RARE
HealthcareUltragenyx Pharmaceutical Inc. · Biotechnology · $2B
What is Ultragenyx Pharmaceutical Inc.?
Ultragenyx Pharmaceutical is a biopharmaceutical company dedicated to developing and commercializing treatments for rare and ultra-rare genetic diseases. Operating across North America, Europe, and international markets, it targets conditions that have historically had few or no approved therapies.
Ultragenyx generates revenue by bringing approved biologic therapies to patients with rare genetic disorders, while simultaneously advancing a pipeline of gene therapies and other novel modalities. The company earns through direct product sales and benefits from collaboration and licensing agreements with partners including Kyowa Kirin, REGENXBIO, Bayer Healthcare, and others. Its business model centers on identifying underserved rare disease populations and developing targeted treatments — biologics, enzyme replacement therapies, and gene therapies — for those patients.
Founded in 2014 and headquartered in Novato, California, Ultragenyx has built a focused pipeline around genetic medicine.
- Crysvita (burosumab) — antibody therapy for X-linked hypophosphatemia and tumor-induced osteomalacia
- Mepsevii — enzyme replacement therapy for Mucopolysaccharidosis VII
- Dojolvi — treatment for long-chain fatty acid oxidation disorders
- Evkeeza (evinacumab) — therapy for homozygous familial hypercholesterolemia
- Gene therapy pipeline — AAV8 candidates including DTX401, DTX301, and UX701
Is RARE a Good Stock to Buy?
UQS Score rates RARE as Below Average overall, reflecting meaningful trade-offs across its five quality pillars.
The Growth pillar stands out as a relative bright spot, consistent with a company expanding its commercial footprint in rare disease markets. The Risk pillar also registers as Good, suggesting the business carries a manageable risk profile relative to early-stage biotech peers — a reflection of its diversified approved product base and established partnerships.
The Quality pillar scores as Weak, pointing to underlying profitability challenges common in rare disease companies still scaling commercial operations. Valuation is flagged as Elevated, meaning the market may already be pricing in considerable future success.
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Past performance does not guarantee future results. UQS Score is based on fundamental data and is not a buy/sell recommendation.
Does RARE pay dividends?
No — Ultragenyx Pharmaceutical Inc. does not currently pay a dividend.
Ultragenyx does not currently pay a dividend. This is typical for clinical-stage and early-commercial biopharmaceutical companies, which generally reinvest available capital into research, pipeline development, and commercialization efforts rather than returning cash to shareholders. Investors in RARE are generally seeking growth from pipeline advancement rather than income.
When does RARE report earnings?
Ultragenyx Pharmaceutical reports earnings on a quarterly cadence, consistent with standard practice for US-listed equities.
Each quarterly report provides updates on commercial product revenues, pipeline milestones, and operating expenditures — all closely watched by investors given the company's ongoing investment in gene therapy and other novel programs. Results tend to be evaluated in the context of product uptake trends and clinical progress rather than near-term profitability.
For the most recent quarter's results and upcoming reporting dates, visit Ultragenyx's investor relations page directly.
RARE Price History
-76.3% over 5Y
Monthly close, adjusted for stock splits and dividend reinvestment.
What if I invested in Ultragenyx Pharmaceutical Inc.?
Based on Ultragenyx Pharmaceutical Inc.'s historical closing prices, adjusted for stock splits and dividend reinvestment. Past performance does not guarantee future results. This is for informational purposes only and is not financial advice.
RARE Long-term Outlook
The UQS Growth pillar rating of Good suggests Ultragenyx has identifiable drivers that could support revenue expansion over the medium term, primarily through continued uptake of its approved therapies and potential pipeline readouts. However, the Elevated Valuation pillar indicates the current share price may leave limited margin of safety if execution falls short. The Good Risk rating provides some reassurance that the company's risk profile is not extreme relative to sector peers, but the Weak Quality pillar is a reminder that durable profitability has not yet been established.
Growth drivers
- Continued commercial expansion of Crysvita and other approved rare disease therapies
- Pipeline advancement of gene therapy candidates toward potential approval
- Collaboration milestones and licensing revenue from established partnerships
Key risks
- Sustained operating losses weighing on the Quality pillar and cash runway
- Elevated valuation leaving limited buffer against clinical or commercial setbacks
- Execution risk inherent in gene therapy development and regulatory pathways
RARE vs Peers
Ultragenyx operates in a competitive rare disease and specialty biotech landscape alongside companies pursuing similarly focused therapeutic strategies.
Arcus focuses primarily on immuno-oncology combinations, differentiating it from Ultragenyx's genetic disease and enzyme replacement therapy focus.
Kodiak concentrates on retinal disease treatments, representing a distinct therapeutic area compared to Ultragenyx's rare metabolic and genetic disorder portfolio.
Ascentage targets hematologic malignancies and solid tumors with apoptosis-focused therapies, a different disease focus from Ultragenyx's rare genetic disease mission.
Frequently Asked Questions
What does Ultragenyx Pharmaceutical do?
Ultragenyx is a biopharmaceutical company that develops and commercializes treatments for rare and ultra-rare genetic diseases. It has multiple approved biologic therapies on the market and an active pipeline of gene therapies and other novel treatments targeting conditions with limited or no existing options.
Does RARE pay dividends?
No, Ultragenyx does not pay a dividend. The company reinvests its resources into research, pipeline development, and commercialization of rare disease therapies. Investors in RARE are typically focused on potential growth from pipeline and commercial expansion rather than dividend income.
When does RARE report earnings?
Ultragenyx reports on a standard quarterly cadence. Each report covers commercial product revenues, pipeline updates, and operating expenses. For confirmed upcoming reporting dates, check the investor relations section of the Ultragenyx website directly.
Is RARE a good stock to buy?
UQS Score rates RARE as Below Average overall. The Growth and Risk pillars show relative strength, but the Weak Quality pillar and Elevated Valuation are meaningful considerations. Whether RARE fits a portfolio depends on an investor's risk tolerance and time horizon — the full pillar breakdown is available to Pro members.
Is RARE overvalued?
The UQS Valuation pillar for RARE is rated Elevated, suggesting the market is pricing in a significant amount of future success. This does not necessarily mean the stock will decline, but it does indicate that the current price leaves less room for error if commercial or clinical execution disappoints.
How does RARE compare to its competitors?
Ultragenyx is differentiated by its specific focus on rare and ultra-rare genetic diseases, with an approved product portfolio spanning biologics and enzyme replacement therapies alongside a gene therapy pipeline. Peers like Arcus Biosciences and Kodiak Sciences operate in distinct therapeutic areas, making direct comparisons more about business model and risk profile than head-to-head competition.
What is RARE's market cap bracket?
Ultragenyx Pharmaceutical is classified as a mid-cap company. This places it in a category that typically offers more established commercial operations than small-cap biotechs, while still carrying meaningful pipeline-driven growth potential and associated risk.
Who founded Ultragenyx Pharmaceutical?
Ultragenyx was founded in 2014. Information about its founding team is widely available through public sources including the company's own website and SEC filings, which provide detailed background on its leadership history.
Is RARE a long-term quality investment?
As a long-term quality indicator, the UQS Score currently rates RARE as Below Average. The Growth and Risk pillars offer some encouragement for long-term investors, but the Weak Quality pillar reflects that the company has not yet demonstrated durable profitability. Long-term quality depends heavily on pipeline execution and commercial scaling.
What is the main competitive advantage of Ultragenyx?
Ultragenyx's focus on rare and ultra-rare genetic diseases provides a degree of natural insulation from broad competitive pressure, as these markets are small and require highly specialized development expertise. Its established partnerships with academic institutions and biotech companies also support its pipeline development capabilities. The UQS Moat pillar rates this advantage as Neutral.
What sector does RARE belong to?
Ultragenyx Pharmaceutical operates in the Healthcare sector, specifically within the biopharmaceutical industry. Its work spans rare disease biologics and next-generation gene therapy, placing it among specialty biotech companies rather than broad-market pharmaceutical firms.
Is RARE a growth stock or value stock?
Based on its UQS pillar profile, RARE leans toward growth — the Growth pillar is rated Good, reflecting commercial and pipeline expansion potential. However, the Elevated Valuation pillar means investors are already paying a premium for that growth, which is a characteristic more common in growth-oriented names than traditional value stocks.
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Pro Analysis
RARE — Score History
| Date | UQS | Quality | Moat | Growth | Risk | Value | Change |
|---|---|---|---|---|---|---|---|
| May 20, 2026 | 43.2 | 32.1 | 40.0 | 70.9 | 73.1 | 0.0 | +3.6 |
| Apr 2, 2026 | 39.6 | 15.4 | 40.0 | 70.9 | 77.5 | 0.0 | — |
RARE — Pillar Breakdown
Quality
— 32.1/100 (25%)Ultragenyx Pharmaceutical Inc. currently shows below-average quality metrics, suggesting challenges with profitability.
How effectively capital is deployed to generate returns.
Profitability relative to shareholders' equity.
Ability to convert revenue into operating profit.
Bottom-line profit as a share of revenue.
Asset productivity — how much gross profit each dollar of assets generates.
Free cash flow relative to market value.
Growth
— 70.9/100 (20%)Ultragenyx Pharmaceutical Inc. demonstrates healthy growth trends across revenue and earnings.
Revenue trajectory over the last twelve months.
Compound annual revenue growth rate over 3 years.
Year-over-year earnings per share growth.
Analyst consensus for future revenue growth.
Risk
— 73.1/100 (15%)Ultragenyx Pharmaceutical Inc. maintains a reasonable risk profile with manageable debt levels.
Debt levels relative to earnings capacity.
Total debt relative to shareholder equity.
Short-term liquidity — ability to pay near-term obligations.
Earnings capacity relative to interest payments.
Valuation
— 0.0/100 (15%)Ultragenyx Pharmaceutical Inc. appears expensively valued relative to its fundamentals and growth prospects.
Moat
— 40/100 (25%)Ultragenyx Pharmaceutical Inc. possesses some competitive advantages but faces meaningful competition. The Moat pillar evaluates competitive advantages across five dimensions: Switching Costs, Network Effects, Cost Advantage, Intangible Assets, and Scale & Ecosystem. Sign in to customize moat ratings for RARE.
Score Composition
Financial Data
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How is the RARE UQS Score Calculated?
The UQS (Unified Quality Score) for Ultragenyx Pharmaceutical Inc. is calculated using a proprietary 6-pillar framework with 29 financial metrics. Each pillar evaluates a different dimension on a 0–100 scale, then combines into a single weighted score. Scoring thresholds are calibrated per sector. Momentum is an optional Pro toggle — without it, you get the 5-pillar / 25-metric core shown below.
Quality (25%) measures profitability and capital efficiency — ROIC, ROE, margins, GP/Assets, and FCF Yield.
Moat (25%) assesses Ultragenyx Pharmaceutical Inc.'s competitive advantages across switching costs, network effects, cost advantages, intangible assets, and ecosystem scale.
Growth (20%) tracks revenue trajectory and earnings momentum, combining historical results with analyst forward estimates.
Risk (15%) is inversely scored — lower leverage and strong balance sheet health result in higher scores.
Valuation (15%) measures whether Ultragenyx Pharmaceutical Inc. is fairly priced using earnings yield, price-to-FCF, PEG ratio, and EV/EBITDA relative to sector peers.
Six investor-inspired presets are available, each with different pillar weights: Balanced, Buffett, Munger, Lynch, Cathie Wood, and Graham. The public score shown here uses the Balanced preset. Learn more in our FAQ.