | 0.4% | Raised | Guidance: 3.16 Bil for 2025 | |
| 2025 VOXZOGO Revenue | Reported | 900.00 Mil | 917.50 Mil | 935.00 Mil | 0 | Affirmed | Guidance: 917.50 Mil for 2025 | |
| 2025 Non-GAAP Operating Margin | Reported | 26.0% | 26.5% | 27.0% | -7.0% | Lowered | Guidance: 33.5% for 2025 | |
| 2025 Non-GAAP Diluted EPS | Reported | 3.5 | 3.55 | 3.6 | -20.7% | Lowered | Guidance: 4.47 for 2025 | |
Insider Activity
Updated 9/3/2026| # | Owner | Title | Holding | Action | Filing Date | Price | Shares | Transacted Value | Value of Held Shares | Form |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | Davis, George Eric | EVP, Chief Legal Officer | Direct | Sell | 9032026 | 66.28 | 17,557 | 1,163,748 | 4,043,523 | Form |
| 2 | Friberg, Gregory R | EVP, Chief R&D Officer | Direct | Sell | 5112026 | 53.85 | 3,281 | 176,682 | 2,790,399 | Form |
| 3 | Guyer, Charles Greg | EVP, Chief Technical Officer | Direct | Sell | 3122026 | 60.46 | 16,486 | 996,744 | 4,833,958 | Form |
| 4 | Friberg, Gregory R | EVP, Chief R&D Officer | Direct | Sell | 2262026 | 60.38 | 6,326 | 381,937 | 2,268,802 | Form |
| 5 | Davis, George Eric | EVP, Chief Legal Officer | Direct | Sell | 2262026 | 61.36 | 26,061 | 1,599,103 | 4,445,716 | Form |
| # | Owner | Title | Holding | Action | Filing Date | Price | Shares | Transacted Value | Value of Held Shares | Form |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | Davis, George Eric | EVP, Chief Legal Officer | Direct | Sell | 9032026 | 66.28 | 17,557 | 1,163,748 | 4,043,523 | Form |
| 2 | Friberg, Gregory R | EVP, Chief R&D Officer | Direct | Sell | 5112026 | 53.85 | 3,281 | 176,682 | 2,790,399 | Form |
| 3 | Guyer, Charles Greg | EVP, Chief Technical Officer | Direct | Sell | 3122026 | 60.46 | 16,486 | 996,744 | 4,833,958 | Form |
| 4 | Friberg, Gregory R | EVP, Chief R&D Officer | Direct | Sell | 2262026 | 60.38 | 6,326 | 381,937 | 2,268,802 | Form |
| 5 | Davis, George Eric | EVP, Chief Legal Officer | Direct | Sell | 2262026 | 61.36 | 26,061 | 1,599,103 | 4,445,716 | Form |
Investor Activity (13F)
Updated Sep 25, 202613F holdings as of Mar 31, 2026 (Q1 2026)
Active managers (13F portfolio over $250M, at least 3 holdings) with a position over $5M that is either over 10% of their portfolio or held in a concentrated book of 50 or fewer total positions. Index/ETF, sovereign, bank, community-bank and charitable/donor-advised filers are excluded.
| Active Manager | Value | % of Portfolio | Total Positions | QoQ | Filing |
|---|---|---|---|---|---|
| Jones Hill Capital LP | $71.7 Mil | 15.4% | 26 | New | 13F |
| Boone Capital Management LLC | $30.9 Mil | 9.5% | 13 | ADD +33.0% | 13F |
| Eagle Health Investments LP | $28.6 Mil | 6.0% | 26 | ADD +37.9% | 13F |
| Sector Gamma AS | $13.6 Mil | 3.2% | 42 | ADD +20.9% | 13F |
| Novo Holdings A/S | $37.3 Mil | 3.0% | 22 | Hold | 13F |
| Opti Capital Management, LP | $27.1 Mil | 2.0% | 36 | TRIM -12.5% | 13F |
| Redmile Group, LLC | $9.8 Mil | 0.7% | 35 | New | 13F |
| Active Manager | Value | % of Portfolio | Total Positions | QoQ | Filing |
|---|---|---|---|---|---|
| Jones Hill Capital LP | $71.7 Mil | 15.4% | 26 | New | 13F |
| Novo Holdings A/S | $37.3 Mil | 3.0% | 22 | Hold | 13F |
| Boone Capital Management LLC | $30.9 Mil | 9.5% | 13 | ADD +33.0% | 13F |
| Eagle Health Investments LP | $28.6 Mil | 6.0% | 26 | ADD +37.9% | 13F |
| Opti Capital Management, LP | $27.1 Mil | 2.0% | 36 | TRIM -12.5% | 13F |
| Sector Gamma AS | $13.6 Mil | 3.2% | 42 | ADD +20.9% | 13F |
| Redmile Group, LLC | $9.8 Mil | 0.7% | 35 | New | 13F |
Industry Resources
External Quote Links
| Y Finance | Barrons |
| TradingView | Morningstar |
| SeekingAlpha | ValueLine |
| Motley Fool | Robinhood |
| CNBC | Etrade |
| MarketWatch | Unusual Whales |
| YCharts | Perplexity Finance |
| FinViz |
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Core Cache Last Updated: 9/24/2026