Ultragenyx Pharmaceutical Inc. (RARE)

US — Healthcare Sector
Peers: MREO  BPMC  AGIO  DAWN  BMRN  PTCT  QURE 

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Key Metrics & Ratings

  • symbol RARE
  • Rev/Share 6.197
  • Book/Share 1.6076
  • PB 20.2178
  • Debt/Equity 5.836
  • CurrentRatio 2.4546
  • ROIC -0.4662

 

  • MktCap 2993783754.0
  • FreeCF/Share -4.3398
  • PFCF -7.0063
  • PE -5.7393
  • Debt/Assets 0.6759
  • DivYield 0
  • ROE -2.3748

 

  • Rating C-
  • Score 1
  • Recommendation Strong Sell
  • P/E Score 1
  • DCF Score 4
  • P/B Score 1
  • D/E Score 1

Recent Analyst Ratings

Type Ticker Analyst Firm Previous Rating Current Rating Previous Price Target Current Price Target Date
Resumed RARE H.C. Wainwright -- Buy -- $80 July 28, 2025
Initiation RARE William Blair -- Outperform -- -- May 28, 2025

News

Ultragenyx Pharmaceutical Inc. (RARE) Presents At Morgan Stanley 23rd Annual Global Healthcare Conference Transcript
RARE
Published: September 10, 2025 by: Seeking Alpha
Sentiment: Neutral

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE ) Morgan Stanley 23rd Annual Global Healthcare Conference September 9, 2025 4:05 PM EDT Company Participants Eric Crombez - Chief Medical Officer & Executive VP Conference Call Participants Maxwell Skor - Morgan Stanley, Research Division Presentation Maxwell Skor Equity Analyst Great. Thank you, everyone, for joining.

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image for news Ultragenyx Pharmaceutical Inc. (RARE) Presents At Morgan Stanley 23rd Annual Global Healthcare Conference Transcript
RARE Reports Positive Long-Term Data From Metabolic Disorder Study
RARE
Published: September 09, 2025 by: Zacks Investment Research
Sentiment: Positive

Ultragenyx posts 96-week data showing DTX401 sharply cuts cornstarch needs and improves life quality in GSDIa patients.

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image for news RARE Reports Positive Long-Term Data From Metabolic Disorder Study
Ultragenyx Pharmaceutical Inc. (RARE) Presents At Cantor Global Healthcare Conference 2025 Transcript
RARE
Published: September 04, 2025 by: Seeking Alpha
Sentiment: Neutral

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE ) Cantor Global Healthcare Conference 2025 September 4, 2025 10:55 AM EDT Company Participants Eric Crombez - Chief Medical Officer & Executive VP Howard Horn - Executive VP of Corporate Strategy & CFO Conference Call Participants Kristen Kluska - Cantor Fitzgerald & Co., Research Division Presentation Kristen Kluska Analyst Okay. Good morning, everybody.

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image for news Ultragenyx Pharmaceutical Inc. (RARE) Presents At Cantor Global Healthcare Conference 2025 Transcript
INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
RARE
Published: August 29, 2025 by: GlobeNewsWire
Sentiment: Neutral

NEW YORK, Aug. 29, 2025 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.

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image for news INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
RARE
Published: August 27, 2025 by: PRNewsWire
Sentiment: Neutral

NEW YORK , Aug. 27, 2025 /PRNewswire/ -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. ("Ultragenyx" or the "Company") (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext.

Read More
image for news INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
RARE
Published: August 25, 2025 by: GlobeNewsWire
Sentiment: Neutral

NEW YORK, Aug. 25, 2025 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.

Read More
image for news INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
RARE
Published: August 21, 2025 by: GlobeNewsWire
Sentiment: Neutral

NOVATO, Calif., Aug. 21, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 113,572 restricted stock units of the company's common stock to 33 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of August 16, 2025, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq …

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image for news Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
RARE
Published: August 21, 2025 by: GlobeNewsWire
Sentiment: Neutral

NEW YORK, Aug. 21, 2025 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.

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image for news INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
Ultragenyx Initiates Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)
RARE
Published: August 18, 2025 by: GlobeNewsWire
Sentiment: Neutral

Company expects to complete BLA submission in the fourth quarter of 2025 Company expects to complete BLA submission in the fourth quarter of 2025

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image for news Ultragenyx Initiates Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)
INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
RARE
Published: August 17, 2025 by: GlobeNewsWire
Sentiment: Neutral

NEW YORK, Aug. 17, 2025 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.

Read More
image for news INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
RARE
Published: August 13, 2025 by: GlobeNewsWire
Sentiment: Neutral

NEW YORK, Aug. 13, 2025 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.

Read More
image for news INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
Ultragenyx's Q2 Loss Narrower Than Expected, Revenues Rise Y/Y
RARE
Published: August 06, 2025 by: Zacks Investment Research
Sentiment: Negative

RARE posts a narrower Q2 loss and 13% revenue growth as demand rises across all four marketed rare disease drugs.

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image for news Ultragenyx's Q2 Loss Narrower Than Expected, Revenues Rise Y/Y
INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
RARE
Published: July 31, 2025 by: PRNewsWire
Sentiment: Neutral

NEW YORK , July 31, 2025 /PRNewswire/ -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. ("Ultragenyx" or the "Company") (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext.

Read More
image for news INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
Ultragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update
RARE
Published: July 30, 2025 by: GlobeNewsWire
Sentiment: Neutral

NOVATO, Calif., July 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Tuesday, August 5, 2025, to discuss its financial results and corporate update for the quarter ending June 30, 2025.

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image for news Ultragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update
Ultragenyx: July Selloff Creates Buying Opportunity Ahead Of UX143 Catalyst
RARE
Published: July 29, 2025 by: Seeking Alpha
Sentiment: Positive

Ultragenyx's 35% July selloff is overblown; both UX143 and UX111 programs remain on track with key catalysts ahead. UX143's Phase 3 trial continues for scientific rigor, not due to failure; pivotal data expected Q4 2025 could be transformative. UX111's FDA delay is manufacturing-related, not safety or efficacy; resubmission is planned, with approval possible in 2026.

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image for news Ultragenyx: July Selloff Creates Buying Opportunity Ahead Of UX143 Catalyst
INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
RARE
Published: July 28, 2025 by: GlobeNewsWire
Sentiment: Neutral

NEW YORK, July 28, 2025 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.

Read More
image for news INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
RARE
Published: July 23, 2025 by: GlobeNewsWire
Sentiment: Neutral

NEW YORK, July 23, 2025 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.

Read More
image for news INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
RARE
Published: July 22, 2025 by: PRNewsWire
Sentiment: Neutral

NEW YORK , July 22, 2025 /PRNewswire/ -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. ("Ultragenyx" or the "Company") (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext.

Read More
image for news INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
RARE Investors Have Opportunity to Join Ultragenyx Pharmaceutical Inc. Fraud Investigation with the Schall Law Firm
RARE
Published: July 18, 2025 by: Business Wire
Sentiment: Neutral

LOS ANGELES--(BUSINESS WIRE)---- $RARE--RARE Investors Have Opportunity to Join Ultragenyx Pharmaceutical Inc. Fraud Investigation with the Schall Law Firm.

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image for news RARE Investors Have Opportunity to Join Ultragenyx Pharmaceutical Inc. Fraud Investigation with the Schall Law Firm
INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
RARE
Published: July 18, 2025 by: GlobeNewsWire
Sentiment: Neutral

NEW YORK, July 18, 2025 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.

Read More
image for news INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
Ultragenyx Pharmaceuticals Gets CRL for UX111 Gene Therapy for MPS IIIA
RARE
Published: July 15, 2025 by: Zacks Investment Research
Sentiment: Positive

RARE faces regulatory setback as FDA issues a CRL for UX111, delaying approval of the MPS IIIA gene therapy until 2026.

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image for news Ultragenyx Pharmaceuticals Gets CRL for UX111 Gene Therapy for MPS IIIA
Ultragenyx shares plunge after bone disorder trial misses key interim goal
RARE
Published: July 10, 2025 by: Proactive Investors
Sentiment: Negative

Shares of Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) slumped around 26% on Thursday afternoon after the company and partner Mereo BioPharma said their late-stage study of setrusumab in osteogenesis imperfecta failed to meet a key interim goal. Ultragenyx is developing setrusumab as a potential first-in-class treatment for osteogenesis imperfecta, a rare genetic bone disorder with no approved therapies in the US.

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image for news Ultragenyx shares plunge after bone disorder trial misses key interim goal
RARE, MREO Fall as Osteogenesis Study Likely to Continue Till Year End
MREO, RARE
Published: July 10, 2025 by: Zacks Investment Research
Sentiment: Negative

Ultragenyx Pharmaceutical and Mereo BioPharma shares tumble as investors await key phase III UX143 osteogenesis data now set for release at year-end.

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image for news RARE, MREO Fall as Osteogenesis Study Likely to Continue Till Year End
Ultragenyx (RARE) Surges 5.1%: Is This an Indication of Further Gains?
RARE
Published: July 10, 2025 by: Zacks Investment Research
Sentiment: Positive

Ultragenyx (RARE) witnessed a jump in share price last session on above-average trading volume. The latest trend in earnings estimate revisions for the stock doesn't suggest further strength down the road.

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image for news Ultragenyx (RARE) Surges 5.1%: Is This an Indication of Further Gains?
RARE's GTX-102 Gets FDA's Breakthrough Status for Angelman Syndrome
RARE
Published: June 30, 2025 by: Zacks Investment Research
Sentiment: Positive

Ultragenyx Pharmaceuticals' GTX-102 earns FDA Breakthrough status after early data shows sustained progress in Angelman syndrome patients.

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image for news RARE's GTX-102 Gets FDA's Breakthrough Status for Angelman Syndrome
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
RARE
Published: June 20, 2025 by: GlobeNewsWire
Sentiment: Neutral

NOVATO, Calif., June 20, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 48,731 restricted stock units of the company's common stock to 26 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of June 16, 2025, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq …

Read More
image for news Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
Ultragenyx (RARE) Up 9.6% Since Last Earnings Report: Can It Continue?
RARE
Published: June 05, 2025 by: Zacks Investment Research
Sentiment: Positive

Ultragenyx (RARE) reported earnings 30 days ago. What's next for the stock?

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image for news Ultragenyx (RARE) Up 9.6% Since Last Earnings Report: Can It Continue?
Ultragenyx to Participate at Bank of America's 2025 Healthcare Conference
RARE
Published: May 07, 2025 by: GlobeNewsWire
Sentiment: Neutral

NOVATO, Calif., May 07, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Howard Horn, the company's chief financial officer and executive vice president, will participate in a fireside at Bank of America's 2025 Healthcare Conference on Tuesday, May 13, 2025, at 8:00 a.m. PT.

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image for news Ultragenyx to Participate at Bank of America's 2025 Healthcare Conference
Ultragenyx's Q1 Loss Wider Than Expected, Revenues Increase Y/Y
RARE
Published: May 07, 2025 by: Zacks Investment Research
Sentiment: Negative

RARE reports weaker-than-expected first-quarter results, as earnings and revenues miss estimates despite a year-over-year increase in product sales.

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image for news Ultragenyx's Q1 Loss Wider Than Expected, Revenues Increase Y/Y
Ultragenyx (RARE) Reports Q1 Earnings: What Key Metrics Have to Say
RARE
Published: May 06, 2025 by: Zacks Investment Research
Sentiment: Positive

The headline numbers for Ultragenyx (RARE) give insight into how the company performed in the quarter ended March 2025, but it may be worthwhile to compare some of its key metrics to Wall Street estimates and the year-ago actuals.

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image for news Ultragenyx (RARE) Reports Q1 Earnings: What Key Metrics Have to Say

About Ultragenyx Pharmaceutical Inc. (RARE)

  • IPO Date 2014-01-31
  • Website https://www.ultragenyx.com
  • Industry Biotechnology
  • CEO Emil D. Kakkis
  • Employees 1294

Ultragenyx Pharmaceutical Inc., 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, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.