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
QuarterlyIQ Insights · RARE
What must go right, what could break, what the price assumes, and what evidence comes next.
The current health of the standing investment case.
Recent financial performance recently climbed back into the top half of its industry — confirming the recovery.
One-year growth currently built into the price, compared with our model's estimate.
| Measure | Marker shape | Value |
|---|---|---|
| Growth built into the price | circle | -75.8% |
| Our one-year growth estimate | diamond |
For each item: what to watch, what would become a concern, and what would make it less concerning.
A reporting-risk estimate, not a forecast of the stock-price response.
Historical relationships that met the evidence threshold. They do not prove cause or forecast a move.
Usually moved in the same direction.
Price observations: 365 days
Most sensitive to the broad stock market.
Based on historical daily prices through 2026-09-04.
Past price behavior in dollars on a $10,000 position. This does not measure permanent business risk.
How much price usually moves either way.
Use the underlying financial and sector pages to investigate the cause.
Not investment advice. Scores describe historical and current data; they are not forecasts of future returns. Consult a licensed advisor before making investment decisions.
| 27.3% |
Growth built into the price is above our model estimate.
The price assumes 103.1 percentage points less one-year growth.
The one-year revenue growth assumption on Valuation.
Model as of 2026-09-04 · Compared with 398 industry peers · Company calendar date is not available
RARE — litigation filed
Dated 2026-08-11
Other Events. On August 7, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) and Baylor Research Institute (“BRI”) entered into a settlement agreement with Esjay Pharma Private Limited and Esjay Pharma LLC (collectively, “Esjay”) resolving patent litigation related to the Company’s DOJOLVI® (triheptanoin) oral liquid. The litigation, which was filed in the United States District Court for the District of New Jersey, resulted from submission by Esjay of an Abbreviated New Drug Application (…
Why it matters: The FDA's decision affects future growth and market potential for UX111.
Supportive ifFDA approval for UX111 gene therapy is granted.
Worry ifFDA denies approval for UX111 gene therapy.
Why it matters: Changes in sector growth can affect Ultragenyx. This can change how it competes.
Watch forHealthcare sector revenue growth speeds up above 10%. This helps Ultragenyx.
Also watch forHealthcare sector revenue growth drops below 5%. This shows more challenges ahead.
Why it matters: UX111's approval could improve the company's place in the gene therapy market.
Supportive ifThe FDA approves UX111 for the treatment of Sanfilippo syndrome type A.
Worry ifThe FDA denies approval or requires further studies, delaying the launch.
Why it matters: The data could show if the therapy works for Angelman syndrome. Good results may help the company's growth.
Supportive ifThe GTX-102 Phase 3 Aspire study shows good results in patients.
Worry ifThe study shows no good results or negative effects.
We watch for confirming and disproving signals on each item. Resolutions are found automatically where possible and checked by hand for unclear cases. Last 90 days shown.
A larger daily loss that occurred about once in every 20 trading days.
Deepest peak-to-trough drop in the last year.
| Measure | Value | Meaning |
|---|---|---|
| Typical day | ±$235 on $10,000 · ±2.4% | How much price usually moves either way. |
| Bad day | $441 loss on $10,000 · 4.4% | A larger daily loss that occurred about once in every 20 trading days. |
| Largest peak-to-trough drop in the past year | $5,936 loss on $10,000 · 59.4% | Deepest peak-to-trough drop in the last year. |
Past year. 1.00 means similar movement; 1.20 means about 20% more.
Past year. 1.00 means similar movement to the sector. This is secondary context.
Latest 30 trading days, shown as an annual percentage.
Latest 252 trading days, shown as an annual percentage.
20-trading-day average in US dollars.
Trading days used by the source risk snapshot.
Past results, not a forecast. Not investment advice.
Why it matters: The FDA's decision on DTX401 will impact the company's revenue and growth. Approval could lead to significant sales.
Supportive ifFDA approves the BLA for DTX401, allowing it to be marketed.
Worry ifFDA rejects the BLA or requests additional studies.
Why it matters: Revenue growth trends will show if Ultragenyx is improving or continuing to struggle.
Watch forQ2 revenue growth is over 10% compared to last year. This shows recovery.
Also watch forQ2 revenue growth is below 0%, showing continued decline.
Why it matters: The FDA's decision on UX111 will also affect revenue and growth. Approval could enhance the product portfolio.
Supportive ifFDA approves the BLA for UX111, allowing it to be marketed.
Worry ifFDA rejects the BLA or requests additional studies.
Why it matters: The 10% workforce cut aims to reduce costs. Success here affects profitability.
Supportive ifThe cost savings from reducing the workforce are more than we first thought.
Worry ifCost savings from workforce reduction are less than expected.
Why it matters: The FDA's choice could prove the gene therapy works and help revenue grow. Approval would help the company make money.
Supportive ifFDA approval of DTX401 for glycogen storage disease type Ia by the August 23, 2026 PDUFA date.
Worry ifFDA says no to DTX401 or wants more studies, which delays approval.
Why it matters: The FDA's choice could show if the gene therapy works for Sanfilippo syndrome. Approval would improve the company's product line.
Supportive ifFDA approval of UX111 by the September 19, 2026 PDUFA date.
Worry ifFDA says no to UX111 or wants more studies, which delays approval.
Why it matters: Achieving this revenue target would confirm the growth trajectory for Dojolvi. It is key for overall revenue guidance.
Supportive ifDojolvi revenue reaches or exceeds $100 million for the full year 2026.
Worry ifDojolvi revenue falls below $100 million for the full year 2026.