Ultragenyx Pharmaceutical Inc. (RARE)
| Market Cap | 1.42B -48.9% |
| Revenue (ttm) | 717.00M +17.5% |
| Net Income | -586.00M |
| EPS | -5.83 |
| Shares Out | 98.29M |
| PE Ratio | n/a |
| Forward PE | n/a |
| Dividend | n/a |
| Ex-Dividend Date | n/a |
| Volume | 4,939,905 |
| Open | 14.33 |
| Previous Close | 14.50 |
| Day's Range | 14.11 - 14.47 |
| 52-Week Range | 12.73 - 39.89 |
| Beta | 0.32 |
| Analysts | Buy |
| Price Target | 28.05 (+94.66%) |
| Est. Earnings | Nov 3, 2026 |
About RARE
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, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. 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... [Read more]
Financial Performance
In 2025, Ultragenyx Pharmaceutical's revenue was $673.00 million, an increase of 20.18% compared to the previous year's $560.00 million. Losses were -$575.00 million, 1.05% more than in 2024.
Financial StatementsAnalyst Summary
According to 20 analysts, the average rating for RARE stock is "Buy." The 12-month stock price target is $28.05, which is an increase of 94.66% from the latest price.
News
Ultragenyx Pharmaceutical Transcript: FDA announcement
FDA granted full approval to FAYUVI, the first gene therapy for Sanfilippo syndrome type A, based on robust long-term data. The therapy will be available in the U.S. within 30-60 days at a $3.95M price, with plans for global expansion and ongoing patient monitoring.
Ultragenyx Pharmaceutical Press release: FDA announcement
Ultragenyx Pharmaceutical issued a press release on September 17, 2026, disclosing material business information to investors.
Ultragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)
FAYUVI is a highly anticipated, first-ever treatment option with the potential to stop or slow the devastating, irreversible neurologic progression and loss of function associated with Sanfilippo synd...
US FDA approves Ultragenyx's gene therapy for rare disorder
The U.S Food and Drug Administration said on Thursday it has approved Ultragenyx's gene therapy for the treatment of a rare, fatal disease.
Ultragenyx Stock Sinks 47% on Drug Trial Failure. Why Wall Street Isn't Giving Up Yet.
Ultragenyx Pharmaceuticals looks to cut costs following the trial failure, while attention remains squared on its burgeoning commercial business.
Ultragenyx shares crater after Angelman syndrome drug fails late-stage trial
Shares of Ultragenyx Pharmaceutical plunged more than 40% in premarket on Thursday after its neurodevelopmental disorder treatment failed a closely watched late-stage trial, dealing a major blow to o...
Ultragenyx's rare disease drug fails in late-stage trial; shares plunge
Ultragenyx Pharmaceutical said on Wednesday its experimental drug for a rare genetic neurodevelopmental disorder did not meet the main goal in a late-stage trial, sending its shares down 40% in af...
Ultragenyx Announces Phase 3 Aspire results in Angelman Syndrome
Phase 3 Aspire did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI)
Ultragenyx Announces the Publication of a Successful 96-Week Randomized, Placebo-Controlled Trial with Crossover Treatment of GENGLYCOS™ (also known as DTX401) AAV Gene Therapy in GSDIa in The Journal of Inherited Metabolic Disease
At Week 96, participants across treatment and crossover groups experienced mean reduction in daily cornstarch intake of 61% while maintaining glycemic control, with most participants achieving reducti...
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
NOVATO, Calif., Aug. 25, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rar...
Ultragenyx's gene therapy for rare metabolic disorder secures US FDA nod
The U.S. Food and Drug Administration approved Ultragenyx Pharmaceutical's gene therapy to treat a rare metabolic disorder, the company said on Wednesday.
Ultragenyx Announces U.S. FDA Approval of GENGLYCOS™ Gene Therapy, the First-Ever FDA-Approved Treatment Designed to Treat the Underlying Cause of Glycogen Storage Disease Type Ia (GSDIa)
GENGLYCOS is the first gene therapy approval, and fifth FDA approval overall, for the company Approval provides a long-awaited first-ever option to reduce the burden of care associated with GSDIa Ultr...
Ultragenyx Pharmaceutical Transcript: Status update
GENGLYCOS received FDA approval as the first gene therapy for GSDIa, with rapid commercial launch planned and strong anticipated demand due to urgent unmet need. The company will collect confirmatory data, expand treatment centers, and pursue global commercialization, with net pricing expected near $2 million.
Ultragenyx Pharmaceutical Press release: Status update
Ultragenyx Pharmaceutical issued a press release on August 19, 2026, disclosing material business information to investors.
Ultragenyx Pharmaceutical Earnings Call Transcript: Q2 2026
Record Q2 revenue of $214M driven by strong global demand and commercial execution. Two gene therapies, DTX-401 and UX-111, are under FDA review with launches anticipated soon, while phase III data for GTX-102 in Angelman syndrome is expected in the fall. Cash position remains strong at $436M.
Ultragenyx Pharmaceutical Earnings release: Q2 2026
Ultragenyx Pharmaceutical released its Q2 2026 earnings on August 4, 2026, summarizing the period's financial results.
Ultragenyx Pharmaceutical Quarterly report: Q2 2026
Ultragenyx Pharmaceutical has published its Q2 2026 quarterly earnings report on August 4, 2026.
Ultragenyx Reports Second Quarter 2026 Financial Results and Corporate Update
Second quarter total revenue of $214 million, Crysvita® revenue of $156 million and Dojolvi® revenue of $27 million Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 m...
Ultragenyx to Host Conference Call for Second Quarter 2026 Financial Results and Corporate Update
NOVATO, Calif., July 28, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for seri...
Johnson Fistel, PLLP Investigates Claims on Behalf of Long-Term Shareholders of Ultragenyx Pharmaceutical Inc. (RARE)
SAN DIEGO, July 15, 2026 (GLOBE NEWSWIRE) -- Johnson Fistel, PLLP is investigating potential claims on behalf of current, long-term shareholders of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) agains...
Ultragenyx Releases 2025 Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global Impact
NOVATO, Calif., July 08, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2025 Impact Report, detailing the company's work on behalf of patients and families living ...
Ultragenyx Pharmaceutical Transcript: Goldman Sachs 47th Annual Global Healthcare Conference 2026
Key phase III Angelman data and two gene therapy launches are expected to drive a transformative year, with profitability targeted for 2027. Strong clinical results, robust manufacturing, and strategic PRV monetization support a solid financial outlook and pipeline expansion.
Ultragenyx to Participate at Goldman Sachs 47th Annual Global Healthcare Conference
NOVATO, Calif., June 03, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for ser...
Ultragenyx Pharmaceutical Transcript: AGM 2026
The meeting established a quorum, presented four key proposals, and saw all measures—including director elections, incentive plan approval, auditor ratification, and executive compensation—approved by shareholders. No questions were raised during the Q&A.
Ultragenyx Pharmaceutical Transcript: Bank of America Global Healthcare Conference 2026
Revenue guidance is reaffirmed for the year, with profitability targeted for 2027 through growth in current and new products. Key catalysts include upcoming phase III Angelman data and two gene therapy launches, both supported by strong clinical and commercial preparation.