Ultragenyx Pharmaceutical (RARE)


Market Price (8/19/2026): $25.89 | Market Cap: $2.6 BilSector: Health Care | Industry: Biotechnology

Ultragenyx Pharmaceutical (RARE)


Market Price (8/19/2026): $25.89
Market Cap: $2.6 Bil
Sector: Health Care
Industry: Biotechnology

Investment Highlights Why It Matters Detailed financial logic regarding cash flow yields vs trend-riding momentum.

0

Cash is significant % of market cap
Net D/ENet Debt/Equity. Debt net of cash. Negative indicates net cash. Equity is taken as the Market Capitalization is -11%

Megatrend and thematic drivers
Megatrends include Biotechnology & Genomics, and Precision Medicine. Themes include Gene Editing & Therapy, Targeted Therapies, Show more.

Weak multi-year price returns
2Y Excs Rtn is -92%, 3Y Excs Rtn is -102%

Meaningful short interest
Short 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 15%

Not profitable at operating income level
Op 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 compensation
SBC/Rev LTMShare Based Compensation / Revenue (Sales), Last Twelve Months (LTM) is 19%

Not cash flow generative
CFO/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 negative
ERPEquity Risk Premium (ERP) = Total Yield - Risk Free Rate, Reflects the premium above risk free assets offered by the investment. is -27%

Key risks
RARE key risks include [1] an over-reliance on its single commercial product, Show more.

0 Cash is significant % of market cap
Net D/ENet Debt/Equity. Debt net of cash. Negative indicates net cash. Equity is taken as the Market Capitalization is -11%
1 Megatrend and thematic drivers
Megatrends include Biotechnology & Genomics, and Precision Medicine. Themes include Gene Editing & Therapy, Targeted Therapies, Show more.
2 Weak multi-year price returns
2Y Excs Rtn is -92%, 3Y Excs Rtn is -102%
3 Meaningful short interest
Short 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 15%
4 Not profitable at operating income level
Op 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%
5 Significant share based compensation
SBC/Rev LTMShare Based Compensation / Revenue (Sales), Last Twelve Months (LTM) is 19%
6 Not cash flow generative
CFO/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%
7 Yield minus risk free rate is negative
ERPEquity Risk Premium (ERP) = Total Yield - Risk Free Rate, Reflects the premium above risk free assets offered by the investment. is -27%
8 Key risks
RARE key risks include [1] an over-reliance on its single commercial product, Show more.

RARE in ETFs

Weight = RARE's share of each fund

VTI0.00%
ITOT0.00%
IWM0.08%
VB0.03%
XBI0.93%
IBB0.18%
IWN0.09%
VTWO0.08%
+8 more covered ETFs

Valuation & Metrics

Price Chart

Why The Stock Moved

Qualitative Assessment

AI Analysis | Feedback

Updated on 8/4/2026

Ultragenyx Pharmaceutical (RARE) stock has gained about 5% since 4/30/2026 because of the following key factors:

1. Reaffirmed 2026 Revenue Guidance and 2027 Profitability Outlook.

Despite reporting a net loss of $185 million in fiscal Q1 2026, Ultragenyx Pharmaceutical reaffirmed its full-year 2026 total revenue guidance of $730 million to $760 million. The company also reiterated its strategic objective to achieve profitability in fiscal 2027, which provided investors with confidence in its long-term financial trajectory.

2. Anticipation of Key Regulatory Approvals for Gene Therapies.

Investor sentiment was positively influenced by the approaching Prescription Drug User Fee Act (PDUFA) action dates for two of Ultragenyx's gene therapy candidates: DTX401 for Glycogen Storage Disease Type Ia, with an action date of August 23, 2026, and UX111 for Sanfilippo syndrome type A, with an action date of September 19, 2026. These represent potential new product launches that could significantly expand the company's commercial portfolio.

Show more
Updated on 8/4/2026

Ultragenyx Pharmaceutical (RARE) stock has gained about 5% since 4/30/2026 because of the following key factors:

1. Reaffirmed 2026 Revenue Guidance and 2027 Profitability Outlook.

Despite reporting a net loss of $185 million in fiscal Q1 2026, Ultragenyx Pharmaceutical reaffirmed its full-year 2026 total revenue guidance of $730 million to $760 million. The company also reiterated its strategic objective to achieve profitability in fiscal 2027, which provided investors with confidence in its long-term financial trajectory.

2. Anticipation of Key Regulatory Approvals for Gene Therapies.

Investor sentiment was positively influenced by the approaching Prescription Drug User Fee Act (PDUFA) action dates for two of Ultragenyx's gene therapy candidates: DTX401 for Glycogen Storage Disease Type Ia, with an action date of August 23, 2026, and UX111 for Sanfilippo syndrome type A, with an action date of September 19, 2026. These represent potential new product launches that could significantly expand the company's commercial portfolio.

3. Positive Clinical Development for Angelman Syndrome Program.

The company's stock benefited from continued positive longer-term data from the Phase 1/2 clinical study of GTX-102 for Angelman syndrome. This, coupled with the expectation of pivotal Phase 3 data from the ASPIRE study in the second half of fiscal 2026, contributed to sustained investor optimism regarding its late-stage pipeline.

4. Strong Fiscal Q2 2026 Financial Results.

Ultragenyx reported record total revenue of $214 million for fiscal Q2 2026, a 28.5% year-over-year increase, significantly surpassing the Zacks Consensus Estimate of $181.01 million. The company also narrowed its net loss to $92 million, or $0.90 per share, beating the analyst estimate of a $1.22 loss per share. This strong financial performance for the quarter, reported at the end of the analysis period, reinforced the positive trend.

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Stock Movement Drivers

Fundamental Drivers

The 3.2% change in RARE stock from 4/30/2026 to 8/18/2026 was primarily driven by a 6.5% change in the company's Total Revenues ($ Mil).
(LTM values as of)43020268182026Change
Stock Price ($)24.6925.493.2%
Change Contribution By: 
Total Revenues ($ Mil)6737176.5%
P/S Multiple3.73.6-1.1%
Shares Outstanding (Mil)100102-2.0%
Cumulative Contribution3.2%

LTM = Last Twelve Months as of date shown

Market Drivers

4/30/2026 to 8/18/2026
ReturnCorrelation
RARE3.2% 
Market (SPY)6.8%28.1%
Sector (XLV)16.3%8.4%

Fundamental Drivers

The 5.9% change in RARE stock from 1/31/2026 to 8/18/2026 was primarily driven by a 13.7% change in the company's Total Revenues ($ Mil).
(LTM values as of)13120268182026Change
Stock Price ($)24.0725.495.9%
Change Contribution By: 
Total Revenues ($ Mil)63171713.7%
P/S Multiple3.83.6-4.9%
Shares Outstanding (Mil)100102-2.1%
Cumulative Contribution5.9%

LTM = Last Twelve Months as of date shown

Market Drivers

1/31/2026 to 8/18/2026
ReturnCorrelation
RARE5.9% 
Market (SPY)11.2%38.6%
Sector (XLV)10.1%20.6%

Fundamental Drivers

The -6.7% change in RARE stock from 7/31/2025 to 8/18/2026 was primarily driven by a -18.8% change in the company's P/S Multiple.
(LTM values as of)73120258182026Change
Stock Price ($)27.3225.49-6.7%
Change Contribution By: 
Total Revenues ($ Mil)59071721.5%
P/S Multiple4.53.6-18.8%
Shares Outstanding (Mil)96102-5.5%
Cumulative Contribution-6.7%

LTM = Last Twelve Months as of date shown

Market Drivers

7/31/2025 to 8/18/2026
ReturnCorrelation
RARE-6.7% 
Market (SPY)22.4%24.7%
Sector (XLV)31.8%19.9%

Fundamental Drivers

The -40.9% change in RARE stock from 7/31/2023 to 8/18/2026 was primarily driven by a -54.2% change in the company's P/S Multiple.
(LTM values as of)73120238182026Change
Stock Price ($)43.1225.49-40.9%
Change Contribution By: 
Total Revenues ($ Mil)38471786.8%
P/S Multiple7.93.6-54.2%
Shares Outstanding (Mil)70102-30.9%
Cumulative Contribution-40.9%

LTM = Last Twelve Months as of date shown

Market Drivers

7/31/2023 to 8/18/2026
ReturnCorrelation
RARE-40.9% 
Market (SPY)73.6%28.9%
Sector (XLV)32.5%28.1%

Return vs. Risk

Price Returns Compared

 202120222023202420252026Total [1]
Returns
RARE Return-39%-45%3%-12%-45%14%-81%
Peers Return-30%-7%5%-35%190%9%40%
S&P 500 Return27%-19%24%23%16%13%106%

Monthly Win Rates [3]
RARE Win Rate33%25%33%33%58%50% 
Peers Win Rate42%50%42%33%67%75% 
S&P 500 Win Rate75%42%67%75%67%50% 

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 8/18/2026 (YTD)

How Low Can It Go

EventRARES&P 500
Summer-Fall 2023 Five Percent Yield Shock
  % Loss-23.7%-9.5%
  % Gain to Breakeven31.0%10.5%
  Time to Breakeven48 days24 days
2023 SVB Regional Banking Crisis
  % Loss-15.6%-6.7%
  % Gain to Breakeven18.4%7.1%
  Time to Breakeven39 days31 days
2020 COVID-19 Crash
  % Loss-45.1%-33.7%
  % Gain to Breakeven82.1%50.9%
  Time to Breakeven35 days140 days
Q4 2018 Fed Policy Error / Growth Scare
  % Loss-49.5%-19.2%
  % Gain to Breakeven98.2%23.8%
  Time to Breakeven546 days105 days
2016-2017 Trump Reflation Bond Selloff
  % Loss-26.4%-3.7%
  % Gain to Breakeven35.8%3.9%
  Time to Breakeven6 days6 days
2015-2016 China Devaluation / Global Growth Scare
  % Loss-55.8%-12.2%
  % Gain to Breakeven126.1%13.9%
  Time to Breakeven1737 days62 days

Compare to ARWR, IONS, APMD, ATTO, BLSM

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.

EventRARES&P 500
Summer-Fall 2023 Five Percent Yield Shock
  % Loss-23.7%-9.5%
  % Gain to Breakeven31.0%10.5%
  Time to Breakeven48 days24 days
2020 COVID-19 Crash
  % Loss-45.1%-33.7%
  % Gain to Breakeven82.1%50.9%
  Time to Breakeven35 days140 days
Q4 2018 Fed Policy Error / Growth Scare
  % Loss-49.5%-19.2%
  % Gain to Breakeven98.2%23.8%
  Time to Breakeven546 days105 days
2016-2017 Trump Reflation Bond Selloff
  % Loss-26.4%-3.7%
  % Gain to Breakeven35.8%3.9%
  Time to Breakeven6 days6 days
2015-2016 China Devaluation / Global Growth Scare
  % Loss-55.8%-12.2%
  % Gain to Breakeven126.1%13.9%
  Time to Breakeven1737 days62 days
2014-2016 Oil Price Collapse
  % Loss-26.7%-6.8%
  % Gain to Breakeven36.5%7.3%
  Time to Breakeven29 days15 days

Compare to ARWR, IONS, APMD, ATTO, BLSM

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.

AI Analysis | Feedback

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.

AI Analysis | Feedback

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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.
```

AI Analysis | Feedback

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.

AI Analysis | Feedback

  • 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)

AI Analysis | Feedback

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.

AI Analysis | Feedback

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.

  1. 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.
  2. 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.
  3. 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.

AI Analysis | Feedback

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AI Analysis | Feedback

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%.

AI Analysis | Feedback

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:

  1. 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.
  2. 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.
  3. 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.
  4. 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.

AI Analysis | Feedback

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.

Better Bets vs. Ultragenyx Pharmaceutical (RARE)

Peer Outperformance in Biotechnology

RARE has outperformed 54% of its 364 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 RARE has outperformed, by holding period. Peers without a full price history over a given window are excluded from that window.
1Y
42%
of 460 industry peers · -14.2% return
3Y
50%
of 406 industry peers · -27.4% return
5Y
54%
of 364 industry peers · -67.3% return
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Median price return by industry across the Health Care sector, ranked by 5Y. Biotechnology is RARE's own industry. Use this to see which corners of the sector have carried the returns.
Industry Names 1Y med3Y med5Y med Top 3 by 5Y
Health Care Distributors 7 9.7%79.7%140.2% CAH 411% · MCK 348% · COR 179%
Health Care Services 20 20.0%41.9%9.5% HIMS 302% · RDNT 143% · DGX 72%
Managed Health Care 8 33.2%-3.6%1.3% OSCR 142% · HQY 64% · ELV 14%
Health Care Facilities 24 11.9%-3.3%-15.7% THC 289% · NHC 263% · ENSG 132%
Health Care Equipment 51 -2.3%-9.2%-18.9% POCI 10738% · UFPT 396% · GKOS 288%
Health Care Supplies 12 38.3%-12.4%-27.5% LNTH 314% · HAE 81% · MMSI 31%
Pharmaceuticals 63 3.1%-1.7%-31.9% LQDA 2959% · INDV 1388% · ETON 1385%
Life Sciences Tools & Services 108 -8.4%-32.7%-70.6% SNWV 1953% · ELMD 259% · MEDP 235%
Health Care Technology 20 -28.4%-53.8%-73.9% SPOK 37% · HSTM -2% · VEEV -24%
Biotechnology ← 365 6.6%-27.4%-77.2% BRTX 106400% · DNTH 1803% · CELZ 1443%
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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Peer Comparisons

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Financials

RAREARWRIONSAPMDATTOBLSMMedian
NameUltragen.Arrowhea.Ionis Ph.Apnimed Attovia .BlossomH. 
Mkt Price25.4987.9059.6231.8123.2416.1028.65
Mkt Cap2.612.69.9---9.9
Rev LTM717669874---717
Op Inc LTM-528-226-594----528
FCF LTM-494-75-568----494
FCF 3Y Avg-476-208-410----410
CFO LTM-485-59-496----485
CFO 3Y Avg-459-130-358----358

Growth & Margins

RAREARWRIONSAPMDATTOBLSMMedian
NameUltragen.Arrowhea.Ionis Ph.Apnimed Attovia .BlossomH. 
Rev Chg LTM17.5%16.8%-7.4%---16.8%
Rev Chg 3Y Avg21.3%913.6%12.5%---21.3%
Rev Chg Q28.1%171.0%-40.7%---28.1%
QoQ Delta Rev Chg LTM7.0%7.6%-17.4%---7.0%
Op Inc Chg LTM-4.5%-109.7%-123.0%----109.7%
Op Inc Chg 3Y Avg7.3%-77.5%-24.5%----24.5%
Op Mgn LTM-73.6%-33.8%-68.0%----68.0%
Op Mgn 3Y Avg-90.2%-945.9%-47.0%----90.2%
QoQ Delta Op Mgn LTM10.1%1.9%-34.7%---1.9%
CFO/Rev LTM-67.6%-8.8%-56.8%----56.8%
CFO/Rev 3Y Avg-78.1%-598.1%-41.2%----78.1%
FCF/Rev LTM-68.9%-11.2%-64.9%----64.9%
FCF/Rev 3Y Avg-81.3%-908.4%-47.1%----81.3%

Valuation

RAREARWRIONSAPMDATTOBLSMMedian
NameUltragen.Arrowhea.Ionis Ph.Apnimed Attovia .BlossomH. 
Mkt Cap2.612.69.9---9.9
P/S3.618.811.3---11.3
P/Op Inc-4.9-55.7-16.6----16.6
P/EBIT-5.1-79.0-20.6----20.6
P/E-4.4-39.4-17.4----17.4
P/CFO-5.4-213.4-19.9----19.9
Total Yield-22.6%-2.5%-5.7%----5.7%
Dividend Yield0.0%0.0%0.0%---0.0%
FCF Yield 3Y Avg-13.6%-6.0%-4.8%----6.0%
D/E0.00.10.2---0.1
Net D/E-0.1-0.0-0.0----0.0

Returns

RAREARWRIONSAPMDATTOBLSMMedian
NameUltragen.Arrowhea.Ionis Ph.Apnimed Attovia .BlossomH. 
1M Rtn-12.9%18.3%9.6%27.2%6.1%0.6%7.9%
3M Rtn13.5%20.1%-19.3%27.2%6.1%0.6%9.8%
6M Rtn26.3%38.0%-26.7%27.2%6.1%0.6%16.2%
12M Rtn-14.2%341.5%37.3%27.2%6.1%0.6%16.7%
3Y Rtn-27.4%211.0%53.5%27.2%6.1%0.6%16.7%
1M Excs Rtn-14.0%19.3%9.3%23.9%2.8%-2.7%6.0%
3M Excs Rtn1.5%15.0%-21.7%23.3%2.2%-3.3%1.8%
6M Excs Rtn13.9%25.6%-39.1%14.8%-6.3%-11.8%3.8%
12M Excs Rtn-33.4%309.3%16.9%8.0%-13.1%-18.6%-2.6%
3Y Excs Rtn-102.1%132.4%-24.1%-45.1%-66.2%-71.7%-55.6%

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  DOJOLVItriheptanoinliquid63020208.8%88.5%21.9%-23.7%-66.6%
BLA761047  MEPSEVIIvestronidase alfa-vjbkinjectable111520170.7%35.1%8.4%-23.8%-44.8%
Collapse to Preview
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  DOJOLVItriheptanoinliquid63020208.8%88.5%21.9%-23.7%-66.6%
BLA761047  MEPSEVIIvestronidase alfa-vjbkinjectable111520170.7%35.1%8.4%-23.8%-44.8%

Financials

Segment Financials

Revenue by Segment
$ Mil20252024202320222021
Research, development and commercialization of its products673560434363 
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
Total673560434363351


Price Behavior

Price Behavior
Market Price$25.49 
Market Cap ($ Bil)2.6 
First Trading Date01/31/2014 
Distance from 52W High-30.2% 
   50 Days200 Days
DMA Price$28.26$26.62
DMA Trenddownup
Distance from DMA-9.8%-4.2%
 3M1YR
Volatility53.5%67.4%
Downside Capture-13.61173.18
Upside Capture45.22119.03
Correlation (SPY)26.6%26.2%
RARE Betas & Captures as of 7/31/2026

 1M2M3M6M1Y3Y
Beta1.270.971.111.491.281.05
Up Beta3.183.242.922.711.971.11
Down Beta1.551.601.481.370.580.69
Up Capture-148%20%41%104%131%97%
Bmk +ve Days11223567138427
Stock +ve Days5193061124368
Down Capture276%-5%51%113%135%108%
Bmk -ve Days11212859114326
Stock -ve Days17243364126379

[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-14.2%67.2%0.10-
Sector ETF (XLV)26.3%15.5%1.3119.0%
Equity (SPY)20.4%12.8%1.1726.3%
Gold (GLD)29.8%28.5%0.9124.2%
Commodities (DBC)40.2%20.2%1.56-5.4%
Real Estate (VNQ)13.1%13.9%0.658.4%
Bitcoin (BTCUSD)-45.4%42.7%-1.3014.5%

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-22.7%54.6%-0.25-
Sector ETF (XLV)6.6%15.0%0.2531.6%
Equity (SPY)13.1%17.2%0.5934.1%
Gold (GLD)19.8%18.6%0.8714.1%
Commodities (DBC)9.8%19.6%0.393.7%
Real Estate (VNQ)2.4%18.9%0.0230.7%
Bitcoin (BTCUSD)7.1%52.6%0.3216.1%

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-9.0%55.2%0.06-
Sector ETF (XLV)10.4%16.7%0.5141.1%
Equity (SPY)15.2%17.9%0.7240.8%
Gold (GLD)12.1%16.2%0.619.5%
Commodities (DBC)7.8%18.1%0.3510.6%
Real Estate (VNQ)4.8%20.7%0.2029.9%
Bitcoin (BTCUSD)59.9%66.0%1.0011.4%

Smart multi-asset allocation framework can stack odds in your favor. Learn How

Short Interest

Short Interest: As Of Date7312026
Short Interest: Shares Quantity15.0 Mil
Short Interest: % Change Since 715202611.4%
Average Daily Volume1.8 Mil
Days-to-Cover Short Interest8.3 days
Basic Shares Quantity101.9 Mil
Short % of Basic Shares14.7%

Earnings Returns History

Updated 8/13/2026
Expand for More
 Forward Returns
Earnings Date1D Returns5D Returns21D Returns
8/4/2026-3.4%4.5% 
5/5/20263.7%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   
# Positive101110
# Negative141313
Median Positive3.7%6.5%12.0%
Median Negative-4.0%-5.8%-7.3%
Max Positive14.3%13.9%28.6%
Max Negative-13.4%-24.1%-25.8%
Collapse to Preview
 Forward Returns
Earnings Date1D Returns5D Returns21D Returns
8/4/2026-3.4%4.5% 
5/5/20263.7%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%
8/1/202414.3%13.9%28.6%
5/2/2024-1.0%-4.6%-4.8%
2/15/20245.8%7.0%0.7%
11/2/20236.1%-4.5%12.6%
8/3/2023-8.8%-11.6%-8.5%
5/4/20233.1%2.8%11.9%
1/6/2023-13.0%-6.7%2.1%
11/2/2022-6.0%-16.9%-1.0%
7/28/20222.7%7.5%-4.6%
5/5/2022-13.4%-24.1%-25.8%
2/10/20222.1%-5.8%-12.9%
11/2/20211.4%-4.6%-6.7%
8/2/20213.6%11.3%19.1%
5/4/20214.1%6.5%-7.3%
2/11/2021-7.7%-9.0%-20.6%
10/27/2020-2.5%4.7%23.0%
SUMMARY STATS   
# Positive101110
# Negative141313
Median Positive3.7%6.5%12.0%
Median Negative-4.0%-5.8%-7.3%
Max Positive14.3%13.9%28.6%
Max Negative-13.4%-24.1%-25.8%

SEC Filings

Expand for More
Report DateFiling DateFiling
06/30/202608/05/202610-Q
03/31/202605/06/202610-Q
12/31/202502/18/202610-K
09/30/202511/05/202510-Q
06/30/202508/06/202510-Q
03/31/202505/07/202510-Q
12/31/202402/19/202510-K
09/30/202411/06/202410-Q
06/30/202408/02/202410-Q
03/31/202405/03/202410-Q
12/31/202302/21/202410-K
09/30/202311/03/202310-Q
06/30/202308/04/202310-Q
03/31/202305/05/202310-Q
12/31/202202/17/202310-K
09/30/202211/03/202210-Q
Collapse to Preview
Report DateFiling DateFiling
06/30/202608/05/202610-Q
03/31/202605/06/202610-Q
12/31/202502/18/202610-K
09/30/202511/05/202510-Q
06/30/202508/06/202510-Q
03/31/202505/07/202510-Q
12/31/202402/19/202510-K
09/30/202411/06/202410-Q
06/30/202408/02/202410-Q
03/31/202405/03/202410-Q
12/31/202302/21/202410-K
09/30/202311/03/202310-Q
06/30/202308/04/202310-Q
03/31/202305/05/202310-Q
12/31/202202/17/202310-K
09/30/202211/03/202210-Q
06/30/202207/29/202210-Q
03/31/202205/06/202210-Q
12/31/202102/15/202210-K
09/30/202111/03/202110-Q
06/30/202108/03/202110-Q
03/31/202105/05/202110-Q
12/31/202002/12/202110-K
09/30/202010/27/202010-Q
06/30/202007/31/202010-Q
03/31/202005/07/202010-Q
12/31/201902/14/202010-K
09/30/201911/06/201910-Q

Recent Forward Guidance

Updated 8/5/2026

Latest: Q2 2026 Earnings Reported 8/4/2026

Forward GuidanceGuidance Change
MetricLowMidHigh% Chg% DeltaChangePrior
2026 Total Revenue730.00 Mil745.00 Mil760.00 Mil0 AffirmedGuidance: 745.00 Mil for 2026
2026 Crysvita Revenue500.00 Mil510.00 Mil520.00 Mil0 AffirmedGuidance: 510.00 Mil for 2026
2026 Dojolvi Revenue100.00 Mil105.00 Mil110.00 Mil0 AffirmedGuidance: 105.00 Mil for 2026

null

Insider Activity

Updated 8/4/2026
Expand for More
#OwnerTitleHoldingActionFiling DatePriceSharesTransacted
Value
Value of
Held Shares
Form
1Parschauer, Karah HerdmanEVP and Chief Legal OfficerDirectSell804202624.921,90047,3482,306,645Form
2Horn, HowardChief Financial OfficerDirectSell804202624.924,696117,0242,399,671Form
3Horn, HowardChief Financial OfficerDirectSell706202633.134,698155,6453,345,832Form
4Sanders, Corazon (corsee) D DirectSell615202625.052,00050,100528,430Form
5Parschauer, Karah HerdmanEVP and Chief Legal OfficerDirectSell615202624.621,89946,7532,325,654Form
Collapse to Preview
#OwnerTitleHoldingActionFiling DatePriceSharesTransacted
Value
Value of
Held Shares
Form
1Parschauer, Karah HerdmanEVP and Chief Legal OfficerDirectSell804202624.921,90047,3482,306,645Form
2Horn, HowardChief Financial OfficerDirectSell804202624.924,696117,0242,399,671Form
3Horn, HowardChief Financial OfficerDirectSell706202633.134,698155,6453,345,832Form
4Sanders, Corazon (corsee) D DirectSell615202625.052,00050,100528,430Form
5Parschauer, Karah HerdmanEVP and Chief Legal OfficerDirectSell615202624.621,89946,7532,325,654Form
6Horn, HowardChief Financial OfficerDirectSell601202623.774,653110,6022,512,228Form
7Suliman, Shehnaaz DirectSell518202625.125,740144,189702,129Form
8Crombez, EricEVP and Chief Medical OfficerDirectSell506202624.963448,5862,775,277Form
9Horn, HowardChief Financial OfficerDirectSell504202624.904,683116,6072,747,516Form
10Horn, HowardChief Financial OfficerDirectSell402202620.974,68398,2031,684,960Form
11Parschauer, Karah HerdmanEVP and Chief Legal OfficerDirectSell310202622.673,50879,5261,514,809Form
12Parschauer, Karah HerdmanEVP and Chief Legal OfficerDirectSell310202621.406,018128,7851,505,019Form
13Horn, HowardChief Financial OfficerDirectSell303202622.401,63536,6241,904,762Form
14Pinion, John RichardSee RemarksDirectSell303202622.8010,029228,6612,508,593Form
15Harris, ErikEVP & Chief Commercial OfficerDirectSell303202622.8010,539240,2892,040,942Form
16Kakkis, Emil DPresident & CEODirectSell303202622.8054,4041,240,41115,025,063Form
17Parschauer, Karah HerdmanEVP and Chief Legal OfficerDirectSell303202622.808,135185,4781,740,689Form
18Huizenga, Theodore AlanSVP, Chief Accounting OfficerDirectSell303202622.801,63237,2101,109,676Form
19Horn, HowardChief Financial OfficerDirectSell303202622.708,409190,9141,967,695Form
20Crombez, EricEVP and Chief Medical OfficerDirectSell303202622.807,029160,2611,739,663Form
21Horn, HowardChief Financial OfficerDirectSell203202623.563,06172,1172,095,309Form
22Horn, HowardChief Financial OfficerDirectSell105202622.843,15071,9462,101,189Form
23Huizenga, Theodore AlanSVP, Chief Accounting OfficerDirectSell1230202534.38852,9221,734,471Form
24Horn, HowardChief Financial OfficerDirectSell1211202536.383,081112,0873,461,411Form
25Horn, HowardChief Financial OfficerDirectSell1014202531.517,942250,2523,095,133Form
26Huizenga, Theodore AlanSVP, Chief Accounting OfficerDirectSell919202529.17641,8671,465,559Form
27Parschauer, Karah HerdmanEVP and Chief Legal OfficerDirectSell912202531.172,45076,3662,283,857Form
28Huizenga, Theodore AlanSVP, Chief Accounting OfficerDirectSell908202531.39842,6371,579,105Form

Investor Activity (13F)

Updated Aug 19, 2026
13F 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 ManagerValue% of PortfolioTotal PositionsQoQFiling
Jones Hill Capital LP$24.4 Mil5.2%26New13F
Eagle Health Investments LP$18.5 Mil3.9%26New13F
Catalio Capital Management, LP$17.8 Mil3.7%41ADD +423.4%13F
Newtyn Management, LLC$8.4 Mil0.9%33ADD +35.2%13F
Active ManagerValue% of PortfolioTotal PositionsQoQFiling
Jones Hill Capital LP$24.4 Mil5.2%26New13F
Eagle Health Investments LP$18.5 Mil3.9%26New13F
Catalio Capital Management, LP$17.8 Mil3.7%41ADD +423.4%13F
Newtyn Management, LLC$8.4 Mil0.9%33ADD +35.2%13F
Active ManagerValue% of PortfolioTotal PositionsQoQFiling
Great Point Partners LLC$6.9 Mil2.2%29Exited13F
Active ManagerValue% of PortfolioTotal PositionsQoQFiling
Jones Hill Capital LP$24.4 Mil5.2%26New13F
Eagle Health Investments LP$18.5 Mil3.9%26New13F
Catalio Capital Management, LP$17.8 Mil3.7%41ADD +423.4%13F
Newtyn Management, LLC$8.4 Mil0.9%33ADD +35.2%13F
Core Cache Last Updated: 8/18/2026