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what it is
Ultragenyx sells drugs for ultra-rare genetic diseases, where small patient pools can still produce very real revenue.
how it gets paid
Last year Ultragenyx Pharma made $673M in revenue.
why it's growing
Revenue grew 20.2% last year. $466M mattered because it proved demand can spike.
what just happened
The quarter showed explosive top-line growth, with revenue hitting $466M, but the loss was still brutal.
At a glance
B balance sheet — gets the job done, barely
50/100 earnings predictability — expect surprises
-$5.84 fy2025 eps est
$2B fy2026 rev est
79.5% operating margin
xvary composite: 35/100 — weak
What they do
Ultragenyx sells drugs for ultra-rare genetic diseases, where small patient pools can still produce very real revenue.
Rare disease markets are tiny, which is exactly why they can be sticky. If your treatment targets a disease few companies touch, competition stays thin and pricing power lasts longer. Ultragenyx turned that setup into $673 million of 2025 revenue, up 20.2% vs. prior year, even while serving narrow patient groups.
How they make money
$673M
annual revenue · their business grew +20.2% last year
total revenue
$673M
+20.2%
The products that matter
treats x-linked hypophosphatemia
Crysvita
$673M · +20.2% growth
this is the commercial center of gravity. Crysvita generated $673M in 2025 revenue, which sits uncomfortably close to the entire company's $730–$760M 2026 revenue guide.
core revenue driver
enzyme replacement therapy
Mepsevii
approved product · no segment figure here
Mepsevii matters because it shows Ultragenyx can get therapies approved and sold. This snapshot does not break out revenue, which tells you Crysvita is still the number that mattered most.
commercial proof
clinical-stage rare disease assets
Pipeline assets
phase 3 readout on mar 12, 2026
the future sits here. Positive Phase 3 data on Mar 12, 2026 helped sentiment, but a 41% stock plunge on Dec 29, 2025 already showed you how fast this name reprices when the pipeline disappoints.
binary upside
Key numbers
$673M
2025 revenue
That is the hard proof the commercial business is real, not just a pipeline promise.
+20.2%
revenue growth
Sales are still rising fast, which matters because growth is the only thing investors tolerate when profits are missing.
79.5%
operating margin
Operating margin → what is left after running the business → so what: Ultragenyx still loses heavily on every dollar of sales.
$764M
long-term debt
Debt matters more when earnings are negative, because you cannot pay lenders with clinical optimism.
Financial health
B
strength
- balance sheet grade B — adequate — nothing special
- risk rank 4 — safer than 20% of stocks
- price stability 15 / 100
- long-term debt $764M (27% of capital)
B — functional but not a standout on the balance sheet.
Total return vs. market
Return history isn't available for RARE right now.
source: institutional data · return history unavailable
What just happened
missed estimates
The quarter showed explosive top-line growth, with revenue hitting $466M, but the loss was still brutal.
Latest-quarter revenue rose 191% vs. prior year to $466M, while EPS fell to -$4.55. Deadpan version: sales surged, and the company still lost a lot of money.
$168M
revenue
$4.55
eps
n/a
n/a
the number that mattered
$466M mattered because it proved demand can spike, but with EPS at -$4.55, scale still is not fixing the income statement.
source: company earnings report, 2026
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What could go wrong
Crysvita gives you a real revenue base. The problem is that RARE still loses about 85 cents for every $1 it brings in, so you are underwriting science, cost discipline, and time all at once.
med
the losses are not close to solved
An -85.44% profit margin and -608.47% return on equity tell you the commercial business is nowhere near covering the cost structure. This is not a rounding error and it is not something one good quarter fixes.
If margins stay anywhere near this negative, revenue growth by itself does not create much value for you. It just buys time.
med
pipeline results still set the mood
Positive Phase 3 data on Mar 12, 2026 helped. The 41% plunge on Dec 29, 2025 is the other half of the lesson. In biotech, one readout can rewrite the script faster than a year's worth of sales updates.
A failed or delayed program would hit future revenue hopes before the current income statement has stabilized.
med
Crysvita still carries the story
Crysvita generated $673M in 2025 revenue while full-company 2026 guidance is $730–$760M. That's a concentration story hiding inside a diversified-pipeline narrative.
If Crysvita slows, there is not much disclosed diversification here to cushion the hit.
med
the stock itself is part of the risk
A 15 / 100 price stability score and a $18–$42 52-week range tell you sentiment swings are part of the package. The business may change slowly. The stock does not.
If you own this name, you need room for ugly marks on the screen while the underlying science plays out.
the bear case is simple: losses stay deep, Crysvita stops carrying the weight, and the pipeline fails to arrive on time. That leaves you with a $764M debt load and a business the market values mainly on hope.
source: institutional data · regulatory filings · risk analysis
Pay attention to
the number that mattered
2026 revenue guidance
Management guided to $730–$760M. If that range moves higher while losses narrow from the recent -$1.29 quarterly EPS print, sentiment gets room to recover. If the range slips, the market will treat recent optimism as rented, not owned.
next catalyst
Q1 2026 earnings
The next report is due around May 2026. Watch the estimated -$1.48 EPS, but here's the thing: guidance will probably matter more than the quarter itself.
commercial trend
Crysvita growth durability
Crysvita grew 20.2% to $673M. You want to see whether that pace holds, because one product is still doing most of the current revenue work.
headline risk
class action timeline
The Apr 6, 2026 deadline does not change intrinsic value by itself. It does keep trust on the table in a stock that already has a 15 / 100 price stability score.
Analyst rankings
earnings predictability
50 / 100
in human-speak, analysts think the quarterly numbers swing around enough to surprise you.
risk rank
4
this is safer than roughly 20% of stocks. Put plainly: it is riskier than most of the market.
beta
1.2
beta measures how much the stock moves versus the market. When the market moves 10%, RARE has historically moved about 12%.
source: institutional data
Institutional activity
institutional ownership data for RARE is being compiled.
source: institutional data
Price targets
3-5 year target range
n/a
n/a
$21
current price
n/a
target midpoint · n/a from current
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