Last Update 02 Feb 26

Fair value Increased 0.16%

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. Here is how bullish and bearish analysts are framing the story around growth, profitability and execution risk.

Bullish Takeaways

Bullish analysts point to the reaffirmed FY25 guidance and FY27 profitability timeline as key supports for their positive stance on execution and longer term margin improvement.
The beat in Crysvita Q3 revenues, along with Dojolvi performance in line with expectations, is viewed as evidence that the current commercial portfolio can support the revenue forecasts that underpin higher valuation targets.
The new Overweight initiation is interpreted as a sign that some analysts see the risk or reward balance as attractive at current levels, particularly with modeled revenue growth and profit margin assumptions built into their targets.
Some analysts frame Ultragenyx within a broader view that U.S. small to mid cap biotech names could act more like capital producers over time, which supports a more constructive stance on cash generation and balance sheet strength.

Bearish Takeaways

Price target trims to US$75, US$57 and US$50 signal that cautious analysts are tempering expectations for upside, even while keeping positive ratings in place.
Commentary around total revenues being light, despite the Crysvita Q3 beat, highlights concerns about how consistently Ultragenyx can meet or exceed aggregate revenue expectations.
While the sector level view on small to mid cap biotech is constructive, some analysts flag that execution risk around the transition to stronger profitability remains an important factor in their more conservative target assumptions.
The clustering of target cuts suggests that, for now, cautious analysts prefer to factor in a margin of safety around growth and profitability timelines, which can cap near term valuation upside in their models.

What's in the News

Resubmission of the Biologics License Application for UX111 AAV9 gene therapy for Sanfilippo syndrome type A to the FDA, including longer term neurologic and biomarker data and updated chemistry, manufacturing and controls responses, with a Priority Review already granted in February 2025 and a potential PDUFA date expected in the third quarter of 2026 if an action date is assigned and review proceeds as anticipated (Key Developments).
Completion of the rolling Biologics License Application submission for DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia, supported by data from 52 treated patients and up to six years of follow up, with Phase 3 results showing reductions in daily cornstarch intake, maintained hypoglycemia control, improved euglycemia and fasting tolerance, and patient reported quality of life improvements on the PGIC scale, alongside an acceptable safety profile (Key Developments).
Phase 3 Orbit and Cosmic studies of setrusumab (UX143) in Osteogenesis Imperfecta meeting primary endpoints on reduction in annualized clinical fracture rate versus placebo or bisphosphonates, and secondary endpoints on bone mineral density improvements, with no change reported in the safety profile (Key Developments).
Updated 2025 earnings view with total revenue for 2025 estimated at US$672 million to US$674 million, described as above the top end of the guidance range and characterized as approximately 20% growth versus 2024 (Key Developments).
Reaffirmed 2025 total revenue guidance of US$640 million to US$670 million, with management indicating an expected total revenue growth range of approximately 14% to 20% compared to 2024 (Key Developments).

Valuation Changes

Fair Value: The modelled fair value is essentially unchanged, moving from about US$63.90 to about US$64.00 per share.
Discount Rate: The discount rate in the model has edged lower from about 7.77% to about 7.67%.
Revenue Growth: Forecast revenue growth is broadly stable, shifting slightly from about 27.52% to about 27.58%.
Net Profit Margin: The assumed net profit margin has risen significantly from about 0.73% to about 32.24%.
Future P/E: The forward P/E input has been reduced sharply from a very large multiple to about 2,044x.

Expansion of the rare disease market and favorable regulatory environment are driving strong revenue growth and pricing power for Ultragenyx's therapies.
Advancing late-stage pipeline, successful product launches, and gene therapy innovation position the company for diversified growth and improved profitability.
Ongoing losses, regulatory setbacks, pricing pressures, industry competition, and reliance on key launches and vouchers threaten revenue growth, profitability, and financial stability.

Catalysts

About Ultragenyx Pharmaceutical

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.What are the underlying business or industry changes driving this perspective?

The accelerating global identification and diagnosis of rare diseases, supported by advancements in genomics and genetic testing, are expanding the addressable patient pool for Ultragenyx's existing and future therapies, which should drive sustained long-term revenue growth.
Healthcare payers and regulatory agencies are increasingly recognizing the value of orphan drugs, as evidenced by faster development timelines (FDA Breakthrough Therapy designation for GTX-102) and ongoing pricing/reimbursement wins in regions like Latin America and EMEA, supporting robust revenue streams and pricing power over the next several years.
Ultragenyx's clinical pipeline is advancing with five Phase III programs (including UX143 and GTX-102), multiple BLA submissions expected in the coming quarters, and near-term Phase III data readouts (notably for UX143 in OI by year-end and GTX-102 in Angelman syndrome in 2026) serving as upcoming value inflection points that can diversify and significantly accelerate the company's revenue base.
Commercial execution remains strong, with ongoing double-digit top-line growth (20% YoY revenue increase in 1H25) across a diversified, globally expanding portfolio, while new product launches (Evkeeza, continued Crysvita growth, and Dojolvi expansion) and new payer agreements in large international markets are expected to further scale revenues and improve operating leverage.
Ongoing investment and leadership in gene therapy and mRNA modalities positions Ultragenyx to benefit from process technology improvements that enable scalable, lower-cost manufacturing of complex biologics, offering the prospect of higher long-term margins and improving the company's path to profitability (targeted for GAAP break-even in 2027).

Ultragenyx Pharmaceutical Earnings and Revenue Growth

Ultragenyx Pharmaceutical Future Earnings and Revenue Growth

Assumptions

How have these above catalysts been quantified?

Analysts are assuming Ultragenyx Pharmaceutical's revenue will grow by 32.0% annually over the next 3 years.
Analysts assume that profit margins will increase from -87.3% today to 3.3% in 3 years time.
Analysts expect earnings to reach $46.9 million (and earnings per share of $0.4) by about September 2028, up from $-532.9 million today. However, there is a considerable amount of disagreement amongst the analysts with the most bullish expecting $227.4 million in earnings, and the most bearish expecting $-438 million.
In order for the above numbers to justify the analysts price target, the company would need to trade at a PE ratio of 243.6x on those 2028 earnings, up from -5.7x today. This future PE is greater than the current PE for the US Biotechs industry at 15.5x.
Analysts expect the number of shares outstanding to grow by 4.25% per year for the next 3 years.
To value all of this in today's terms, we will use a discount rate of 7.09%, as per the Simply Wall St company report.

Ultragenyx Pharmaceutical Future Earnings Per Share Growth

Risks

What could happen that would invalidate this narrative?

Persistent lack of profitability and a high cash burn rate-Ultragenyx reported a net loss of $115 million in Q2 2025 and expects net cash used in operations to increase in 2025, risking dilution or financial strain if revenue ramp-up and cost control do not materialize, which would negatively impact future earnings and margins.
Heavy reliance on regulatory success for pipeline assets (e.g., UX111 and DTX401)-recent delays due to a Complete Response Letter (CRL) from the FDA and CMC issues highlight vulnerability to further regulatory or operational setbacks, which could result in unpredictable or declining revenue streams.
Increasing pricing and reimbursement pressure-commercial performance relies on navigating country-by-country pricing negotiations and payer approval, with acknowledgments of "lumpiness" and risk of future tighter restrictions from governments or payors, potentially constraining long-term topline revenue growth.
Intensifying industry competition and technological disruption-rising competition in the rare disease and gene therapy space (including gene-editing and CRISPR technologies) alongside the risk that new modalities could outpace existing Ultragenyx treatments, posing threats to revenue sustainability and gross margin stability.
Dependence on Priority Review Vouchers (PRVs) and successful launches for achieving future profitability-company guidance assumes receipt and monetization of multiple PRVs, but timing and regulatory reauthorization risks around the PRV program or launch execution shortfalls could delay or reduce anticipated cash inflows, impacting liquidity and earnings targets.

Valuation

How have all the factors above been brought together to estimate a fair value?

The analysts have a consensus price target of $86.05 for Ultragenyx Pharmaceutical based on their expectations of its future earnings growth, profit margins and other risk factors. However, there is a degree of disagreement amongst analysts, with the most bullish reporting a price target of $128.0, and the most bearish reporting a price target of just $34.0.
In order for you to agree with the analyst's consensus, you'd need to believe that by 2028, revenues will be $1.4 billion, earnings will come to $46.9 million, and it would be trading on a PE ratio of 243.6x, assuming you use a discount rate of 7.1%.
Given the current share price of $31.55, the analyst price target of $86.05 is 63.3% higher.
We always encourage you to reach your own conclusions though. So sense check these analyst numbers against your own assumptions and expectations based on your understanding of the business and what you believe is probable.

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Disclaimer

AnalystConsensusTarget is a tool utilizing a Large Language Model (LLM) that ingests data on consensus price targets, forecasted revenue and earnings figures, as well as the transcripts of earnings calls to produce qualitative analysis. The narratives produced by AnalystConsensusTarget are general in nature and are based solely on analyst data and publicly-available material published by the respective companies. These scenarios are not indicative of the company's future performance and are exploratory in nature. Simply Wall St has no position in the company(s) mentioned. Simply Wall St may provide the securities issuer or related entities with website advertising services for a fee, on an arm's length basis. These relationships have no impact on the way we conduct our business, the content we host, or how our content is served to users. The price targets and estimates used are consensus data, and do not constitute a recommendation to buy or sell any stock, and they do not take account of your objectives, or your financial situation. Note that AnalystConsensusTarget's analysis may not factor in the latest price-sensitive company announcements or qualitative material.

Advancements In Genomics And Rare Therapies Will Expand Prospects