News

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 ...
FDA rejects Ultragenyx gene therapy over manufacturing concerns
While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...
William Blair Maintains a Buy on Ultragenyx Pharmaceutical (RARE)
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the best oversold NASDAQ stocks to buy now. On July 14, William Blair ...
US FDA declines to approve Ultragenyx's gene therapy for rare inherited disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
BioSpace2d
Ultragenyx Takes Second Hit as FDA Rejects Gene Therapy for Sanfilippo Syndrome
The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...
Fierce Pharma2d
Ultragenyx takes another hit as FDA snubs AAV gene therapy over manufacturing issues
The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...
FDA Delays Approval For Ultragenyx's Gene Therapy, But Not A Dealbreaker, Says Analyst
Ultragenyx's UX111 gene therapy faces FDA delay due to facility issues; analysts adjust price targets amid uncertain ...
STAT2d
FDA declines to approve Ultragenyx gene therapy
In his first 100 days as commissioner of the Food and Drug Administration, Marty Makary has made a handful of splashy ...
The Pharma Letter2d
Ultragenyx hit with FDA delay for gene therapy targeting rare childhood disorder
Ultragenyx Pharmaceutical (Nasdaq: RARE) has been dealt a regulatory setback, as the American medicines regulator has ...
BioSpace11h
FDA Layoffs, 200 Rejection Letters, User Fees and Priority Vouchers, Bad Week for Rare Disease
Around 3,500 FDA employees received termination emails; FDA Commissioner Marty Makary suggests lowering industry user fees ...
Ultragenyx Pharmaceutical, Apple And Other Big Stocks Moving Lower In Monday's Pre-Market Session
Ultragenyx received a Complete Response Letter from the FDA for UX111 AAV gene therapy to treat Sanfilippo Syndrome Type A ...
Ultragenyx: The High Stakes Of Binary Events And A Short Cash Runway
Ultragenyx's high burn rate, costly R&D, and recent setbacks increase dilution risk, impacting future profitability. Learn ...