Ultragenyx Pharmaceutical (RARE)
Market Price (9/3/2026): $14.11 | Market Cap: $1.4 BilSector: Health Care | Industry: Biotechnology
Ultragenyx Pharmaceutical (RARE)
Market Price (9/3/2026): $14.11Market Cap: $1.4 BilSector: Health CareIndustry: Biotechnology
Investment Highlights Why It Matters Detailed financial logic regarding cash flow yields vs trend-riding momentum.
Cash is significant % of market capNet D/ENet Debt/Equity. Debt net of cash. Negative indicates net cash. Equity is taken as the Market Capitalization is -11% 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 -91%, 3Y Excs Rtn is -101% Meaningful short interestShort Interest % of Basic SharesShort Interest % of Basic Shares = (Short Interest Quantity) / (Basic Shares Outstanding). A high fraction of short interest can indicate potential risk of a short squeeze. is 16% | Not profitable at operating income levelOp Inc LTMOperating Income, Last Twelve Months is -528 Mil, Op Mgn LTMOperating Margin = Operating Income / Revenue Reflects profitability before taxes and before impact of capital structure (interest payments). is -74% Significant share based compensationSBC/Rev LTMShare Based Compensation / Revenue (Sales), Last Twelve Months (LTM) is 19% Not cash flow generativeCFO/Rev LTMCash Flow from Operations / Revenue (Sales), Last Twelve Months (LTM) is -68%, FCF/Rev LTMFree Cash Flow / Revenue (Sales), Last Twelve Months (LTM) is -69% 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 -26% Key risksRARE key risks include [1] an over-reliance on its single commercial product, Show more. |
| Cash is significant % of market capNet D/ENet Debt/Equity. Debt net of cash. Negative indicates net cash. Equity is taken as the Market Capitalization is -11% |
| 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 -91%, 3Y Excs Rtn is -101% |
| Meaningful short interestShort Interest % of Basic SharesShort Interest % of Basic Shares = (Short Interest Quantity) / (Basic Shares Outstanding). A high fraction of short interest can indicate potential risk of a short squeeze. is 16% |
| Not profitable at operating income levelOp Inc LTMOperating Income, Last Twelve Months is -528 Mil, Op Mgn LTMOperating Margin = Operating Income / Revenue Reflects profitability before taxes and before impact of capital structure (interest payments). is -74% |
| Significant share based compensationSBC/Rev LTMShare Based Compensation / Revenue (Sales), Last Twelve Months (LTM) is 19% |
| Not cash flow generativeCFO/Rev LTMCash Flow from Operations / Revenue (Sales), Last Twelve Months (LTM) is -68%, FCF/Rev LTMFree Cash Flow / Revenue (Sales), Last Twelve Months (LTM) is -69% |
| 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 -26% |
| Key risksRARE key risks include [1] an over-reliance on its single commercial product, Show more. |
Qualitative Assessment
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Ultragenyx Pharmaceutical (RARE) stock has gained about 10% since 5/31/2026 because of the following key factors:
1. Strong Fiscal Q2 2026 Financial Performance Exceeding Expectations.
Ultragenyx Pharmaceutical reported robust financial results for its fiscal second quarter ended June 30, 2026, on August 4, 2026. The company posted a diluted earnings per share (EPS) of -$0.90, outperforming analysts' consensus estimates of -$1.22 by $0.32. Quarterly revenue reached a record $214 million, marking a 28.5% year-over-year increase and surpassing analyst projections of $183.08 million. Management also reaffirmed its full-year 2026 revenue guidance, projected between $730 million and $760 million, and reiterated its path to profitability in 2027. This strong financial showing, particularly the significant revenue beat, boosted investor confidence during the period.
2. U.S. FDA Accelerated Approval of GENGLYCOS (DTX401), the Company's First Gene Therapy.
On August 19, 2026, Ultragenyx achieved a significant regulatory milestone with the U.S. Food and Drug Administration's (FDA) accelerated approval of GENGLYCOS (pariglasgene brecaparvovec-opnr), also known as DTX401. This gene therapy is approved for adult and pediatric patients aged eight years and older with glycogen storage disease type Ia (GSDIa), marking Ultragenyx's first gene therapy approval and its fifth overall FDA approval. GENGLYCOS is the first FDA-approved treatment designed to address the underlying cause of GSDIa, a rare genetic disorder. The approval was supported by Phase 3 data demonstrating a statistically significant reduction in cornstarch requirements. This approval not only validates Ultragenyx's investment in gene therapy but also opens a new potential growth avenue with the commercial launch expected within 30 to 60 days.
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Ultragenyx Pharmaceutical (RARE) stock has gained about 10% since 5/31/2026 because of the following key factors:
1. Strong Fiscal Q2 2026 Financial Performance Exceeding Expectations.
Ultragenyx Pharmaceutical reported robust financial results for its fiscal second quarter ended June 30, 2026, on August 4, 2026. The company posted a diluted earnings per share (EPS) of -$0.90, outperforming analysts' consensus estimates of -$1.22 by $0.32. Quarterly revenue reached a record $214 million, marking a 28.5% year-over-year increase and surpassing analyst projections of $183.08 million. Management also reaffirmed its full-year 2026 revenue guidance, projected between $730 million and $760 million, and reiterated its path to profitability in 2027. This strong financial showing, particularly the significant revenue beat, boosted investor confidence during the period.
2. U.S. FDA Accelerated Approval of GENGLYCOS (DTX401), the Company's First Gene Therapy.
On August 19, 2026, Ultragenyx achieved a significant regulatory milestone with the U.S. Food and Drug Administration's (FDA) accelerated approval of GENGLYCOS (pariglasgene brecaparvovec-opnr), also known as DTX401. This gene therapy is approved for adult and pediatric patients aged eight years and older with glycogen storage disease type Ia (GSDIa), marking Ultragenyx's first gene therapy approval and its fifth overall FDA approval. GENGLYCOS is the first FDA-approved treatment designed to address the underlying cause of GSDIa, a rare genetic disorder. The approval was supported by Phase 3 data demonstrating a statistically significant reduction in cornstarch requirements. This approval not only validates Ultragenyx's investment in gene therapy but also opens a new potential growth avenue with the commercial launch expected within 30 to 60 days.
3. Continued Positive Analyst Sentiment and Upward Price Target Revisions.
During the period, analysts largely maintained positive ratings for Ultragenyx, with a consensus of "Moderate Buy" or "Strong Buy." Following the Q2 2026 earnings release and the GENGLYCOS approval, several analyst firms increased their 12-month price targets for RARE. For instance, JP Morgan raised its target from $75 to $80 on August 25, 2026, and Cantor Fitzgerald increased its target from $96 to $103 on August 20, 2026. The median 12-month price target from 20 Wall Street analysts stood at $49 as of August 28, 2026, indicating a significant potential upside from the stock's closing price.
4. Anticipation of Key Pipeline Catalysts in the Second Half of Fiscal 2026.
Investor optimism was further fueled by upcoming regulatory decisions and pivotal clinical trial data expected in the latter half of fiscal 2026. Ultragenyx has a PDUFA (Prescription Drug User Fee Act) action date of September 19, 2026, for UX111, a gene therapy for Sanfilippo syndrome type A. Additionally, Phase 3 data from the Aspire study for GTX-102 in Angelman syndrome are anticipated in the September or October timeframe. These near-term catalysts have positioned the company for potential further growth and expanded its multi-product pipeline outlook.
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Stock Movement Drivers
Fundamental Drivers
The 10.8% change in RARE stock from 5/31/2026 to 9/2/2026 was primarily driven by a 7.0% change in the company's Total Revenues ($ Mil).| (LTM values as of) | 5312026 | 9022026 | Change |
|---|---|---|---|
| Stock Price ($) | 23.94 | 26.53 | 10.8% |
| Change Contribution By: | |||
| Total Revenues ($ Mil) | 670 | 717 | 7.0% |
| P/S Multiple | 3.6 | 3.8 | 4.9% |
| Shares Outstanding (Mil) | 101 | 102 | -1.3% |
| Cumulative Contribution | 10.8% |
Market Drivers
5/31/2026 to 9/2/2026| Return | Correlation | |
|---|---|---|
| RARE | 10.8% | |
| Market (SPY) | 1.1% | 27.5% |
| Sector (XLV) | 15.7% | 18.3% |
Fundamental Drivers
The 13.4% change in RARE stock from 2/28/2026 to 9/2/2026 was primarily driven by a 8.7% change in the company's P/S Multiple.| (LTM values as of) | 2282026 | 9022026 | Change |
|---|---|---|---|
| Stock Price ($) | 23.39 | 26.53 | 13.4% |
| Change Contribution By: | |||
| Total Revenues ($ Mil) | 673 | 717 | 6.5% |
| P/S Multiple | 3.5 | 3.8 | 8.7% |
| Shares Outstanding (Mil) | 100 | 102 | -2.0% |
| Cumulative Contribution | 13.4% |
Market Drivers
2/28/2026 to 9/2/2026| Return | Correlation | |
|---|---|---|
| RARE | 13.4% | |
| Market (SPY) | 11.8% | 41.3% |
| Sector (XLV) | 8.4% | 28.1% |
Fundamental Drivers
The -11.4% change in RARE stock from 8/31/2025 to 9/2/2026 was primarily driven by a -22.1% change in the company's P/S Multiple.| (LTM values as of) | 8312025 | 9022026 | Change |
|---|---|---|---|
| Stock Price ($) | 29.96 | 26.53 | -11.4% |
| Change Contribution By: | |||
| Total Revenues ($ Mil) | 610 | 717 | 17.5% |
| P/S Multiple | 4.8 | 3.8 | -22.1% |
| Shares Outstanding (Mil) | 98 | 102 | -3.3% |
| Cumulative Contribution | -11.4% |
Market Drivers
8/31/2025 to 9/2/2026| Return | Correlation | |
|---|---|---|
| RARE | -11.4% | |
| Market (SPY) | 19.6% | 27.3% |
| Sector (XLV) | 27.5% | 20.7% |
Fundamental Drivers
The -27.9% change in RARE stock from 8/31/2023 to 9/2/2026 was primarily driven by a -41.8% change in the company's P/S Multiple.| (LTM values as of) | 8312023 | 9022026 | Change |
|---|---|---|---|
| Stock Price ($) | 36.79 | 26.53 | -27.9% |
| Change Contribution By: | |||
| Total Revenues ($ Mil) | 403 | 717 | 78.0% |
| P/S Multiple | 6.5 | 3.8 | -41.8% |
| Shares Outstanding (Mil) | 71 | 102 | -30.4% |
| Cumulative Contribution | -27.9% |
Market Drivers
8/31/2023 to 9/2/2026| Return | Correlation | |
|---|---|---|
| RARE | -27.9% | |
| Market (SPY) | 75.9% | 29.1% |
| Sector (XLV) | 35.9% | 28.9% |
Price Returns Compared
| 2021 | 2022 | 2023 | 2024 | 2025 | 2026 | Total [1] | |
|---|---|---|---|---|---|---|---|
| Returns | |||||||
| RARE Return | -39% | -45% | 3% | -12% | -45% | 12% | -81% |
| Peers Return | -30% | -7% | 5% | -35% | 190% | 6% | 36% |
| S&P 500 Return | 27% | -19% | 24% | 23% | 16% | 11% | 103% |
Monthly Win Rates [3] | |||||||
| RARE Win Rate | 33% | 25% | 33% | 33% | 58% | 56% | |
| Peers Win Rate | 42% | 50% | 42% | 33% | 67% | 66% | |
| S&P 500 Win Rate | 75% | 42% | 67% | 75% | 67% | 44% | |
Max Drawdowns [4] | |||||||
| RARE Max Drawdown | -56% | -61% | -39% | -30% | -57% | -31% | |
| Peers Max Drawdown | -48% | -43% | -34% | -46% | -40% | -32% | |
| S&P 500 Max Drawdown | -5% | -25% | -10% | -8% | -19% | -9% | |
[1] Cumulative total returns since the beginning of 2021
[2] Peers: ARWR, IONS, APMD, ATTO, BLSM. See RARE 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 9/2/2026 (YTD)
How Low Can It Go
| Event | RARE | S&P 500 |
|---|---|---|
| Summer-Fall 2023 Five Percent Yield Shock | ||
| % Loss | -23.7% | -9.5% |
| % Gain to Breakeven | 31.0% | 10.5% |
| Time to Breakeven | 48 days | 24 days |
| 2023 SVB Regional Banking Crisis | ||
| % Loss | -15.6% | -6.7% |
| % Gain to Breakeven | 18.4% | 7.1% |
| Time to Breakeven | 39 days | 31 days |
| 2020 COVID-19 Crash | ||
| % Loss | -45.1% | -33.7% |
| % Gain to Breakeven | 82.1% | 50.9% |
| Time to Breakeven | 35 days | 140 days |
| Q4 2018 Fed Policy Error / Growth Scare | ||
| % Loss | -49.5% | -19.2% |
| % Gain to Breakeven | 98.2% | 23.8% |
| Time to Breakeven | 546 days | 105 days |
| 2016-2017 Trump Reflation Bond Selloff | ||
| % Loss | -26.4% | -3.7% |
| % Gain to Breakeven | 35.8% | 3.9% |
| Time to Breakeven | 6 days | 6 days |
| 2015-2016 China Devaluation / Global Growth Scare | ||
| % Loss | -55.8% | -12.2% |
| % Gain to Breakeven | 126.1% | 13.9% |
| Time to Breakeven | 1737 days | 62 days |
In The Past
Ultragenyx Pharmaceutical's stock fell 0.0% during the 2024 Yen Carry Trade Unwind. Such a loss loss requires a 0.0% gain to breakeven.
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| Event | RARE | S&P 500 |
|---|---|---|
| Summer-Fall 2023 Five Percent Yield Shock | ||
| % Loss | -23.7% | -9.5% |
| % Gain to Breakeven | 31.0% | 10.5% |
| Time to Breakeven | 48 days | 24 days |
| 2020 COVID-19 Crash | ||
| % Loss | -45.1% | -33.7% |
| % Gain to Breakeven | 82.1% | 50.9% |
| Time to Breakeven | 35 days | 140 days |
| Q4 2018 Fed Policy Error / Growth Scare | ||
| % Loss | -49.5% | -19.2% |
| % Gain to Breakeven | 98.2% | 23.8% |
| Time to Breakeven | 546 days | 105 days |
| 2016-2017 Trump Reflation Bond Selloff | ||
| % Loss | -26.4% | -3.7% |
| % Gain to Breakeven | 35.8% | 3.9% |
| Time to Breakeven | 6 days | 6 days |
| 2015-2016 China Devaluation / Global Growth Scare | ||
| % Loss | -55.8% | -12.2% |
| % Gain to Breakeven | 126.1% | 13.9% |
| Time to Breakeven | 1737 days | 62 days |
| 2014-2016 Oil Price Collapse | ||
| % Loss | -26.7% | -6.8% |
| % Gain to Breakeven | 36.5% | 7.3% |
| Time to Breakeven | 29 days | 15 days |
In The Past
Ultragenyx Pharmaceutical's stock fell 0.0% during the 2024 Yen Carry Trade Unwind. Such a loss loss requires a 0.0% 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 Ultragenyx Pharmaceutical (RARE)
Ultragenyx Pharmaceutical Inc. (RARE) is a biopharmaceutical company focused on the identification, development, and commercialization of novel therapies for rare and ultra-rare genetic diseases. The company's mission is to bring innovative treatments to patients who suffer from severe conditions with significant unmet medical needs. It engages in the full lifecycle of drug development, from initial research and acquisition of promising compounds to clinical trials and global market distribution.
Ultragenyx currently has several approved biologic products that generate revenue. These include Crysvita (burosumab), an antibody used to treat X-linked hypophosphatemia and tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for Mucopolysaccharidosis VII; Dojolvi, which addresses long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab), approved for homozygous familial hypercholesterolemia. These commercialized therapies represent a range of approaches to tackle various rare genetic conditions.
Beyond its marketed products, Ultragenyx maintains a diverse pipeline of investigational therapies, utilizing advanced modalities like gene therapy, monoclonal antibodies, and antisense oligonucleotides. These candidates aim to treat conditions such as glycogen storage diseases, ornithine transcarbamylase deficiency, osteogenesis imperfecta, Angelman syndrome, and Wilson disease. The company primarily serves patients, both children and adults, diagnosed with these rare and ultra-rare genetic disorders across North America, Europe, and other international markets.
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Here are 1-3 brief analogies for Ultragenyx Pharmaceutical (RARE):
- Like Vertex Pharmaceuticals, but focused on a broader spectrum of rare and ultra-rare genetic diseases.
- A highly specialized version of BioMarin Pharmaceutical, with an even stronger emphasis on ultra-rare genetic conditions and advanced gene therapies.
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- Crysvita (burosumab): An antibody for the treatment of X-linked hypophosphatemia and tumor-induced osteomalacia.
- Mepsevii: An enzyme replacement therapy for the treatment of Mucopolysaccharidosis VII.
- Dojolvi: A medication for treating long-chain fatty acid oxidation disorders.
- Evkeeza (evinacumab): A treatment for homozygous familial hypercholesterolemia.
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Ultragenyx Pharmaceutical Inc. (RARE) is a biopharmaceutical company that develops and commercializes highly specialized prescription drugs for rare and ultra-rare genetic diseases. As such, it sells primarily to other companies within the healthcare supply chain rather than directly to individuals.
The major customers for Ultragenyx are the large pharmaceutical wholesalers and distributors who purchase drugs directly from manufacturers and then distribute them to hospitals, clinics, and pharmacies. These entities serve as the crucial intermediary link in getting Ultragenyx's products to patients.
The primary customer companies that Ultragenyx sells to include the following major pharmaceutical wholesalers:
- McKesson Corporation (NYSE: MCK)
- AmerisourceBergen Corporation (NYSE: ABC)
- Cardinal Health, Inc. (NYSE: CAH)
These distributors then supply a wide network of healthcare providers, including hospitals, clinics, and specialty pharmacies, which are the points of care where patients receive Ultragenyx's treatments.
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- Kyowa Kirin Co., Ltd. (KYKOF)
- REGENXBIO Inc. (RGNX)
- Mereo BioPharma PLC (MREO)
- Arcturus Therapeutics Holdings Inc. (ARCT)
- Solid Biosciences Inc. (SLDB)
- Daiichi Sankyo Co., Ltd. (DSNKY)
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Emil Kakkis, M.D., Ph.D. Chief Executive Officer, President, and Director
Dr. Kakkis founded Ultragenyx in 2010. He is recognized as a pioneer in developing treatments for rare diseases. Before founding Ultragenyx, he served as Chief Medical Officer at BioMarin Pharmaceutical, where he was instrumental in the development and approval of treatments for rare disorders such as MPS I, MPS VI, and PKU. He also initiated the enzyme therapy program for MPS I at Harbor-UCLA Medical Center. Dr. Kakkis is also the founder of the EveryLife Foundation for Rare Diseases, a non-profit organization focused on accelerating biotech innovation for rare diseases.
Howard Horn Chief Financial Officer and Executive Vice President, Corporate Strategy
Mr. Horn joined Ultragenyx in October 2023. Prior to this, he was the Chief Financial Officer at Vir Biotechnology, Inc. from March 2017 to April 2023, where he was a member of the founding management team and played a key role in guiding the company to become a public, commercial-stage organization through rapid growth, financings, and foundational transactions. He previously held positions at Biogen as Vice President, Head of Business Planning, and Vice President, Strategic Corporate Finance. Mr. Horn also has experience as a consultant in the Pharmaceutical and Medical Products Practice at McKinsey & Company and as an equity research analyst in the Life Sciences group at UBS Group AG. He is also a Co-Founder of Noble.MD.
Dennis Huang Chief Technical Operations Officer and Executive Vice President, Gene Therapy Research and Development
Mr. Huang joined Ultragenyx in May 2015. He is responsible for leading the company's gene therapy research group, global CMC (Chemistry, Manufacturing, and Controls) development, manufacturing, and supply chain functions across various technical modalities. His career includes senior leadership roles at InterMune Inc. as Senior Vice President of Manufacturing and Supply Chain, Vice President of Biologics Manufacturing and Development at Allergan, Inc., and positions at Novartis AG, Genentech, Inc., and Synergen (now Amgen).
Erik Harris Chief Commercial Officer and Executive Vice President
Mr. Harris was promoted to Chief Commercial Officer and Executive Vice President in June 2019. He joined Ultragenyx in 2017 as Senior Vice President, Head of North American Commercial Operations, where he was instrumental in the launches of Crysvita® and Mepsevii®. Before Ultragenyx, Mr. Harris spent six years at Crescendo Bioscience, a subsidiary of Myriad Genetics, Inc., holding various leadership positions including Vice President of Commercial. He also held roles in commercial organizations at Intermune, Elan Pharmaceuticals, Genentech, Inc., and Bristol-Myers Squibb.
Eric Crombez, M.D. Chief Medical Officer and Executive Vice President
Dr. Crombez assumed the role of Executive Vice President, Chief Medical Officer, effective May 1, 2023. Prior to this appointment, he served as Ultragenyx's Chief Medical Officer for Gene Therapy and Inborn Errors of Metabolism.
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The key risks to Ultragenyx Pharmaceutical's business, trading under the symbol RARE, are primarily concentrated around the inherent challenges of drug development and commercialization in the biopharmaceutical industry, compounded by financial and legal pressures.
- Clinical Trial Success and Regulatory Approval: A predominant risk for Ultragenyx Pharmaceutical is the success of its numerous product candidates in clinical trials and the subsequent attainment of regulatory approvals. The company has a diverse pipeline, including several gene therapies and monoclonal antibodies in various stages of development. The failure of a drug candidate in late-stage clinical trials, such as the recent setbacks with setrusumab in Phase 3 trials for osteogenesis imperfecta, can significantly impact the company's valuation and future prospects. The inherent uncertainty of drug development, the challenge of demonstrating statistically significant results in smaller patient populations typical of rare diseases, and the rigorous regulatory approval process pose ongoing hurdles to the company's growth trajectory and market position.
- Financial Health and Path to Profitability: Ultragenyx Pharmaceutical faces significant financial challenges, operating as a development-stage company with no imminent path to sustained profitability. The company has reported substantial operational losses, evidenced by a net margin of -85.44% and an operating margin of -79.5%, along with a negative equity position, raising concerns about long-term financial stability. Furthermore, it has demonstrated a high rate of cash burn, and underperformance in R&D expenses (e.g., $216 million compared to estimates of $166 million in Q3 2025) has raised concerns about financial management and resource allocation, amplifying long-term profitability risks. While the company anticipates reaching GAAP profitability by 2027, this outlook is subject to operational adjustments and successful commercialization efforts.
- Legal and Reputational Risks: Ultragenyx is currently exposed to significant legal and financial risks due to a class-action lawsuit. The lawsuit alleges that the company made misleading statements regarding its Phase 3 clinical trials for setrusumab and failed to adequately disclose material risks concerning the study designs and the limitations of relying on bone mineral density as a predictor of fracture reduction. This legal challenge introduces considerable uncertainty, with potential for substantial legal fees and settlement expenses, which could negatively impact its financial statements and damage the company's reputation among investors and the broader market.
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Ultragenyx Pharmaceutical Inc. operates in several rare and ultra-rare genetic disease markets, with key products addressing specific patient populations. The addressable markets for their main products are as follows:
- Crysvita (burosumab) for X-linked hypophosphatemia (XLH): The global market for X-linked hypophosphatemia was valued at approximately USD 2.64 billion in 2024 and is projected to reach about USD 5.42 billion by 2031, growing at a compound annual growth rate (CAGR) of 10.8% from 2025 to 2031. North America represented the largest share of this market in 2025. An estimated 1 in 20,000 individuals globally are affected by X-linked hypophosphatemia.
- Mepsevii (vestronidase alfa) for Mucopolysaccharidosis VII (MPS VII): The global treatment market for Mucopolysaccharidosis type VII (MPS VII) was valued at approximately USD 391.8 million in 2024 and is expected to reach about USD 562.5 million by 2031, with a CAGR of 5.3% from 2025 to 2031. North America is anticipated to hold a dominant position in the MPS VII treatment market.
- Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders (LC-FAOD): The global market for molecularly long-chain fatty acid oxidation disorders was valued at USD 11.54 billion in 2025 and is projected to grow to USD 18.53 billion by 2033, with a CAGR of 6.10% during this forecast period. In the United States, an estimated 2,000 to 3,500 children and adults are affected by LC-FAOD. The U.S. market held the largest revenue share within North America for this indication in 2025.
- Evkeeza (evinacumab) for homozygous familial hypercholesterolemia (HoFH): The total market size for homozygous familial hypercholesterolemia across the 7 Major Markets (7MM) was approximately USD 108.23 million in 2022 and is expected to increase through 2034. The U.S. market accounted for approximately USD 83.37 million of this in 2022, representing the largest share among the 7MM. The global Homozygous Familial Hypercholesterolemia market is expected to grow from USD 94.78 million in 2025 to USD 111.01 million by 2031, at a CAGR of 2.67%.
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Ultragenyx Pharmaceutical (RARE) is poised for future revenue growth over the next 2-3 years, driven by the continued expansion of its existing commercial products and the anticipated launch and advancement of several key pipeline therapies.
Here are 3-5 expected drivers of future revenue growth:
- Continued Growth of Commercial Products: Ultragenyx expects sustained double-digit growth from its established commercial products, including Crysvita (burosumab), Dojolvi (triheptanoin), and Evkeeza (evinacumab). Crysvita is anticipated to grow through increased demand in Latin America and Turkey, as well as continued penetration of pediatric and adult X-linked hypophosphatemia (XLH) markets in the U.S. Evkeeza is also experiencing substantial demand growth following its launches in territories outside the United States. Mepsevii continues to contribute a stable revenue base.
- Launch of DTX401 for Glycogen Storage Disease Type Ia (GSDIa): A significant revenue driver is the potential approval and launch of DTX401, an adeno-associated virus 8 (AAV8) gene therapy for GSDIa. The company has a PDUFA (Prescription Drug User Fee Act) date set for August 2026, and management is optimistic about its durability and commercial potential, marking it as a crucial regulatory milestone and a potential first gene therapy launch for Ultragenyx.
- Potential Launch of UX111 for Sanfilippo Syndrome Type A: Despite a previous Complete Response Letter from the FDA, Ultragenyx is actively working to address regulatory feedback for UX111, a gene therapy for Sanfilippo syndrome type A. The company expects potential approval for this therapy in the second half of 2026, which would represent another significant new product introduction.
- Advancement of GTX-102 for Angelman Syndrome: The advancement of GTX-102, an antisense oligonucleotide for Angelman syndrome, is a key pipeline driver. A pivotal Phase 3 data readout is expected in the second half of 2026. Positive results from this study would pave the way for future commercialization, significantly accelerating Ultragenyx's revenue trajectory.
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Capital Allocation Decisions (Last 3-5 Years)
Share Repurchases
- Ultragenyx Pharmaceutical has not made significant share repurchases over the last 3-5 years. The buyback yield was reported as -8.95% in a recent analysis, indicating a net issuance rather than repurchases.
Share Issuance
- Ultragenyx Pharmaceutical's shares outstanding increased by 0.2% in Q4 2025 compared to the prior quarter, reaching 97 million shares.
- Executive officers and other insiders engaged in stock-based compensation activities, with some shares subsequently sold to cover tax withholdings, such as in March 2026, which effectively results in issuance from the company's perspective.
- The company's book value per share decreased significantly between December 2021 ($277.75) and December 2025 (-$14.96), which can be influenced by share issuances among other factors.
Inbound Investments
- In 2024, partnerships, including those with Kyowa Kirin and Daiichi Sankyo in Japan, contributed over $120 million in collaboration revenue to Ultragenyx.
- Ultragenyx Pharmaceutical entered into a Royalty Purchase Agreement with an OMERS investment vehicle on July 14, 2022, involving a portion of future Crysvita royalty payments in the United States and Canada.
Outbound Investments
- In July 2022, Ultragenyx Pharmaceutical acquired GeneTx BioTherapeutics LLC for a total purchase consideration of $91.2 million, which included an option exercise price of $75.0 million plus outstanding cash and working capital adjustments.
- Ultragenyx is actively pursuing pre-commercial and commercial-stage acquisitions to enhance its neurology and metabolic disease franchises, supported by over $900 million in cash and investments as of Q1 2025.
Capital Expenditures
- Capital expenditures were reported as $6.0 million for the last 12 months ending February 2026.
- In Q4 2025, Ultragenyx Pharm invested $938,000 in capital expenditures.
- Historical capital expenditures were approximately $4.08 million in 2025 and $2.79 million in 2024.
Peer Outperformance in Biotechnology
| Industry | Names | 1Y med | 3Y med | 5Y med | Top 3 by 5Y |
|---|---|---|---|---|---|
| Health Care Distributors | 7 | 14.9% | 97.1% | 148.9% | CAH 410% · MCK 359% · COR 183% |
| Managed Health Care | 8 | 32.0% | 3.1% | 3.9% | OSCR 67% · HQY 52% · ELV 15% |
| Health Care Services | 20 | 19.8% | 49.6% | -0.6% | HIMS 249% · RDNT 140% · DGX 70% |
| Health Care Facilities | 24 | 2.8% | -5.4% | -20.1% | NHC 242% · THC 241% · ENSG 111% |
| Health Care Equipment | 50 | -0.5% | -6.4% | -23.3% | POCI 11025% · UFPT 323% · GKOS 204% |
| Health Care Supplies | 12 | 33.0% | -15.3% | -34.7% | LNTH 271% · HAE 59% · MMSI 23% |
| Pharmaceuticals | 63 | 2.3% | 3.2% | -35.1% | LQDA 2443% · INDV 1152% · ETON 1089% |
| Life Sciences Tools & Services | 109 | 0.8% | -32.0% | -73.6% | SNWV 1716% · MEDP 204% · AXGN 178% |
| Biotechnology ← | 363 | 8.7% | -27.2% | -76.5% | BRTX 100750% · DNTH 1529% · CELZ 1514% |
| Health Care Technology | 20 | -22.3% | -58.7% | -78.1% | SPOK 66% · HSTM 0% · VEEV -12% |
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Peer Comparisons
| Peers to compare with: |
Financials
| Median | |
|---|---|
| Name | |
| Mkt Price | 27.18 |
| Mkt Cap | 10.1 |
| Rev LTM | 693 |
| Op Inc LTM | -377 |
| FCF LTM | -285 |
| FCF 3Y Avg | -410 |
| CFO LTM | -273 |
| CFO 3Y Avg | -358 |
Growth & Margins
| Median | |
|---|---|
| Name | |
| Rev Chg LTM | 16.8% |
| Rev Chg 3Y Avg | 21.3% |
| Rev Chg Q | 28.1% |
| QoQ Delta Rev Chg LTM | 7.0% |
| Op Inc Chg LTM | -109.7% |
| Op Inc Chg 3Y Avg | -24.5% |
| Op Mgn LTM | -70.8% |
| Op Mgn 3Y Avg | -90.2% |
| QoQ Delta Op Mgn LTM | 1.9% |
| CFO/Rev LTM | -62.2% |
| CFO/Rev 3Y Avg | -78.1% |
| FCF/Rev LTM | -66.9% |
| FCF/Rev 3Y Avg | -81.3% |
Valuation
| Median | |
|---|---|
| Name | |
| Mkt Cap | 10.1 |
| P/S | 11.6 |
| P/Op Inc | -17.1 |
| P/EBIT | -21.2 |
| P/E | -18.0 |
| P/CFO | -20.4 |
| Total Yield | -5.6% |
| Dividend Yield | 0.0% |
| FCF Yield 3Y Avg | -6.0% |
| D/E | 0.1 |
| Net D/E | -0.1 |
Returns
| Median | |
|---|---|
| Name | |
| 1M Rtn | 8.7% |
| 3M Rtn | 14.5% |
| 6M Rtn | 16.2% |
| 12M Rtn | 9.0% |
| 3Y Rtn | 16.2% |
| 1M Excs Rtn | 7.8% |
| 3M Excs Rtn | 13.0% |
| 6M Excs Rtn | 4.6% |
| 12M Excs Rtn | -10.5% |
| 3Y Excs Rtn | -56.7% |
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 |
| NDA213687 | DOJOLVI | triheptanoin | liquid | 6302020 | 8.8% | 88.5% | 21.9% | -23.7% | -66.1% |
| BLA761047 | MEPSEVII | vestronidase alfa-vjbk | injectable | 11152017 | 0.7% | 35.1% | 8.4% | -23.8% | -43.9% |
| Post-Approval Fwd Returns | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| FDA BLA # | Brand Name | Generic Name | License Type | First Approval | Latest Approval | 3M Rtn | 6M Rtn | 1Y Rtn | 2Y Rtn | Total Rtn |
| 125858 | GENGLYCOS | pariglasgene brecaparvovec-opnr | 351(a) | 8/19/2026 | 8/19/2026 | 1.1% | 1.1% | 1.1% | 1.1% | 1.1% |
| Post-Approval Fwd Returns | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| FDA App # | Brand Name | Generic Name | Dosage Form | FDA Approval | 3M Rtn | 6M Rtn | 1Y Rtn | 2Y Rtn | Total Rtn |
| NDA213687 | DOJOLVI | triheptanoin | liquid | 6302020 | 8.8% | 88.5% | 21.9% | -23.7% | -66.1% |
| BLA761047 | MEPSEVII | vestronidase alfa-vjbk | injectable | 11152017 | 0.7% | 35.1% | 8.4% | -23.8% | -43.9% |
| Post-Approval Fwd Returns | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| FDA BLA # | Brand Name | Generic Name | License Type | First Approval | Latest Approval | 3M Rtn | 6M Rtn | 1Y Rtn | 2Y Rtn | Total Rtn |
| 125858 | GENGLYCOS | pariglasgene brecaparvovec-opnr | 351(a) | 8/19/2026 | 8/19/2026 | 1.1% | 1.1% | 1.1% | 1.1% | 1.1% |
Clinical Pipeline
| Drug Candidate | Indication | Phase 3 Trials | Est. Primary Completion |
|---|---|---|---|
| Setrusumab (UX143) | Osteogenesis Imperfecta | 3 | Jan 2027 |
| Triheptanoin (UX007) | Long-chain Fatty Acid Oxidation Disorders | 1 | Aug 2027 |
| UX111 (ABO-102) | Mucopolysaccharidosis Type IIIA | 2 | Aug 2027 |
| DTX301 | Ornithine Transcarbamylase Deficiency | 1 | Sep 2027 |
| GTX-102 | Angelman Syndrome | 2 | Feb 2029 |
Segment Financials
Revenue by Segment| $ Mil | 2025 | 2024 | 2023 | 2022 | 2021 |
|---|---|---|---|---|---|
| Research, development and commercialization of its products | 673 | 560 | 434 | 363 | |
| Crysvita | 21 | ||||
| Crysvita collaboration revenue in profit-share territory | 171 | ||||
| Crysvita royalty revenue | 18 | ||||
| Crysvita royalty revenue in European territory | 0 | ||||
| Daiichi Sankyo | 85 | ||||
| Dojolvi | 40 | ||||
| Evkeeza | 0 | ||||
| Mepsevii | 16 | ||||
| Total | 673 | 560 | 434 | 363 | 351 |
Price Behavior
| Market Price | $26.53 | |
| Market Cap ($ Bil) | 2.7 | |
| First Trading Date | 01/31/2014 | |
| Distance from 52W High | -27.4% | |
| 50 Days | 200 Days | |
| DMA Price | $28.36 | $26.23 |
| DMA Trend | down | up |
| Distance from DMA | -6.5% | 1.1% |
| 3M | 1YR | |
| Volatility | 52.0% | 67.2% |
| Downside Capture | 36.50 | 187.92 |
| Upside Capture | 113.68 | 125.95 |
| Correlation (SPY) | 27.9% | 26.3% |
| 1M | 2M | 3M | 6M | 1Y | 3Y | |
|---|---|---|---|---|---|---|
| Beta | 1.55 | 1.44 | 1.06 | 1.53 | 1.41 | 1.06 |
| Up Beta | 0.50 | 1.76 | 2.39 | 2.07 | 2.03 | 1.11 |
| Down Beta | 6.56 | 2.95 | 1.44 | 1.30 | 0.78 | 0.75 |
| Up Capture | 138% | -12% | 53% | 141% | 138% | 101% |
| Bmk +ve Days | 10 | 21 | 32 | 68 | 138 | 427 |
| Stock +ve Days | 10 | 15 | 29 | 60 | 119 | 369 |
| Down Capture | 198% | 218% | 36% | 138% | 144% | 108% |
| Bmk -ve Days | 11 | 21 | 32 | 59 | 113 | 324 |
| Stock -ve Days | 11 | 27 | 35 | 66 | 130 | 376 |
[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 RARE | |
|---|---|---|---|---|
| RARE | -17.2% | 67.2% | 0.04 | - |
| Sector ETF (XLV) | 27.5% | 16.0% | 1.33 | 20.7% |
| Equity (SPY) | 20.6% | 12.8% | 1.19 | 27.3% |
| Gold (GLD) | 23.8% | 29.1% | 0.73 | 23.8% |
| Commodities (DBC) | 41.7% | 20.4% | 1.60 | -6.5% |
| Real Estate (VNQ) | 9.7% | 13.6% | 0.43 | 10.4% |
| Bitcoin (BTCUSD) | -30.7% | 43.5% | -0.72 | 13.2% |
Smart multi-asset allocation framework can stack odds in your favor. Learn How
Based On 5-Year Data
| Annualized Return | Annualized Volatility | Sharpe Ratio | Correlation with RARE | |
|---|---|---|---|---|
| RARE | -21.8% | 54.4% | -0.23 | - |
| Sector ETF (XLV) | 6.7% | 15.1% | 0.25 | 32.1% |
| Equity (SPY) | 12.7% | 17.2% | 0.57 | 34.0% |
| Gold (GLD) | 19.2% | 18.8% | 0.83 | 14.3% |
| Commodities (DBC) | 10.8% | 19.5% | 0.43 | 2.9% |
| Real Estate (VNQ) | 1.9% | 18.9% | -0.01 | 30.8% |
| Bitcoin (BTCUSD) | 9.5% | 52.6% | 0.37 | 16.2% |
Smart multi-asset allocation framework can stack odds in your favor. Learn How
Based On 10-Year Data
| Annualized Return | Annualized Volatility | Sharpe Ratio | Correlation with RARE | |
|---|---|---|---|---|
| RARE | -9.2% | 55.1% | 0.06 | - |
| Sector ETF (XLV) | 10.6% | 16.7% | 0.52 | 41.1% |
| Equity (SPY) | 15.1% | 17.9% | 0.72 | 40.8% |
| Gold (GLD) | 12.2% | 16.3% | 0.61 | 9.8% |
| Commodities (DBC) | 7.8% | 18.1% | 0.35 | 10.4% |
| Real Estate (VNQ) | 4.8% | 20.7% | 0.19 | 30.0% |
| Bitcoin (BTCUSD) | 63.0% | 66.1% | 1.03 | 11.4% |
Smart multi-asset allocation framework can stack odds in your favor. Learn How
Returns Analyses
Earnings Returns History
Updated 9/3/2026| Forward Returns | |||
|---|---|---|---|
| Earnings Date | 1D Returns | 5D Returns | 21D Returns |
| 8/4/2026 | -3.4% | 4.5% | 2.8% |
| 5/5/2026 | 3.7% | 7.3% | -6.8% |
| 2/12/2026 | -12.7% | -3.3% | -7.1% |
| 11/4/2025 | -1.8% | 2.4% | 12.1% |
| 8/5/2025 | -3.0% | -2.8% | 8.2% |
| 5/6/2025 | -4.7% | 1.0% | 5.3% |
| 2/13/2025 | -2.4% | -2.1% | -8.9% |
| 11/5/2024 | -3.2% | -8.3% | -9.3% |
| ... | |||
| SUMMARY STATS | |||
| # Positive | 10 | 11 | 11 |
| # Negative | 14 | 13 | 13 |
| Median Positive | 3.7% | 6.5% | 11.9% |
| Median Negative | -4.0% | -5.8% | -7.3% |
| Max Positive | 14.3% | 13.9% | 28.6% |
| Max Negative | -13.4% | -24.1% | -25.8% |
| Forward Returns | |||
|---|---|---|---|
| Earnings Date | 1D Returns | 5D Returns | 21D Returns |
| 8/4/2026 | -3.4% | 4.5% | 2.8% |
| 5/5/2026 | 3.7% | 7.3% | -6.8% |
| 2/12/2026 | -12.7% | -3.3% | -7.1% |
| 11/4/2025 | -1.8% | 2.4% | 12.1% |
| 8/5/2025 | -3.0% | -2.8% | 8.2% |
| 5/6/2025 | -4.7% | 1.0% | 5.3% |
| 2/13/2025 | -2.4% | -2.1% | -8.9% |
| 11/5/2024 | -3.2% | -8.3% | -9.3% |
| 8/1/2024 | 14.3% | 13.9% | 28.6% |
| 5/2/2024 | -1.0% | -4.6% | -4.8% |
| 2/15/2024 | 5.8% | 7.0% | 0.7% |
| 11/2/2023 | 6.1% | -4.5% | 12.6% |
| 8/3/2023 | -8.8% | -11.6% | -8.5% |
| 5/4/2023 | 3.1% | 2.8% | 11.9% |
| 1/6/2023 | -13.0% | -6.7% | 2.1% |
| 11/2/2022 | -6.0% | -16.9% | -1.0% |
| 7/28/2022 | 2.7% | 7.5% | -4.6% |
| 5/5/2022 | -13.4% | -24.1% | -25.8% |
| 2/10/2022 | 2.1% | -5.8% | -12.9% |
| 11/2/2021 | 1.4% | -4.6% | -6.7% |
| 8/2/2021 | 3.6% | 11.3% | 19.1% |
| 5/4/2021 | 4.1% | 6.5% | -7.3% |
| 2/11/2021 | -7.7% | -9.0% | -20.6% |
| 10/27/2020 | -2.5% | 4.7% | 23.0% |
| SUMMARY STATS | |||
| # Positive | 10 | 11 | 11 |
| # Negative | 14 | 13 | 13 |
| Median Positive | 3.7% | 6.5% | 11.9% |
| Median Negative | -4.0% | -5.8% | -7.3% |
| Max Positive | 14.3% | 13.9% | 28.6% |
| Max Negative | -13.4% | -24.1% | -25.8% |
SEC Filings
Expand for More| Report Date | Filing Date | Filing |
|---|---|---|
| 06/30/2026 | 08/05/2026 | 10-Q |
| 03/31/2026 | 05/06/2026 | 10-Q |
| 12/31/2025 | 02/18/2026 | 10-K |
| 09/30/2025 | 11/05/2025 | 10-Q |
| 06/30/2025 | 08/06/2025 | 10-Q |
| 03/31/2025 | 05/07/2025 | 10-Q |
| 12/31/2024 | 02/19/2025 | 10-K |
| 09/30/2024 | 11/06/2024 | 10-Q |
| 06/30/2024 | 08/02/2024 | 10-Q |
| 03/31/2024 | 05/03/2024 | 10-Q |
| 12/31/2023 | 02/21/2024 | 10-K |
| 09/30/2023 | 11/03/2023 | 10-Q |
| 06/30/2023 | 08/04/2023 | 10-Q |
| 03/31/2023 | 05/05/2023 | 10-Q |
| 12/31/2022 | 02/17/2023 | 10-K |
| 09/30/2022 | 11/03/2022 | 10-Q |
| Report Date | Filing Date | Filing |
|---|---|---|
| 06/30/2026 | 08/05/2026 | 10-Q |
| 03/31/2026 | 05/06/2026 | 10-Q |
| 12/31/2025 | 02/18/2026 | 10-K |
| 09/30/2025 | 11/05/2025 | 10-Q |
| 06/30/2025 | 08/06/2025 | 10-Q |
| 03/31/2025 | 05/07/2025 | 10-Q |
| 12/31/2024 | 02/19/2025 | 10-K |
| 09/30/2024 | 11/06/2024 | 10-Q |
| 06/30/2024 | 08/02/2024 | 10-Q |
| 03/31/2024 | 05/03/2024 | 10-Q |
| 12/31/2023 | 02/21/2024 | 10-K |
| 09/30/2023 | 11/03/2023 | 10-Q |
| 06/30/2023 | 08/04/2023 | 10-Q |
| 03/31/2023 | 05/05/2023 | 10-Q |
| 12/31/2022 | 02/17/2023 | 10-K |
| 09/30/2022 | 11/03/2022 | 10-Q |
| 06/30/2022 | 07/29/2022 | 10-Q |
| 03/31/2022 | 05/06/2022 | 10-Q |
| 12/31/2021 | 02/15/2022 | 10-K |
| 09/30/2021 | 11/03/2021 | 10-Q |
| 06/30/2021 | 08/03/2021 | 10-Q |
| 03/31/2021 | 05/05/2021 | 10-Q |
| 12/31/2020 | 02/12/2021 | 10-K |
| 09/30/2020 | 10/27/2020 | 10-Q |
| 06/30/2020 | 07/31/2020 | 10-Q |
| 03/31/2020 | 05/07/2020 | 10-Q |
| 12/31/2019 | 02/14/2020 | 10-K |
| 09/30/2019 | 11/06/2019 | 10-Q |
Recent Forward Guidance
Updated 8/5/2026Latest: Q2 2026 Earnings Reported 8/4/2026
| Forward Guidance | Guidance Change | |||||||
|---|---|---|---|---|---|---|---|---|
| Metric | Source | Low | Mid | High | % Chg | % Delta | Change | Prior |
| 2026 Total Revenue | Reported | 730.00 Mil | 745.00 Mil | 760.00 Mil | 0 | Affirmed | Guidance: 745.00 Mil for 2026 | |
| 2026 Crysvita Revenue | Reported | 500.00 Mil | 510.00 Mil | 520.00 Mil | 0 | Affirmed | Guidance: 510.00 Mil for 2026 | |
| 2026 Dojolvi Revenue | Reported | 100.00 Mil | 105.00 Mil | 110.00 Mil | 0 | Affirmed | Guidance: 105.00 Mil for 2026 | |
Insider Activity
Updated 8/4/2026| # | Owner | Title | Holding | Action | Filing Date | Price | Shares | Transacted Value | Value of Held Shares | Form |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | Parschauer, Karah Herdman | EVP and Chief Legal Officer | Direct | Sell | 8042026 | 24.92 | 1,900 | 47,348 | 2,306,645 | Form |
| 2 | Horn, Howard | Chief Financial Officer | Direct | Sell | 8042026 | 24.92 | 4,696 | 117,024 | 2,399,671 | Form |
| 3 | Horn, Howard | Chief Financial Officer | Direct | Sell | 7062026 | 33.13 | 4,698 | 155,645 | 3,345,832 | Form |
| 4 | Sanders, Corazon (corsee) D | Direct | Sell | 6152026 | 25.05 | 2,000 | 50,100 | 528,430 | Form | |
| 5 | Parschauer, Karah Herdman | EVP and Chief Legal Officer | Direct | Sell | 6152026 | 24.62 | 1,899 | 46,753 | 2,325,654 | Form |
| # | Owner | Title | Holding | Action | Filing Date | Price | Shares | Transacted Value | Value of Held Shares | Form |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | Parschauer, Karah Herdman | EVP and Chief Legal Officer | Direct | Sell | 8042026 | 24.92 | 1,900 | 47,348 | 2,306,645 | Form |
| 2 | Horn, Howard | Chief Financial Officer | Direct | Sell | 8042026 | 24.92 | 4,696 | 117,024 | 2,399,671 | Form |
| 3 | Horn, Howard | Chief Financial Officer | Direct | Sell | 7062026 | 33.13 | 4,698 | 155,645 | 3,345,832 | Form |
| 4 | Sanders, Corazon (corsee) D | Direct | Sell | 6152026 | 25.05 | 2,000 | 50,100 | 528,430 | Form | |
| 5 | Parschauer, Karah Herdman | EVP and Chief Legal Officer | Direct | Sell | 6152026 | 24.62 | 1,899 | 46,753 | 2,325,654 | Form |
| 6 | Horn, Howard | Chief Financial Officer | Direct | Sell | 6012026 | 23.77 | 4,653 | 110,602 | 2,512,228 | Form |
| 7 | Suliman, Shehnaaz | Direct | Sell | 5182026 | 25.12 | 5,740 | 144,189 | 702,129 | Form | |
| 8 | Crombez, Eric | EVP and Chief Medical Officer | Direct | Sell | 5062026 | 24.96 | 344 | 8,586 | 2,775,277 | Form |
| 9 | Horn, Howard | Chief Financial Officer | Direct | Sell | 5042026 | 24.90 | 4,683 | 116,607 | 2,747,516 | Form |
| 10 | Horn, Howard | Chief Financial Officer | Direct | Sell | 4022026 | 20.97 | 4,683 | 98,203 | 1,684,960 | Form |
| 11 | Parschauer, Karah Herdman | EVP and Chief Legal Officer | Direct | Sell | 3102026 | 22.67 | 3,508 | 79,526 | 1,514,809 | Form |
| 12 | Parschauer, Karah Herdman | EVP and Chief Legal Officer | Direct | Sell | 3102026 | 21.40 | 6,018 | 128,785 | 1,505,019 | Form |
| 13 | Horn, Howard | Chief Financial Officer | Direct | Sell | 3032026 | 22.40 | 1,635 | 36,624 | 1,904,762 | Form |
| 14 | Pinion, John Richard | See Remarks | Direct | Sell | 3032026 | 22.80 | 10,029 | 228,661 | 2,508,593 | Form |
| 15 | Harris, Erik | EVP & Chief Commercial Officer | Direct | Sell | 3032026 | 22.80 | 10,539 | 240,289 | 2,040,942 | Form |
| 16 | Kakkis, Emil D | President & CEO | Direct | Sell | 3032026 | 22.80 | 54,404 | 1,240,411 | 15,025,063 | Form |
| 17 | Parschauer, Karah Herdman | EVP and Chief Legal Officer | Direct | Sell | 3032026 | 22.80 | 8,135 | 185,478 | 1,740,689 | Form |
| 18 | Huizenga, Theodore Alan | SVP, Chief Accounting Officer | Direct | Sell | 3032026 | 22.80 | 1,632 | 37,210 | 1,109,676 | Form |
| 19 | Horn, Howard | Chief Financial Officer | Direct | Sell | 3032026 | 22.70 | 8,409 | 190,914 | 1,967,695 | Form |
| 20 | Crombez, Eric | EVP and Chief Medical Officer | Direct | Sell | 3032026 | 22.80 | 7,029 | 160,261 | 1,739,663 | Form |
| 21 | Horn, Howard | Chief Financial Officer | Direct | Sell | 2032026 | 23.56 | 3,061 | 72,117 | 2,095,309 | Form |
| 22 | Horn, Howard | Chief Financial Officer | Direct | Sell | 1052026 | 22.84 | 3,150 | 71,946 | 2,101,189 | Form |
| 23 | Huizenga, Theodore Alan | SVP, Chief Accounting Officer | Direct | Sell | 12302025 | 34.38 | 85 | 2,922 | 1,734,471 | Form |
| 24 | Horn, Howard | Chief Financial Officer | Direct | Sell | 12112025 | 36.38 | 3,081 | 112,087 | 3,461,411 | Form |
| 25 | Horn, Howard | Chief Financial Officer | Direct | Sell | 10142025 | 31.51 | 7,942 | 250,252 | 3,095,133 | Form |
| 26 | Huizenga, Theodore Alan | SVP, Chief Accounting Officer | Direct | Sell | 9192025 | 29.17 | 64 | 1,867 | 1,465,559 | Form |
| 27 | Parschauer, Karah Herdman | EVP and Chief Legal Officer | Direct | Sell | 9122025 | 31.17 | 2,450 | 76,366 | 2,283,857 | Form |
| 28 | Huizenga, Theodore Alan | SVP, Chief Accounting Officer | Direct | Sell | 9082025 | 31.39 | 84 | 2,637 | 1,579,105 | Form |
Investor Activity (13F)
Updated Sep 3, 2026Active 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.
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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