RAREUltragenyx Pharmaceutical Inc.

Ultragenyx stock rises as Genglycos wins FDA's accelerated approval as the first treatment targeting the underlying cause of GSDIa, boosting its rare-disease portfolio.

Analysts raised their price targets on RARE stock after the FDA granted accelerated regulatory approval for its rare metabolic disease therapy.

The clearance of Genglycos for a rare glycogen storage disease gives Ultragenyx a sellable voucher and momentum ahead of an eagerly anticipated study readout.

Ultragenyx Pharmaceutical (NASDAQ:RARE) said the U.S. Food and Drug Administration has approved GENGLYCOS, also known as DTX401, for glycogen storage disease type 1A, or GSD1A. The approval marks the company’s first gene therapy approval and its fifth approved medicine. GENGLYCOS is indicated for a

The FDA granted accelerated approval to GENGLYCOS, also called DTX401, for patients aged eight and older with glycogen storage disease type Ia (GSDIa).

The FDA is due to rule by August 23 on Ultragenyx’s application for DTX401, a one-time gene therapy for von Gierke disease.

Record revenue of $214 million signals momentum ahead of gene therapy launches.

On August 4, 2026, Ultragenyx Pharmaceutical Inc. reported second-quarter revenue of US$214 million versus US$167 million a year earlier, while quarterly net loss narrowed to US$92 million from US$115 million and basic loss per share from continuing operations improved to US$0.90 from US$1.17. Despite a wider six-month net loss of US$277 million compared with US$266 million a year ago, the combination of higher first-half revenue of US$350 million versus US$306 million and flat basic loss...

Ultragenyx Pharmaceutical stock is coming off a difficult longer term stretch, with the share price down about 72.1% over five years. Current valuation checks send a mixed message on whether the present US$25.91 price still embeds too much optimism or has reset expectations enough. A roughly 72.1% share price decline over five years suggests long term holders have already absorbed a significant reset in expectations. The company is pointing to potential growth from upcoming gene therapy...

Ultragenyx Pharmaceutical (RARE) released its second quarter 2026 results on 4 August, reporting revenue of US$214 million and a net loss of US$92 million. Investors are now assessing what this means for the stock. See our latest analysis for Ultragenyx Pharmaceutical. Ultragenyx Pharmaceutical shares trade at US$25.91 after a 4.01% 7 day share price return, although the 30 day share price return is down 25.27%. Over the past year, total shareholder return has declined 5.92%, reflecting mixed...

RARE beats Q2 earnings estimates as revenues surge year over year to a record quarterly high, driven by higher product sales across its portfolio.

Moby summary of Ultragenyx Pharmaceutical Inc.'s Q2 2026 earnings call

Ultragenyx reports its highest quarterly revenue ever at $214 million, driven by strong product growth and advancing two potential first-ever gene therapies toward FDA decisions.

Ultragenyx Pharmaceutical (NASDAQ:RARE) reported second-quarter 2026 revenue of $214 million, led by growth across its commercial rare-disease portfolio, while the company prepared for potential regulatory decisions on two gene therapies and a late-stage data readout in Angelman syndrome. Chief Exe

While the top- and bottom-line numbers for Ultragenyx (RARE) give a sense of how the business performed in the quarter ended June 2026, it could be worth looking at how some of its key metrics compare to Wall Street estimates and year-ago values.

Ultragenyx (RARE) delivered earnings and revenue surprises of +29.13% and +18.23%, respectively, for the quarter ended June 2026. Do the numbers hold clues to what lies ahead for the stock?

Decisions are due by August 23 at the latest on therapies ranging from an mRNA flu vaccine to a gene therapy for a rare metabolic disorder, putting biotech investors on high alert.

Tarsus Pharmaceuticals (TARS) doesn't possess the right combination of the two key ingredients for a likely earnings beat in its upcoming report. Get prepared with the key expectations.

Ultragenyx (RARE) doesn't possess the right combination of the two key ingredients for a likely earnings beat in its upcoming report. Get prepared with the key expectations.