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RARE Overview

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$
0.000
0.000(0.000%)
At close
0.000(0.000%)Aft-market
ET
$
0.000
0.000(0.000%)
At close
0.000(0.000%)Aft-market
ET
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Intellectia

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High
25.215
Open
25.170
VWAP
24.42
Vol
1.39M
Mkt Cap
2.37B
Low
23.950
Amount
33.88M
EV/EBITDA(TTM)
--
Total Shares
98.32M
EV
2.85B
EV/OCF(TTM)
--
P/S(TTM)
3.53
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultrarare genetic diseases. Its therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. Its four approved product candidates include Crysvita (burosumab) for the treatment of X-linked hypophosphatemia (XLH), and tumor-induced osteomalacia (TIO), Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis VII (MPSVII) or Sly Syndrome, Dojolvi (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). Its clinical product candidates include DTX401, DTX301, UX701, UX143, UX111, and GTX-102. UX143 for the treatment of Osteogenesis Imperfecta.
Show More

Events Timeline

(ET)
2026-04-02
09:10:00
Ultragenyx Submits UX111 Gene Therapy Application, FDA Review Set for September 19
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2026-03-30 (ET)
2026-03-30
09:20:00
Ultragenyx Receives FDA Clearance for UX016 IND
select
2026-03-24 (ET)
2026-03-24
16:20:00
Major Averages Close Lower as Oil Surges Above $90
select
2026-03-24
12:00:00
Dow Jones Up 0.31% to 46,350.65 Points
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2026-03-12 (ET)
2026-03-12
08:40:00
Ultragenyx Reports Positive Results from DTX301 Clinical Trial
select

News

CNBC
2.0
04-09CNBC
A Doctor's Journey with His Son's Rare Disease
  • Family Life Restructured: Dr. Joseph D'Orazio's family life was immediately altered upon learning of his son Gabe's diagnosis of Angelman syndrome, necessitating a complete reevaluation of daily routines and future plans to accommodate the realities of a special needs family.
  • Emotional and Professional Intertwining: As a physician, facing his son's rare disease brought profound grief and a sense of loss of control, which transformed his emotional landscape and deepened his empathy and understanding for patients' suffering in his medical career.
  • Patience and Growth: Gabe's condition taught Dr. D'Orazio invaluable lessons in patience and a reevaluation of life, enhancing his focus on emotional needs in medical practice and significantly improving his empathy as a physician.
  • Community and Advocacy: By engaging with the Foundation for Angelman Syndrome Therapeutics, Dr. D'Orazio transformed feelings of isolation into connection, aiding other families and advancing research, showcasing the resilience and unity of families affected by rare diseases.
Globenewswire
7.0
04-06Globenewswire
Ultragenyx Faces Class Action Lawsuit Over Stock Losses
  • Class Action Initiated: Bragar Eagel & Squire, P.C. has filed a class action lawsuit against Ultragenyx in the Northern District of California on behalf of investors who purchased shares between August 3, 2023, and December 26, 2025, indicating significant investor dissatisfaction with the company's future outlook.
  • False Statements Allegations: The complaint alleges that Ultragenyx made overly optimistic statements regarding the expected results of its Phase III Orbit and Cosmic studies for setrusumab, despite actual data showing the drug failed to significantly reduce fracture rates, leading investors to purchase shares at inflated prices and incur substantial losses.
  • Stock Price Plummet: Following the announcement on December 29, 2025, that the studies did not achieve primary endpoints, Ultragenyx's stock price fell from $34.19 to $19.72, a staggering 42.32% drop in a single day, reflecting a severe loss of market confidence in the company's prospects.
  • Investor Rights Protection: Investors who suffered losses during the class period can apply to be lead plaintiffs in the lawsuit by April 6, 2026, demonstrating that legal avenues are available for affected investors seeking potential remedies.
Globenewswire
7.0
04-06Globenewswire
Class Action Lawsuit Reminder Against Ultragenyx Pharmaceutical Inc.
  • Class Action Notice: The Schall Law Firm reminds investors of a class action lawsuit against Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) for violations of securities laws during the period from August 3, 2023, to December 26, 2025.
  • False Statements Exposed: The complaint alleges that Ultragenyx made false and misleading statements regarding its understanding of the effects of its drug candidate on patients with Osteogenesis Imperfecta, leading to an overly optimistic outlook among investors.
  • Study Results Impact: The Phase III ORBIT study revealed that Ultragenyx failed to achieve a statistically significant reduction in annualized fracture rate, highlighting significant errors in the company's drug efficacy claims and resulting in investor losses.
  • Legal Consultation Opportunity: Investors are encouraged to contact the Schall Law Firm before April 6, 2026, to discuss their rights and participate in the lawsuit to seek compensation for their losses.
Globenewswire
7.0
04-05Globenewswire
Ultragenyx Pharmaceutical Class Action Notice
  • Class Action Deadline: Investors who purchased Ultragenyx common stock between August 3, 2023, and December 26, 2025, must submit their applications by April 6, 2026, to participate in the class action, as failing to do so may result in losing their right to compensation.
  • Lawsuit Background: The lawsuit alleges that the company provided overly optimistic expectations regarding setrusumab's efficacy in Phase III Orbit and Cosmic studies, leading investors to purchase shares at inflated prices, which ultimately caused financial losses when the true results were revealed, highlighting the importance of accurate information disclosure.
  • Law Firm Credentials: Rosen Law Firm specializes in securities class actions and has recovered over $438 million for investors in 2019 alone, demonstrating its successful track record in similar cases, thus investors should carefully select their legal counsel.
  • Investor Action Steps: Investors can visit the Rosen Law Firm's website or call the toll-free number for more information on joining the class action, ensuring they receive appropriate legal representation as the case progresses.
Globenewswire
7.0
04-05Globenewswire
Class Action Lawsuit Filed Against Ultragenyx Pharmaceutical Inc.
  • Lawsuit Background: Bronstein, Gewirtz & Grossman, LLC has filed a class action lawsuit against Ultragenyx Pharmaceutical Inc., alleging violations of federal securities laws affecting all investors who purchased securities between August 3, 2023, and December 26, 2025.
  • False Statement Allegations: The complaint claims that throughout the class period, defendants made false and/or misleading statements and failed to disclose risks associated with their Phase III Orbit study, leading investors to have an inaccurate understanding of the drug setrusumab's efficacy.
  • Investor Action: Affected investors are encouraged to apply to be lead plaintiffs by April 6, 2026, to share in any potential recovery, with the assurance that they do not need to serve as lead plaintiffs to participate in any compensation.
  • Fee Arrangement: Bronstein, Gewirtz & Grossman, LLC represents investors on a contingency fee basis, meaning they will only seek reimbursement for expenses and attorney fees if they are successful, minimizing the financial burden on investors.
PRnewswire
7.0
04-04PRnewswire
Ultragenyx Pharmaceutical Class Action Reminder
  • Class Action Notice: Rosen Law Firm reminds investors who purchased Ultragenyx (NASDAQ:RARE) common stock between August 3, 2023, and December 26, 2025, to apply as lead plaintiffs by April 6, 2026, to participate in the class action and seek compensation.
  • Lawsuit Background: The lawsuit alleges that defendants provided false and misleading information regarding setrusumab (UX 143) during Phase III Orbit and Cosmic studies, leading investors to purchase shares at artificially inflated prices.
  • Choosing Legal Representation: Rosen Law Firm emphasizes the importance of selecting qualified counsel with a successful track record, noting that many firms issuing notices lack the necessary resources and experience to effectively litigate securities class actions.
  • Investor Losses: The lawsuit claims that Ultragenyx shareholders suffered losses due to the defendants' false statements, particularly after the true information entered the market, resulting in a significant decline in stock prices and substantial financial losses for investors.
Wall Street analysts forecast RARE stock price to rise
18 Analyst Rating
Wall Street analysts forecast RARE stock price to rise
17 Buy
1 Hold
0 Sell
Strong Buy
Current: 0.000
sliders
Low
35.00
Averages
61.65
High
120.00
Current: 0.000
sliders
Low
35.00
Averages
61.65
High
120.00
Morgan Stanley
NULL
to
Outperform
maintain
$50 -> $67
AI Analysis
2026-04-16
Reason
Morgan Stanley
Price Target
$50 -> $67
AI Analysis
2026-04-16
maintain
NULL
to
Outperform
Reason
Morgan Stanley raised the firm's price target on Ultragenyx to $67 from $50 and keeps an Outperform rating on the shares. The firm, which applies a new probability-of-success framework to Ultragenyx's GTX-102 Phase 3 Angelman readout expected in the second half of 2026, sees "a favorable asymmetric setup" with 50%-70% upside potentia. versus about 20% downside on failure, the analyst tells investors.
Goldman Sachs
Salveen Richter
Buy -> Neutral
downgrade
$61 -> $25
2026-03-24
Reason
Goldman Sachs
Salveen Richter
Price Target
$61 -> $25
2026-03-24
downgrade
Buy -> Neutral
Reason
Goldman Sachs analyst Salveen Richter downgraded Ultragenyx to Neutral from Buy with a price target of $25, down from $61. The firm cites the miss of the setrusumab Phase 3 in osteogenesis imperfecta for the downgrade. GTX-102 in Angelman syndrome is now the key value driver for Ultragenyx, the analyst tells investors in a research note. Goldman sees risk to the Phase 3 data in the second half of the year given the trial design and disease heterogeneity.
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Valuation Metrics

The current forward P/E ratio for Ultragenyx Pharmaceutical Inc (RARE.O) is 0.00, compared to its 5-year average forward P/E of -8.26. For a more detailed relative valuation and DCF analysis to assess Ultragenyx Pharmaceutical Inc's fair value, Click here.

Forward PE

The forward P/E ratio is a valuation metric that divides a company's current stock price by its estimated future earnings per share over the next 12 months.
StronglyUndervaluedUndervaluedFairOvervaluedStronglyOvervalueddotted line Image
N/A
5Y Average PE
-8.26
Current PE
0.00
Overvalued PE
-4.61
Undervalued PE
-11.90

Forward EV/EBITDA

The forward EV/EBITDA ratio is a valuation metric that divides a company's enterprise value (EV) by its estimated future earnings before interest, taxes, depreciation, and amortization (EBITDA) over the next 12 months.
StronglyUndervaluedUndervaluedFairOvervaluedStronglyOvervalueddotted line Image
N/A
5Y Average EV/EBITDA
-8.42
Current EV/EBITDA
-6.18
Overvalued EV/EBITDA
-4.82
Undervalued EV/EBITDA
-12.01

Forward PS

The forward P/S ratio is a valuation metric that divides a company's current stock price by its estimated future sales (or revenue) per share over the next 12 months.
StronglyUndervaluedUndervaluedFairOvervaluedStronglyOvervalueddotted line Image
5Y Average PS
8.40
Current PS
2.44
Overvalued PS
13.02
Undervalued PS
3.78

Financials

AI Analysis
Annual
Quarterly

Whales Holding RARE

S
Suvretta Capital Management, LLC
Holding
RARE
+12.38%
3M Return
S
Sofinnova Investment, Inc.
Holding
RARE
+3.70%
3M Return
B
Baker Bros. Advisors LP
Holding
RARE
+3.17%
3M Return
R
RTW Investments, LP
Holding
RARE
+1.39%
3M Return
R
Rock Springs Capital Management LP
Holding
RARE
-0.00%
3M Return

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Frequently Asked Questions

What is Ultragenyx Pharmaceutical Inc (RARE) stock price today?

The current price of RARE is 24.1 USD — it has decreased -3.83

What is Ultragenyx Pharmaceutical Inc (RARE)'s business?

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultrarare genetic diseases. Its therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. Its four approved product candidates include Crysvita (burosumab) for the treatment of X-linked hypophosphatemia (XLH), and tumor-induced osteomalacia (TIO), Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis VII (MPSVII) or Sly Syndrome, Dojolvi (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). Its clinical product candidates include DTX401, DTX301, UX701, UX143, UX111, and GTX-102. UX143 for the treatment of Osteogenesis Imperfecta.

What is the price predicton of RARE Stock?

Wall Street analysts forecast RARE stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for RARE is61.65 USD with a low forecast of 35.00 USD and a high forecast of 120.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.

What is Ultragenyx Pharmaceutical Inc (RARE)'s revenue for the last quarter?

Ultragenyx Pharmaceutical Inc revenue for the last quarter amounts to 207.28M USD, increased 25.72

What is Ultragenyx Pharmaceutical Inc (RARE)'s earnings per share (EPS) for the last quarter?

Ultragenyx Pharmaceutical Inc. EPS for the last quarter amounts to -1.29 USD, decreased -7.19

How many employees does Ultragenyx Pharmaceutical Inc (RARE). have?

Ultragenyx Pharmaceutical Inc (RARE) has 1371 emplpoyees as of April 22 2026.

What is Ultragenyx Pharmaceutical Inc (RARE) market cap?

Today RARE has the market capitalization of 2.37B USD.