Ultragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update 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. The live and replayed webcast of the call will be available thro...
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) NOVATO, Calif., July 18, 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 45,292 restricted stock units of the company’s common stock to 29 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...
Health Canada Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH) First and only medicine approved by Health Canada for patients as young as 6-months of age with HoFH, an ultrarare, inherited form of high cholesterol TORONTO, July 14, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultra-rare genetic diseases, today announced that Health Canada has extended the approval of ...
Santé Canada étend l’approbation d’Evkeeza® (évinacumab) chez les enfants âgés d’à peine 6 mois atteints d’hypercholestérolémie familiale homozygote (HFHo) Premier et seul médicament approuvé par Santé Canada chez les patients âgés d’à peine 6 mois atteints d’HFHo, une forme héréditaire très rare d’hypercholestérolémie TORONTO, 14 juill. 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ : RARE), une société biopharmaceutique spécialisée dans la mise au point et la commercialisation de nouveaux traitements contre des maladies génétiques rares et très rares, a annoncé aujour...
Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are resolvable FDA clinical reviews acknowledged that the clinical data are robust and biomarker data are supportive NOVATO, Calif., July 11, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its Biologics License...
Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for Osteogenesis Imperfecta Progressing to Final Analysis Data from Orbit and Cosmic studies expected around the end of the year NOVATO, Calif., July 09, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and Mereo BioPharma Group plc (NASDAQ: MREO), today announced that the randomized, placebo-controlled Phase 3 portion of the Orbit study evaluating UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is progressing toward a final analysis consistent with the orig...
Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for GTX-102 (apazunersen) as a treatment for Angelman syndrome. "FDA Breakthrough Therapy Designation underscores...
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) 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...
Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference NOVATO, Calif., June 03, 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 Emil Kakkis, M.D., Ph.D., the company's chief executive officer, will participate in a fireside at the Goldman Sachs 46th Annual Global Healthcare Conference on Tuesday, June 10, 2025, at 10:00 a.m. ET. The live and archived webcast of the panel ...
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