Ultragenyx Pharmaceutical (RARE) Stock Overview
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. More details
| Snowflake Score | |
|---|---|
| Valuation | 4/6 |
| Future Growth | 5/6 |
| Past Performance | 0/6 |
| Financial Health | 0/6 |
| Dividends | 0/6 |
RARE Community Fair Values
Create NarrativeSee what 11 others think this stock is worth. Follow their fair value or set your own to get alerts.
Analyst Price Targets
Ultragenyx Pharmaceutical Inc. Competitors
Price History & Performance
| Historical stock prices | |
|---|---|
| Current Share Price | US$21.59 |
| 52 Week High | US$42.37 |
| 52 Week Low | US$18.29 |
| Beta | 0.38 |
| 1 Month Change | -17.34% |
| 3 Month Change | -5.47% |
| 1 Year Change | -41.71% |
| 3 Year Change | -58.00% |
| 5 Year Change | -79.01% |
| Change since IPO | -48.90% |
Recent News & Updates
Narrative Update • Jun 05
RARE: Gene Therapy Milestones Will Support 2027 Profitability And Renew Pipeline Confidence
Ultragenyx Pharmaceutical's updated analyst price target has shifted modestly as analysts balance a slightly lower fair value estimate of about $52.05 per share with revised assumptions for higher revenue, stronger profit margins, and a lower future P/E, supported by recent research that highlights differing views on the potential of GTX-102 and the broader pipeline. Analyst Commentary Recent Street research on Ultragenyx shows a wide range of views, with some analysts lifting price targets on the back of GTX-102 and others trimming targets or downgrading the stock as they reassess key programs and pipeline risk.
Narrative Update • May 18
RARE: 2026 Guidance And Pipeline Milestones Will Drive Gene Therapy Repricing
Ultragenyx Pharmaceutical's updated analyst price target edges slightly lower by $1, as analysts factor in reduced targets across several firms along with ongoing expectations around the potential of GTX-102 and the broader pipeline. Analyst Commentary Recent Street research around Ultragenyx shows a mix of caution and optimism, with most firms trimming price targets while still highlighting key assets like GTX-102, setrusumab and the existing commercial portfolio.Narrative Update • Apr 28
RARE: 2026 Guidance And Restructuring Will Drive Gene Therapy Repricing
Ultragenyx's updated analyst price target holds steady at $84, with analysts factoring in lower revenue growth and profit margin assumptions, alongside a higher future P/E multiple as they reassess contributions from GTX-102, setrusumab and the broader pipeline. Analyst Commentary Recent Street research shows a mix of cautious and constructive views on Ultragenyx, with several firms revisiting price targets and reworking pipeline assumptions for GTX-102, setrusumab and the commercial portfolio.Recent updates
Narrative Update • Jun 05
RARE: Gene Therapy Milestones Will Support 2027 Profitability And Renew Pipeline Confidence
Ultragenyx Pharmaceutical's updated analyst price target has shifted modestly as analysts balance a slightly lower fair value estimate of about $52.05 per share with revised assumptions for higher revenue, stronger profit margins, and a lower future P/E, supported by recent research that highlights differing views on the potential of GTX-102 and the broader pipeline. Analyst Commentary Recent Street research on Ultragenyx shows a wide range of views, with some analysts lifting price targets on the back of GTX-102 and others trimming targets or downgrading the stock as they reassess key programs and pipeline risk.Narrative Update • May 18
RARE: 2026 Guidance And Pipeline Milestones Will Drive Gene Therapy Repricing
Ultragenyx Pharmaceutical's updated analyst price target edges slightly lower by $1, as analysts factor in reduced targets across several firms along with ongoing expectations around the potential of GTX-102 and the broader pipeline. Analyst Commentary Recent Street research around Ultragenyx shows a mix of caution and optimism, with most firms trimming price targets while still highlighting key assets like GTX-102, setrusumab and the existing commercial portfolio.Narrative Update • Apr 28
RARE: 2026 Guidance And Restructuring Will Drive Gene Therapy Repricing
Ultragenyx's updated analyst price target holds steady at $84, with analysts factoring in lower revenue growth and profit margin assumptions, alongside a higher future P/E multiple as they reassess contributions from GTX-102, setrusumab and the broader pipeline. Analyst Commentary Recent Street research shows a mix of cautious and constructive views on Ultragenyx, with several firms revisiting price targets and reworking pipeline assumptions for GTX-102, setrusumab and the commercial portfolio.
Narrative Update • Apr 14
RARE: Restructuring And Gene Therapy Decisions Will Shape Balanced Risk Reward
Analysts have adjusted Ultragenyx Pharmaceutical's Street price targets to a range of about $34 to $79, reflecting reduced assumptions for select pipeline assets and revenue growth, while revised models still incorporate improved profit margin expectations and a lower future P/E. Analyst Commentary Recent Street research shows that while several firms still maintain positive ratings on Ultragenyx Pharmaceutical, a series of target cuts and at least one downgrade highlight areas where expectations have become more restrained.Seeking Alpha • Apr 12
Ultragenyx Pharmaceutical: The Market Is Overlooking 2026 Catalysts
Summary Ultragenyx Pharmaceutical remains a speculative conviction hold (3/5) as its commercial base funds a robust late-stage rare disease pipeline. Multiple pivotal catalysts in 2026–2027, including GTX-102, DTX401, and UX111, could drive significant revenue growth and shift sentiment. Conservative U.S.-only modeling suggests $1.5B–$2.4B peak annual revenue potential from late-stage programs, with Street estimates supporting multi-bagger upside. Risks include small patient pools, payer friction, gene therapy complexities, competition, and potential dilution if pipeline progress falters. Read the full article on Seeking AlphaNarrative Update • Mar 31
RARE: Gene Therapy Readouts Will Drive 2027 Profitability And Reignite Pipeline Confidence
The updated fair value estimate for Ultragenyx Pharmaceutical has shifted from $64.00 to $52.45 as analysts factor in lower revenue growth assumptions, a higher discount rate, and reduced expectations for key pipeline assets following several price target cuts across the Street. Analyst Commentary Recent Street research on Ultragenyx Pharmaceutical reflects a mixed setup, with price targets broadly reset lower but many ratings still leaning positive.Narrative Update • Mar 17
RARE: 2026 Guidance And Restructuring Are Expected To Unlock Pipeline Repricing
Analysts have reduced their fair value estimate for Ultragenyx Pharmaceutical to $84 from $110.60, reflecting lower price targets across the Street as they factor in recent clinical setbacks, pipeline uncertainty, and updated assumptions around profitability and future P/E levels. Analyst Commentary Street research on Ultragenyx Pharmaceutical shows a clear reset in valuation frameworks, with most firms trimming price targets but largely maintaining positive ratings.Narrative Update • Mar 02
RARE: Recalibrated Outlook Where Restructuring And Angelman Data Will Define Risk Reward
Our updated fair value estimate for Ultragenyx Pharmaceutical moves to $25. This reflects a series of lower Street price targets as analysts factor in softer revenue growth assumptions, reduced profit margin expectations, a higher discount rate, and the recent restructuring following disappointing OI news, even as they retain generally positive views on the long term pipeline and commercial profile.Narrative Update • Feb 16
RARE: 2026 Guidance And Restructuring Are Expected To Support 2027 Profitability
Analysts have trimmed their average price target on Ultragenyx Pharmaceutical by about $10, reflecting more cautious assumptions on long term fair value after recent cuts tied to updated guidance and uneven Crysvita ordering trends. Analyst Commentary Recent Street research on Ultragenyx Pharmaceutical has centered on recalibrated price targets and refreshed assumptions around growth, timing, and execution.Narrative Update • Feb 02
RARE: 2027 Profitability Will Be Driven By Gene Therapy Readouts
Ultragenyx Pharmaceutical's updated analyst price target has edged higher to about US$64, as analysts weigh slightly stronger modeled revenue growth and a much higher projected profit margin against recent price target trims from BofA, Morgan Stanley and TD Cowen, along with a fresh Overweight initiation at Barclays. Analyst Commentary Recent research notes show a mix of optimism and caution around Ultragenyx Pharmaceutical, with price targets adjusted but ratings generally maintained in the positive camp.Narrative Update • Jan 19
RARE: 2027 Profitability Target Will Be Driven By Upcoming Trial Readouts
Ultragenyx Pharmaceutical's updated analyst price target moves lower from about US$81.85 to roughly US$63.90 as analysts factor in reduced revenue growth and profit margin assumptions, along with a higher discount rate, even though recent Street targets remain above current levels. Analyst Commentary Recent Street research on Ultragenyx Pharmaceutical points to a more cautious stance on valuation, even as many analysts maintain constructive views on the company’s long term execution and revenue potential.Narrative Update • Jan 05
RARE: Reaffirmed 2027 Profitability Outlook Will Support Rare Disease Pipeline Execution
Ultragenyx Pharmaceutical's updated analyst price target has shifted modestly lower, with the fair value estimate moving from about $128.00 to roughly $120.29, as analysts factor in recent Street research that reflects adjusted targets following mixed revenue outcomes and reaffirmed medium term guidance. Analyst Commentary Recent Street research shows that bullish analysts are trimming their price targets but still assigning upside potential relative to current trading levels, reflecting a view that the recent revenue mix and reaffirmed guidance continue to support the long term story.Analysis Article • Dec 30
The Market Doesn't Like What It Sees From Ultragenyx Pharmaceutical Inc.'s (NASDAQ:RARE) Revenues Yet As Shares Tumble 43%
Ultragenyx Pharmaceutical Inc. ( NASDAQ:RARE ) shareholders won't be pleased to see that the share price has had a very...Narrative Update • Dec 21
RARE: Pipeline Execution And Angelman Updates Will Shape Future Risk Reward Balance
Ultragenyx Pharmaceutical's narrative fair value has been revised modestly lower, from approximately $38.23 to $35.00 per share, as analysts temper long term revenue growth and valuation assumptions despite generally constructive views following recent price target reductions and reaffirmed profitability milestones. Analyst Commentary Recent Street research reflects a more tempered outlook on Ultragenyx, with several Bearish analysts trimming price targets while broadly maintaining positive ratings.Narrative Update • Dec 07
RARE Upcoming Catalyst Watch And Trial Readouts Will Drive 2027 Profitability
Ultragenyx Pharmaceutical's analyst price target has been modestly trimmed by analysts, with fair value edging down to approximately $81.85 from about $83.40. This reflects slightly lighter total revenue versus strong Crysvita performance, modestly higher risk assumptions, and a still favorable reward or risk profile supported by positive late stage trial expectations and reaffirmed profitability timelines.Narrative Update • Nov 23
RARE: Upcoming Study Data Will Drive Risk Reward Into 2027 Profitability
Ultragenyx Pharmaceutical's analyst price target has been revised downward by analysts, dropping from $84.10 to $83.40. This change reflects recent updates to revenue outlooks and profit margin expectations following lighter-than-anticipated quarterly revenues and sector-wide target adjustments.Narrative Update • Nov 07
RARE: Upcoming Study Results Will Drive Future Reward Versus Risk Profile
Ultragenyx Pharmaceutical’s consensus analyst price target has decreased from approximately $86 to $75, as analysts cite slightly lighter total revenues, partially offset by robust product performance and reaffirmed profitability guidance. Analyst Commentary Analysts provided updated price targets and shared their perspectives on Ultragenyx Pharmaceutical's recent performance, balancing positive revenue highlights with areas of caution regarding growth outlook and valuation.Analysis Article • Oct 28
Is Ultragenyx Pharmaceutical (NASDAQ:RARE) Using Too Much Debt?
Some say volatility, rather than debt, is the best way to think about risk as an investor, but Warren Buffett famously...Analysis Article • Jul 03
Why Investors Shouldn't Be Surprised By Ultragenyx Pharmaceutical Inc.'s (NASDAQ:RARE) Low P/S
You may think that with a price-to-sales (or "P/S") ratio of 6.3x Ultragenyx Pharmaceutical Inc. ( NASDAQ:RARE ) is a...Analysis Article • May 09
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Just Released Its First-Quarter Results And Analysts Are Updating Their Estimates
Ultragenyx Pharmaceutical Inc. ( NASDAQ:RARE ) shareholders are probably feeling a little disappointed, since its...Analysis Article • May 08
Shareholders Will Probably Hold Off On Increasing Ultragenyx Pharmaceutical Inc.'s (NASDAQ:RARE) CEO Compensation For The Time Being
Key Insights Ultragenyx Pharmaceutical's Annual General Meeting to take place on 15th of May Total pay for CEO Emil...Analysis Article • Apr 07
Ultragenyx Pharmaceutical (NASDAQ:RARE) Is Carrying A Fair Bit Of Debt
Warren Buffett famously said, 'Volatility is far from synonymous with risk.' When we think about how risky a company...
New Narrative • Mar 21
FDA Progress Will Accelerate Approval Of UX111 And UX143 In 2025
Global expansion and gene therapy product approvals are positioned to significantly enhance revenue streams and overall earnings.Seeking Alpha • Jan 29
Ultragenyx Pharmaceutical: Advancing On Multiple Fronts
Summary Ultragenyx Pharmaceutical Inc.'s stock has slightly declined since late 2023, even as the company is advancing its pipeline on multiple fronts. RARE has several FDA approved products on the market and should have several more approved in the coming years. While still losing money, Ultragenyx is seeing losses fall, has a solid balance sheet and almost universally optimism in the analyst firm community. An updated analysis of this mid-cap biotech firm follows in the paragraphs below. Read the full article on Seeking AlphaAnalysis Article • Dec 31
Does Ultragenyx Pharmaceutical (NASDAQ:RARE) Have A Healthy Balance Sheet?
The external fund manager backed by Berkshire Hathaway's Charlie Munger, Li Lu, makes no bones about it when he says...Seeking Alpha • Nov 12
Ultragenyx: Ready For More Growth After Q3 Earnings Beat
Summary Ultragenyx's strong Q3 earnings report, with a 42% YoY revenue increase, has reignited my interest in initiating a starter position in RARE. The company’s promising pipeline, including late-stage programs like Setrusumab and DTX401, presents multiple catalysts for future growth and higher valuation. Ultragenyx's $825M cash position and path to profitability by 2026 suggest financial stability, potentially avoiding the need for extensive dilutive financing. Despite significant risks in the rare disease market, I plan to build a position strategically in RARE, leveraging technical setups and catalyst-driven opportunities. Read the full article on Seeking AlphaSeeking Alpha • Nov 06
Ultragenyx: Excellent Company, Lackluster Stock
Summary Ultragenyx has a strong pipeline with four phase 3 programs and a high approval rate, but the stock remains stagnant and uninspiring. Crysvita is the top revenue generator with 2024 estimates of $375mn-$400mn, contributing to a total revenue guidance of $530mn-$570mn. The company plans to file for accelerated approval of UX111 in late 2024 or early 2025, with a PDUFA expected mid-2025. Despite its strengths, RARE remains unprofitable and stagnant, posing a significant risk, yet I am considering a position due to upcoming catalysts. Read the full article on Seeking AlphaAnalysis Article • Sep 04
We Think Ultragenyx Pharmaceutical (NASDAQ:RARE) Has A Fair Chunk Of Debt
David Iben put it well when he said, 'Volatility is not a risk we care about. What we care about is avoiding the...Seeking Alpha • Aug 22
Ultragenyx's Strong Pipeline And Valuation Concerns: A Balanced Perspective
Summary Ultragenyx develops therapies for rare genetic diseases, with a portfolio including Crysvita, Mepsevii, Dojolvi, and Evkeeza. The company’s promising pipeline targets severe diseases like osteogenesis imperfecta and Angelman syndrome, and it has progressed well in clinical trials. The company’s financials show a strong cash position but a high P/B and P/S ratios, as well as significant future royalty liabilities on Crysvita. Despite positive clinical trial results and revenue potential, RARE's expensive valuation and ongoing unprofitability raise concerns, warranting a neutral stance on the stock. Read the full article on Seeking AlphaAnalysis Article • Aug 03
There's Reason For Concern Over Ultragenyx Pharmaceutical Inc.'s (NASDAQ:RARE) Massive 26% Price Jump
Ultragenyx Pharmaceutical Inc. ( NASDAQ:RARE ) shareholders would be excited to see that the share price has had a...Shareholder Returns
| RARE | US Biotechs | US Market | |
|---|---|---|---|
| 7D | -5.5% | -1.7% | -2.7% |
| 1Y | -41.7% | 24.3% | 22.5% |
Return vs Industry: RARE underperformed the US Biotechs industry which returned 26.1% over the past year.
Return vs Market: RARE underperformed the US Market which returned 22.7% over the past year.
Price Volatility
| RARE volatility | |
|---|---|
| RARE Average Weekly Movement | 7.3% |
| Biotechs Industry Average Movement | 10.8% |
| Market Average Movement | 7.2% |
| 10% most volatile stocks in US Market | 16.6% |
| 10% least volatile stocks in US Market | 3.1% |
Stable Share Price: RARE has not had significant price volatility in the past 3 months compared to the US market.
Volatility Over Time: RARE's weekly volatility (7%) has been stable over the past year.
About the Company
| Founded | Employees | CEO | Website |
|---|---|---|---|
| 2010 | 1,371 | Emil Kakkis | 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.
Ultragenyx Pharmaceutical Inc. Fundamentals Summary
| RARE fundamental statistics | |
|---|---|
| Market cap | US$2.15b |
| Earnings (TTM) | -US$609.00m |
| Revenue (TTM) | US$670.00m |
3.2x
P/S Ratio-3.5x
P/E RatioIs RARE overvalued?
See Fair Value and valuation analysisEarnings & Revenue
| RARE income statement (TTM) | |
|---|---|
| Revenue | US$670.00m |
| Cost of Revenue | US$858.00m |
| Gross Profit | -US$188.00m |
| Other Expenses | US$421.00m |
| Earnings | -US$609.00m |
Last Reported Earnings
Mar 31, 2026
Next Earnings Date
n/a
| Earnings per share (EPS) | -6.20 |
| Gross Margin | -28.06% |
| Net Profit Margin | -90.90% |
| Debt/Equity Ratio | -524.0% |
How did RARE perform over the long term?
See historical performance and comparisonCompany Analysis and Financial Data Status
| Data | Last Updated (UTC time) |
|---|---|
| Company Analysis | 2026/06/08 05:59 |
| End of Day Share Price | 2026/06/08 00:00 |
| Earnings | 2026/03/31 |
| Annual Earnings | 2025/12/31 |
Data Sources
The data used in our company analysis is from S&P Global Market Intelligence LLC. The following data is used in our analysis model to generate this report. Data is normalised which can introduce a delay from the source being available.
| Package | Data | Timeframe | Example US Source * |
|---|---|---|---|
| Company Financials | 10 years |
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| Analyst Consensus Estimates | +3 years |
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| Market Prices | 30 years |
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| Ownership | 10 years |
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| Management | 10 years |
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| Key Developments | 10 years |
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* Example for US securities, for non-US equivalent regulatory forms and sources are used.
Unless specified all financial data is based on a yearly period but updated quarterly. This is known as Trailing Twelve Month (TTM) or Last Twelve Month (LTM) Data. Learn more.
Analysis Model and Snowflake
Details of the analysis model used to generate this report is available on our Github page, we also have guides on how to use our reports and tutorials on Youtube.
Learn about the world class team who designed and built the Simply Wall St analysis model.
Industry and Sector Metrics
Our industry and section metrics are calculated every 6 hours by Simply Wall St, details of our process are available on Github.
Analyst Sources
Ultragenyx Pharmaceutical Inc. is covered by 34 analysts. 17 of those analysts submitted the estimates of revenue or earnings used as inputs to our report. Analysts submissions are updated throughout the day.
| Analyst | Institution |
|---|---|
| Jack Allen | Baird |
| Huidong Wang | Barclays |
| Eliana Merle | Barclays |