US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat a rare genetic disorder, the company said on Friday.
Ultragenyx shares plunge after bone disorder trial misses key interim goal
Shares of Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) slumped around 26% on Thursday afternoon after the company and partner Mereo BioPharma said their late-stage study of setrusumab in osteogenesis imperfecta failed to meet a key interim goal. Ultragenyx is developing setrusumab as a potential first-in-class treatment for osteogenesis imperfecta, a rare genetic bone disorder with no approved therapies in the US.
RARE, MREO Fall as Osteogenesis Study Likely to Continue Till Year End
Ultragenyx Pharmaceutical and Mereo BioPharma shares tumble as investors await key phase III UX143 osteogenesis data now set for release at year-end.
Ultragenyx (RARE) Surges 5.1%: Is This an Indication of Further Gains?
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.
RARE's GTX-102 Gets FDA's Breakthrough Status for Angelman Syndrome
Ultragenyx Pharmaceuticals' GTX-102 earns FDA Breakthrough status after early data shows sustained progress in Angelman syndrome patients.
Ultragenyx (RARE) Up 9.6% Since Last Earnings Report: Can It Continue?
Ultragenyx (RARE) reported earnings 30 days ago. What's next for the stock?
Ultragenyx's Q1 Loss Wider Than Expected, Revenues Increase Y/Y
RARE reports weaker-than-expected first-quarter results, as earnings and revenues miss estimates despite a year-over-year increase in product sales.
Ultragenyx (RARE) Reports Q1 Earnings: What Key Metrics Have to Say
The headline numbers for Ultragenyx (RARE) give insight into how the company performed in the quarter ended March 2025, but it may be worthwhile to compare some of its key metrics to Wall Street estimates and the year-ago actuals.
Ultragenyx (RARE) Reports Q1 Loss, Misses Revenue Estimates
Ultragenyx (RARE) came out with a quarterly loss of $1.57 per share versus the Zacks Consensus Estimate of a loss of $1.54. This compares to loss of $2.03 per share a year ago.
Ultragenyx (RARE) Expected to Beat Earnings Estimates: What to Know Ahead of Q1 Release
Ultragenyx (RARE) possesses the right combination of the two key ingredients for a likely earnings beat in its upcoming report. Get prepared with the key expectations.
Ultragenyx: Commercial Engine Fuels Rare Disease Pipeline
Ultragenyx shows strong revenue growth and disciplined expense management, making it a compelling long-term investment despite inherent biotechnology risks. The company's diverse revenue streams from Crysvita, Dojolvi, and Evkeeza fund innovation and buffer against financial volatility. Key catalysts include the FDA's Priority Review of UX111 for Sanfilippo syndrome type A, with a PDUFA date set for August 2025.
Ultragenyx (RARE) Soars 9.2%: Is Further Upside Left in the Stock?
Ultragenyx (RARE) saw its shares surge in the last session with trading volume being higher than average. The latest trend in earnings estimate revisions may not translate into further price increase in the near term.
