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Ultragenyx (RARE)



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Upturn Advisory Summary
08/14/2025: RARE (2-star) is currently NOT-A-BUY. Pass it for now.
1 Year Target Price $86.05
1 Year Target Price $86.05
13 | Strong Buy |
6 | Buy |
1 | Hold |
0 | Sell |
0 | Strong Sell |
Analysis of Past Performance
Type Stock | Historic Profit 4.29% | Avg. Invested days 32 | Today’s Advisory PASS |
Upturn Star Rating ![]() ![]() | Upturn Advisory Performance ![]() | Stock Returns Performance ![]() |
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Key Highlights
Company Size Mid-Cap Stock | Market Capitalization 2.80B USD | Price to earnings Ratio - | 1Y Target Price 86.05 |
Price to earnings Ratio - | 1Y Target Price 86.05 | ||
Volume (30-day avg) 20 | Beta 0.24 | 52 Weeks Range 25.81 - 60.37 | Updated Date 08/15/2025 |
52 Weeks Range 25.81 - 60.37 | Updated Date 08/15/2025 | ||
Dividends yield (FY) - | Basic EPS (TTM) -5.48 |
Analyzing Revenue: Products, Geography and Growth
Revenue by Products
Product revenue - Year on Year
Revenue by Geography
Geography revenue - Year on Year
Earnings Date
Report Date 2025-08-05 | When - | Estimate -1.3 | Actual -1.17 |
Profitability
Profit Margin -87.34% | Operating Margin (TTM) -64.8% |
Management Effectiveness
Return on Assets (TTM) -21.59% | Return on Equity (TTM) -180.44% |
Valuation
Trailing PE - | Forward PE - | Enterprise Value 2354653363 | Price to Sales(TTM) 4.58 |
Enterprise Value 2354653363 | Price to Sales(TTM) 4.58 | ||
Enterprise Value to Revenue 3.86 | Enterprise Value to EBITDA -6.46 | Shares Outstanding 96371600 | Shares Floating 92796064 |
Shares Outstanding 96371600 | Shares Floating 92796064 | ||
Percent Insiders 3.14 | Percent Institutions 100.27 |
Upturn AI SWOT
Ultragenyx

Company Overview
History and Background
Ultragenyx Pharmaceutical Inc. was founded in 2003. It focuses on developing treatments for rare and ultra-rare genetic diseases. The company has grown from a research-focused startup to a commercial-stage biopharmaceutical company with multiple approved products.
Core Business Areas
- Biologics: Development and commercialization of biologics for rare diseases, including enzyme replacement therapies and monoclonal antibodies.
- Small Molecules: Development and commercialization of small molecule drugs for rare genetic disorders.
- Gene Therapy: Development of gene therapies aimed at providing long-term or potentially curative treatments for genetic diseases.
Leadership and Structure
Emil D. Kakkis, M.D., Ph.D., is the CEO. The organizational structure includes research and development, commercial operations, manufacturing, and administrative functions. The company has a board of directors overseeing strategic direction.
Top Products and Market Share
Key Offerings
- Crysvita (burosumab): Crysvita is a monoclonal antibody indicated for the treatment of X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO). Revenue in 2023 was significant, but specific market share data fluctuates and requires up-to-date sources. Competitors include Kyowa Kirin, the developer of Crysvita, with Ultragenyx commercializing in certain regions.
- Dojolvi (triheptanoin): Dojolvi is a medium-chain triglyceride indicated for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD). Market share information varies; however, revenue is consistently growing and it is the only product approved for this indication. There are some alternative therapies and dietary management options for LC-FAOD.
- Mepsevii (vestronidase alfa): Mepsevii is an enzyme replacement therapy used to treat Mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome. Ultragenyx has a dominant market share in this rare disease niche. Competitors are other companies developing therapies for rare diseases, though none specifically targeting MPS VII.
- Evkeeza (evinacumab-dgnb): Evkeeza is a monoclonal antibody indicated as an adjunct to other lipid-lowering therapies for the treatment of adults and adolescents (u226512 years of age) with homozygous familial hypercholesterolemia (HoFH). It is in competition with products from Regeneron and Amgen.
Market Dynamics
Industry Overview
The biopharmaceutical industry, particularly the rare disease segment, is characterized by high R&D costs, complex regulatory pathways, and unmet medical needs. Increased regulatory incentives and scientific advancements are driving growth.
Positioning
Ultragenyx is a leader in developing therapies for rare and ultra-rare genetic diseases. Its competitive advantages include its focus on unmet medical needs, strong R&D capabilities, and expertise in navigating regulatory approvals for orphan drugs.
Total Addressable Market (TAM)
The overall rare disease market is estimated at hundreds of billions of dollars. Ultragenyx is positioned to capture a significant portion of this TAM through its diverse pipeline and approved therapies for specific indications.
Upturn SWOT Analysis
Strengths
- Focus on rare diseases with high unmet needs
- Strong R&D pipeline and drug development capabilities
- Established commercial infrastructure for orphan drugs
- Experienced management team with expertise in rare disease therapeutics
- Proprietary technology platforms
Weaknesses
- High R&D expenses and reliance on successful clinical trials
- Dependence on a limited number of products for revenue generation
- Challenges in manufacturing complex biologics
- Potential competition from other companies developing therapies for the same rare diseases
- Regulatory hurdles and pricing pressures
Opportunities
- Expanding pipeline through internal R&D and strategic acquisitions
- Geographic expansion into new markets
- Leveraging gene therapy technologies to develop curative treatments
- Collaborations and partnerships with other biopharmaceutical companies
- Expanding indications for existing approved therapies
Threats
- Clinical trial failures and regulatory setbacks
- Competition from larger pharmaceutical companies with greater resources
- Pricing pressures and reimbursement challenges for orphan drugs
- Patent expirations and generic competition
- Economic downturns impacting healthcare spending
Competitors and Market Share
Key Competitors
- BMY
- AMGN
- GILD
- VRTX
Competitive Landscape
Ultragenyx benefits from its focus on ultra-rare conditions, often facing less direct competition for individual indications. However, larger pharmaceutical companies with broader rare disease portfolios pose a competitive threat. Ultragenyx needs to continue to innovate in the space to capture more market share.
Major Acquisitions
GeneTx Biotherapeutics
- Year: 2020
- Acquisition Price (USD millions): 76
- Strategic Rationale: Acquisition of GeneTx expanded Ultragenyx's pipeline with a potential treatment for Angelman syndrome.
Growth Trajectory and Initiatives
Historical Growth: Ultragenyx has experienced significant revenue growth since its first product approvals. The company's growth has been fueled by its focus on rare diseases and its ability to successfully commercialize its therapies.
Future Projections: Analysts project continued revenue growth for Ultragenyx, driven by the expansion of existing products and the potential approval of new therapies in its pipeline. Profitability is expected to improve over time as revenue scales and R&D expenses are managed.
Recent Initiatives: Recent initiatives include strategic acquisitions, pipeline expansion, and geographic expansion into new markets.
Summary
Ultragenyx is a focused biopharmaceutical company targeting rare and ultra-rare genetic diseases. While its revenue is growing, the company is not profitable, but it does have a strong R&D pipeline and established commercial infrastructure. High R&D expenses and competition from larger firms pose challenges, but the company is poised for growth through strategic acquisitions and pipeline expansion. It needs to watch competition and maintain its innovative edge.
Peer Comparison
Sources and Disclaimers
Data Sources:
- Company filings (SEC)
- Analyst reports
- Industry publications
- Company Website
Disclaimers:
This analysis is for informational purposes only and does not constitute financial advice. Market share data is approximate and may vary based on source. Financial data is dynamic and should be verified with the latest company filings. This analysis uses past data to predict future results and is not indicative of actual future performance.
AI Summarization is directionally correct and might not be accurate.
Summarized information shown could be a few years old and not current.
Fundamental Rating based on AI could be based on old data.
AI-generated summaries may have inaccuracies (hallucinations). Please verify the information before taking action.
About Ultragenyx
Exchange NASDAQ | Headquaters Novato, CA, United States | ||
IPO Launch date 2014-01-31 | Founder, President, CEO & Director Dr. Emil D. Kakkis M.D., Ph.D. | ||
Sector Healthcare | Industry Biotechnology | Full time employees 1294 | Website https://www.ultragenyx.com |
Full time employees 1294 | Website https://www.ultragenyx.com |
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.

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