RARE
NASDAQ:RARE
Ultragenyx Pharmaceutical Inc.
- Stock
38.97
−0.03%
10.53
news - Aug 02, 2025 - 12:42
Ultragenyx Pharmaceutical Inc. (RARE) Gets FDA CRL for UX111, Vows Quick Resubmission
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) received a FDA Complete Response Letter (CRL) for its Biologics License Application for UX111, a gene therapy for Sanfilippo syndrome type A. The company plans to resolve chemistry, manufacturing, and controls (CMC) issues and resubmit the application within six months. It is also advancing UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome, with the latter receiving FDA Breakthrough Therapy designation. The company expanded Evkeeza’s use to infants with homozygous familial hypercholesterolemia (HoFH) after Health Canada approval.
insidermonkey.comnews - Aug 01, 2025 - 12:00
Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
Pomerantz LLP is investigating potential securities fraud claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), following a press release about clinical trial progress that caused the company's stock to drop 25.11% to $31.04 per share. The law firm, based in New York, Chicago, Los Angeles, London, and Paris, specializes in securities class actions and corporate litigation.
markets.financialcontent.comnews - Aug 01, 2025 - 07:38
Ultragenyx Pharmaceutical Inc. (RARE) Gets FDA CRL for UX111, Vows Quick Resubmission
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) received a FDA Complete Response Letter (CRL) for its gene therapy UX111 targeting Sanfilippo syndrome type A, with the company planning a six-month review and resubmission. The firm is advancing other therapies, including UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome, and has expanded Evkeeza's use to infants with homozygous familial hypercholesterolemia (HoFH) under Health Canada approval.
finance.yahoo.comnews - Jul 31, 2025 - 23:10
Ultragenyx Completes Enrollment for GTX-102 Phase 3 Study
Ultragenyx Pharmaceutical completed enrollment for its Phase 3 Aspire study of GTX-102, a treatment for Angelman Syndrome, with 129 participants. The study, which began in December 2024, is expected to complete in 2026, with plans for regulatory submission. The company's stock is under scrutiny due to financial performance and regulatory risks.
tipranks.comnews - Jul 31, 2025 - 22:05
Ultragenyx Completes Enrollment of Phase 3 Aspire Study
Ultragenyx Pharmaceutical Inc. announced the completion of enrollment for its Phase 3 Aspire study evaluating GTX-102 as a treatment for Angelman Syndrome, with 129 participants ages four to 17. The study, expected to complete in 2026, will assess cognitive and functional improvements in patients with the genetic disorder. The trial, randomized 1:1, will provide topline data for regulatory submission.
globenewswire.comDescription
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 treatme...Show More
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