Ultragenyx Pharmaceutical Inc (RARE)
NASDAQHealth CareBiotechnologySnapshot 2026-09-04
$15.30+3.03%
Close 2026-09-04 · 1-day change
NASDAQHealth CareBiotechnologySnapshot 2026-09-04
Intact: The reason to own it still holds.
Ultragenyx aims for Dojolvi sales between $100M and $110M in 2026. The company is cutting costs with a 10% workforce reduction. Analysts expect revenue to grow about 30% next year. New gene therapy approvals and partnerships could boost future sales.
Ultragenyx is still losing money and missed recent earnings. Legal and regulatory challenges could delay product launches. Revenue guidance for 2026 is much lower than 2025 actuals, signaling growth risks.
The stock price is about 43% below our valuation level and 45% above the Street median. Analysts expect nearly 30% revenue growth next year. Our view aligns with this growth but recognizes ongoing losses and execution risks.
Breaks if: Dojolvi revenue falls below $100M in FY26
Achieve full year 2026 Dojolvi revenue guidance in the range of $100 million to $110 million.
This is not a price target or investment advice.
A long-form read on the 1–3 year hold thesis. It updates when the weekly evidence changes.
This investment represents a turnaround story in the healthcare sector. RARE is currently loss-making and has a volatile management track record, but it is working towards achieving its revenue goals amidst a supportive sector backdrop.
The market seems to have priced in a low level of fragility, with RARE being seen as cheap compared to its peers. However, there is an expectations gap, indicating that the market may not fully believe in the company's ability to meet its revenue guidance.
Management is on track to meet its revenue guidance for 2026, with recent growth in total revenue and Dojolvi sales. However, the mixed results in controlling operating expenses and the overall risk of missing earnings could create volatility in the near term.
The thesis hinges on RARE's ability to maintain its revenue guidance and manage costs effectively. Additionally, the performance of sector bellwethers and broader economic indicators, like job growth, will be crucial in shaping investor sentiment.
The most important moves since the prior daily snapshot.
Signal changed from 'favorable' to 'mild_favorable'.
Yes, our read has weakened. A sharp drop in RARE's stock may indicate a repricing of the thesis. The FDA's review and potential approval of gene therapies UX111 and DTX401 still support the outlook. However, the recent significant decline raises concerns about the company's future prospects.
as of 2026-09-04
Review the evidence to watch, what would become a concern, and what would make it less concerning.
Not investment advice. Scores describe historical and current data; they are not forecasts of future returns. Consult a licensed advisor before making investment decisions.
Stated as a priority in 2 of last 2 quarters. Dojolvi revenue increased from $18 million in 2026-Q1 to $27 million in 2026-Q2. The company reaffirmed full year 2026 Dojolvi revenue guidance of $100 million to $110 million in both quarters. The trajectory is delivering toward the stated revenue guidance.
“The company reaffirms its full year 2026 Dojolvi revenue guidance of $100 million to $110 million.”
“The company reaffirms its full year 2026 Dojolvi revenue guidance of $100 million to $110 million.”
Breaks if: Revenue growth falls below 20% next year
Achieve full year 2026 Dojolvi revenue guidance in the range of $100 million to $110 million.
Stated as a priority in 2 of last 2 quarters. Dojolvi revenue increased from $18 million in 2026-Q1 to $27 million in 2026-Q2. The company reaffirmed full year 2026 Dojolvi revenue guidance of $100 million to $110 million in both quarters. The trajectory is delivering toward the stated revenue guidance.
“The company reaffirms its full year 2026 Dojolvi revenue guidance of $100 million to $110 million.”
“The company reaffirms its full year 2026 Dojolvi revenue guidance of $100 million to $110 million.”
Breaks if: Failure to implement or complete workforce reduction by end of FY26
Implement a strategic restructuring plan including a 10% workforce reduction to reduce expenses and improve cost structure.
Stated as a priority in 2 of last 2 quarters. The company implemented a 10% workforce reduction starting 2026-Q1 and reaffirmed guidance for flat to slightly down combined R&D and SG&A expenses in 2026 versus 2025. Operating expenses decreased from $305 million in 2026-Q1 to $289 million in 2026-Q2, showing limited progress on cost reduction.
“The company reaffirms its full year 2026 and 2027 guidance for combined R&D and SG&A expenses to be flat to slightly down versus 2025.”
“The company began implementation of a strategic restructuring plan including a 10% workforce reduction of approximately 130 employees.”
In the next 1 to 3 years, RARE's performance will depend on its execution against stated goals and external economic factors. Not investment advice.