Ultragenyx Pharmaceutical Inc. (RARE)

US — Healthcare Sector

Peers: DYN CNTA XENE APGE AAPG CELC CGON TVTX IDYA SLNO

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Key Metrics & Ratings

symbol RARE
Rev/Share 6.3204
Book/Share 0.162
PB 270.1527
Debt/Equity 94.2366
CurrentRatio 1.8917
ROIC -0.5861

MktCap 2392643711.0
FreeCF/Share -4.5242
PFCF -5.3006
PE -4.2673
Debt/Assets 0.725
DivYield 0
ROE -4.1417

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
Initiation	RARE	Wells Fargo	--	Overweight	--	$65	Oct. 20, 2025
Resumed	RARE	H.C. Wainwright	--	Buy	--	$80	July 28, 2025
Initiation	RARE	William Blair	--	Outperform	--	--	May 28, 2025

News

INVESTOR ALERT: Pomerantz Law Firm Investigates Claims on Behalf of Investors of Ultragenyx Pharmaceutical Inc. – RARE

RARE
Published: February 03, 2026 by: GlobeNewsWire
Sentiment: Neutral

NEW YORK, Feb. 03, 2026 (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.

image for news INVESTOR ALERT: Pomerantz Law Firm Investigates Claims on Behalf of Investors of Ultragenyx Pharmaceutical Inc. – RARE

Ultragenyx Pharmaceutical Resubmits UX111 BLA for Sanfilippo Syndrome

RARE
Published: February 02, 2026 by: Zacks Investment Research
Sentiment: Neutral

RARE resubmits FDA filing for UX111 after CRL, adding long-term neurologic and biomarker data, with a Q3 2026 decision in sight.

image for news Ultragenyx Pharmaceutical Resubmits UX111 BLA for Sanfilippo Syndrome

INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE

RARE
Published: January 27, 2026 by: GlobeNewsWire
Sentiment: Neutral

NEW YORK, Jan. 27, 2026 (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.

Ultragenyx Stock Declines After Missing Primary Endpoints on its Phase III Orbit and Cosmic Studies

RARE
Published: January 23, 2026 by: PRNewsWire
Sentiment: Neutral

NEW YORK, Jan. 23, 2026 /PRNewswire/ -- Levi & Korsinsky, LLP is investigating Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE) following the Company's announcement of topline results from its Phase III Orbit and Cosmic Studies, focusing on the effectiveness of setrusumab (UX143) on Osteogenesis Imperfecta. Investors with concerns about Ultragenyx's business operations may click here for more information.

image for news Ultragenyx Stock Declines After Missing Primary Endpoints on its Phase III Orbit and Cosmic Studies

INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE

RARE
Published: January 22, 2026 by: PRNewsWire
Sentiment: Neutral

NEW YORK, Jan. 22, 2026 /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.

Investigation Alert: Levi & Korsinsky Investigates Securities Fraud Claims Against Ultragenyx Pharmaceutical Inc. (RARE)

RARE
Published: January 14, 2026 by: Newsfile Corp
Sentiment: Neutral

New York, New York--(Newsfile Corp. - January 14, 2026) - Levi & Korsinsky notifies investors that it has commenced an investigation into Ultragenyx Pharmaceutical Inc. ("Ultragenyx Pharmaceutical Inc.") (NASDAQ: RARE) concerning potential violations of the federal securities laws. What Happened?

image for news Investigation Alert: Levi & Korsinsky Investigates Securities Fraud Claims Against Ultragenyx Pharmaceutical Inc. (RARE)

INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE

RARE
Published: January 08, 2026 by: PRNewsWire
Sentiment: Neutral

NEW YORK, Jan. 8, 2026 /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.

INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE

RARE
Published: January 06, 2026 by: GlobeNewsWire
Sentiment: Neutral

NEW YORK, Jan. 06, 2026 (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.

RARE Rises on Completion of Rolling Submission for AAV Gene Therapy

RARE
Published: December 31, 2025 by: Zacks Investment Research
Sentiment: Positive

Ultragenyx jumps after completing rolling BLA submission to the FDA for DTX401, an AAV gene therapy that could become the first approved treatment for GSDIa.

image for news RARE Rises on Completion of Rolling Submission for AAV Gene Therapy

Ultragenyx (RARE) Moves 15.5% Higher: Will This Strength Last?

RARE
Published: December 31, 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.

image for news Ultragenyx (RARE) Moves 15.5% Higher: Will This Strength Last?

Ultragenyx Pharmaceutical, Inc. Investigated by the Portnoy Law Firm

RARE
Published: December 30, 2025 by: GlobeNewsWire
Sentiment: Neutral

LOS ANGELES, Dec. 30, 2025 (GLOBE NEWSWIRE) -- The Portnoy Law Firm advises Ultragenyx Pharmaceutical, Inc. (“Ultragenyx" or the "Company") (NASDAQ: RARE) investors that the firm has initiated an investigation into possible securities fraud, and may file a class action on behalf of investors.

image for news Ultragenyx Pharmaceutical, Inc. Investigated by the Portnoy Law Firm

RARE, MREO Tank as Osteogenesis Study Fails to Meet Primary Endpoint

MREO, RARE
Published: December 30, 2025 by: Zacks Investment Research
Sentiment: Negative

Ultragenyx and Mereo BioPharma shares fall as phase III Orbit and Cosmic studies of setrusumab in osteogenesis imperfecta miss primary endpoints.

image for news RARE, MREO Tank as Osteogenesis Study Fails to Meet Primary Endpoint

Why Ultragenyx Pharmaceuticals Stock Is Plummeting Today

RARE
Published: December 29, 2025 by: The Motley Fool
Sentiment: Negative

Ultragenyx Pharmaceuticals reported Phase 3 results for setrusumab, a potential treatment for osteogenesis imperfecta. Several firms have reduced their price targets on Ultragenyx stock.

image for news Why Ultragenyx Pharmaceuticals Stock Is Plummeting Today

These biotech stocks are getting hammered by shocking brittle-bone treatment study results

MREO, RARE
Published: December 29, 2025 by: Market Watch
Sentiment: Negative

Shares of both Ultragenyx and Mereo BioPharma were seeing record selloffs after disappointing trial results for setrusumab.

image for news These biotech stocks are getting hammered by shocking brittle-bone treatment study results

Ultragenyx (RARE) Up 14.4% Since Last Earnings Report: Can It Continue?

RARE
Published: December 04, 2025 by: Zacks Investment Research
Sentiment: Positive

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

image for news Ultragenyx (RARE) Up 14.4% Since Last Earnings Report: Can It Continue?

Ultragenyx Pharmaceutical Inc. (RARE) Presents at Evercore 8th Annual Healthcare Conference Transcript

RARE
Published: December 03, 2025 by: Seeking Alpha
Sentiment: Neutral

Ultragenyx Pharmaceutical Inc. (RARE) Presents at Evercore 8th Annual Healthcare Conference Transcript

Ultragenyx Q3 Earnings and Sales Miss Estimates, Increase Y/Y

RARE
Published: November 05, 2025 by: Zacks Investment Research
Sentiment: Negative

RARE's Q3 loss widens and revenues miss estimates, despite year-over-year sales growth of key rare disease drugs.

image for news Ultragenyx Q3 Earnings and Sales Miss Estimates, Increase Y/Y

Will Ultragenyx (RARE) Report Negative Q3 Earnings? What You Should Know

RARE
Published: October 28, 2025 by: Zacks Investment Research
Sentiment: Negative

Ultragenyx (RARE) doesn't possess the right combination of the two key ingredients for a likely earnings beat in its upcoming report. Get prepared with the key expectations.

image for news Will Ultragenyx (RARE) Report Negative Q3 Earnings? What You Should Know

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

RARE
Published: October 24, 2025 by: GlobeNewsWire
Sentiment: Neutral

NOVATO, Calif., Oct. 24, 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 20,317 restricted stock units of the company's common stock to 17 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 October 16, 2025, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq …

image for news Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

Ultragenyx Pharmaceutical Inc. (RARE) Presents at Bank of America Global Healthcare Conference 2025 Transcript

RARE
Published: September 23, 2025 by: Seeking Alpha
Sentiment: Neutral

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE ) Bank of America Global Healthcare Conference 2025 September 23, 2025 6:35 AM EDT Company Participants Eric Crombez - Chief Medical Officer & Executive VP Conference Call Participants Tazeen Ahmad - BofA Securities, Research Division Presentation Tazeen Ahmad BofA Securities, Research Division Good morning to everybody listening in. Welcome to the Bank of America Healthcare Conference.

image for news Ultragenyx Pharmaceutical Inc. (RARE) Presents at Bank of America Global Healthcare Conference 2025 Transcript

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.

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.

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.

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.

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.

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.

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 …

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.

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

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.

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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.