RARE
NASDAQUltragenyx Pharmaceutical Inc.
News · 26 weeks66-57%
2026-04-192026-10-11
Mix3290d
- Other10(31%)
- SEC Filings7(22%)
- Analyst7(22%)
- Earnings4(13%)
- Insider4(13%)
Latest news
25 items- ANALYSTUBS initiated coverage on Ultragenyx Pharma with a new price targetUBS initiated coverage of Ultragenyx Pharma with a rating of Neutral and set a new price target of $16.00
- SECSEC Form 8-K filed by Ultragenyx Pharmaceutical Inc.8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
- PRUltragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $203 MillionNOVATO, Calif., Oct. 14, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that it has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $203 million. Ultragenyx received the PRV upon U.S. Food and Drug Administration (FDA) approval of FAYUVI™ (rebisufligene etisparvovec-hopf), also known as UX111, the first-ever FDA-approved treatment for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A). "The sale of this PRV delivers meaningful non-dilutive funding that strengthens our ability to provide innovative treatments for rare and ultra-rare diseases while supporting our objective of ac
- SECSEC Form 8-K filed by Ultragenyx Pharmaceutical Inc.8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
- PRUltragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $210 MillionNOVATO, Calif., Oct. 07, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that it has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $210 million. Ultragenyx received the PRV upon U.S. Food and Drug Administration (FDA) approval of GENGLYCOS™ (pariglasgene brecaparvovec-opnr), also known as DTX401, the first treatment designed to address the underlying cause of glycogen storage disease type Ia (GSDIa). "Monetizing this PRV provides significant non-dilutive capital to advance our efforts to bring forward first-ever therapies for rare and ultra-rare diseases, and supports our path to profitability,"
- PRUltragenyx Announces Marketing Authorisation Application (MAA) Submission to the European Medicines Agency (EMA) for the First Investigational Gene Therapy for MPS IIIA (Sanfilippo Syndrome Type A)BASEL, Switzerland, Oct. 02, 2026 (GLOBE NEWSWIRE) -- Today Ultragenyx announced that the European Medicines Agency (EMA) has validated the Company's Marketing Authorisation Application (MAA) for its investigational gene therapy, rebisufligene etisparvovec, for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome Type A. EMA validation confirms that the application is sufficiently complete for the Agency to begin its formal scientific review. Validation does not constitute approval of the application, nor does it represent a determination regarding the safety or efficacy of the investigational therapy, nor guarantee that the application will ultim
- SECUltragenyx Pharmaceutical Inc. filed SEC Form 8-K: Other Events8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
- PRUltragenyx 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 syndrome Type A Ultragenyx’s UltraCare® program will support access, and commercial product is expected to be available to ship to Qualified Treatment Centers within 30-60 days FAYUVI marks the second gene therapy approval, and sixth FDA approval overall, for Ultragenyx The Company received a Priority Review Voucher upon FAYUVI approval Ultragenyx to Host Conference Call on September 17, 2026 at 5:30 p.m. Eastern Time NOVATO, Calif., Sept. 17, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutic
- INSIDERSVP, Chief Accounting Officer Huizenga Theodore Alan sold $17,410 worth of shares (1,283 units at $13.57), decreasing direct ownership by 2% to 67,863 units (SEC Form 4)4 - Ultragenyx Pharmaceutical Inc. (0001515673) (Issuer)
- INSIDERSVP, Chief Accounting Officer Huizenga Theodore Alan sold $2,252 worth of shares (161 units at $13.99), decreasing direct ownership by 0.23% to 69,146 units (SEC Form 4) to cover taxes4 - Ultragenyx Pharmaceutical Inc. (0001515673) (Issuer)
- ANALYSTUltragenyx Pharma downgraded by BofA SecuritiesBofA Securities downgraded Ultragenyx Pharma from Buy to Neutral
- ANALYSTUltragenyx Pharma downgraded by William BlairWilliam Blair downgraded Ultragenyx Pharma from Outperform to Mkt Perform
- ANALYSTUltragenyx Pharma downgraded by Evercore ISI with a new price targetEvercore ISI downgraded Ultragenyx Pharma from Outperform to In-line and set a new price target of $16.00
- ANALYSTUltragenyx Pharma downgraded by Robert W. Baird with a new price targetRobert W. Baird downgraded Ultragenyx Pharma from Outperform to Neutral and set a new price target of $16.00
- ANALYSTUltragenyx Pharma downgraded by Analyst with a new price targetAnalyst downgraded Ultragenyx Pharma from Overweight to Neutral and set a new price target of $36.00
- ANALYSTUltragenyx Pharma downgraded by Wells Fargo with a new price targetWells Fargo downgraded Ultragenyx Pharma from Overweight to Equal Weight and set a new price target of $18.00
- SECUltragenyx Pharmaceutical Inc. filed SEC Form 8-K: Other Events8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
- PRUltragenyx Announces Phase 3 Aspire results in Angelman SyndromePhase 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) NOVATO, Calif., Sept. 02, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced results from the Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome. The study 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). The safety profile observed in Aspire was consistent with Phase 1/2. "Based on everything we observed in the robust
- PRUltragenyx 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 DiseaseAt 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 reduction of at least 50% Complete elimination of nighttime cornstarch dosing observed in 33% of DTX401 treatment group and 42% of crossover group, while maintaining glycemic control Analyses at Week 48 showed that 83% of DTX401-treated participants met or exceeded their own expectations for meaningful cornstarch reduction NOVATO, Calif., Sept. 01, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced the publication of 96-week data from its Phase 3 study of GENGLYC
- PRUltragenyx 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 rare and ultra-rare diseases, today reported the grant of 45,984 restricted stock units of the company’s common stock to 23 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, 2026, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4)
- SECUltragenyx Pharmaceutical Inc. filed SEC Form 8-K: Other Events8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
- PRUltragenyx 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 Ultragenyx received a Priority Review Voucher upon approval Ultragenyx to host conference call on 8/19/26 at 6:00 p.m. Eastern Time NOVATO, Calif., Aug. 19, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that the U.S. Food and Drug Administration (FDA) granted accelerated approval for GENGLYCOS™ (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and pediatric patients eight years and older with glycogen storage disease type Ia (GSDIa). "Th
- SECSEC Form SCHEDULE 13G filed by Ultragenyx Pharmaceutical Inc.SCHEDULE 13G - Ultragenyx Pharmaceutical Inc. (0001515673) (Subject)
- SECSEC Form SCHEDULE 13G filed by Ultragenyx Pharmaceutical Inc.SCHEDULE 13G - Ultragenyx Pharmaceutical Inc. (0001515673) (Subject)
- SECUltragenyx Pharmaceutical Inc. filed SEC Form 8-K: Other Events8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
RARE FAQ
7 questionsWhat does Ultragenyx Pharmaceutical Inc. do?
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 the United States. 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; and Dojolvi for treating long-chain fatty acid oxidation...Where does RARE stock trade?
Ultragenyx Pharmaceutical Inc. (RARE) is listed on NASDAQ.What sector and industry is RARE in?
Ultragenyx Pharmaceutical Inc. operates in the Health Care sector, Biotechnology: Pharmaceutical Preparations industry.When did Ultragenyx Pharmaceutical Inc. go public?
Ultragenyx Pharmaceutical Inc. (RARE) completed its IPO in 2014.What are analysts saying about RARE?
Ultragenyx Pharmaceutical Inc. has had 8 recent analyst actions on file. The most recent action was from UBS: Neutral with a $1600.00 price target on 2026-10-15. Recent price targets range from $1600.00 to $3600.00.What companies are similar to RARE?
Notable peers in the same industry include LLY (Eli Lilly and Company), JNJ (Johnson & Johnson), ABBV (AbbVie Inc.), MRK (Merck & Company Inc.), NVS (Novartis AG). Compare RARE side-by-side with any of them on Quantisnow.How can I track RARE on Quantisnow?
Quantisnow aggregates Ultragenyx Pharmaceutical Inc.'s SEC filings, analyst ratings, insider transactions, FDA approvals, and press releases the moment they hit the wire (Wall Street's wire, on your screen.). Follow RARE to receive live email and push alerts on every new disclosure.