RARE Ultragenyx Pharmaceutical Inc.

Ultragenyx shares are trading around $16 after gaining about 9% this week, rebounding from the $12.73 low hit earlier this month.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) just added another commercial product to its portfolio. The FDA on September 17 approved Fayuvi for the treatment of neurologic manifestations of Sanfilippo syndrome Type A in pediatric patients with preserved neurodevelopmental function. Fayuvi becomes the first approved treatment for this rare and fatal childhood disorder. Ultragenyx stock jumped more than […]

In September 2026, Ultragenyx Pharmaceutical received full U.S. FDA approval for FAYUVI (UX111), the first-ever treatment for pediatric patients with Sanfilippo syndrome Type A, along with a Priority Review Voucher. This approval not only addresses a previously untreated ultra-rare, fatal neurodegenerative disease but also expands Ultragenyx’s commercial gene therapy portfolio and manufacturing footprint in the U.S. We’ll now explore how FAYUVI’s first-in-disease approval and the associated...

Ultragenyx jumps 13% after FDA approval of Fayuvi, the first approved treatment for Sanfilippo syndrome Type A, backed by durable clinical data.

Wall Street raised Ultragenyx’s price target after the U.S. Food and Drug Administration approved Fayuvi to treat children with Sanfilippo syndrome Type A.

Ultragenyx (RARE) was a big mover last session on higher-than-average trading volume. The latest trend in earnings estimate revisions might help the stock continue moving higher in the near term.

Ultragenyx’s second gene therapy approval since August gives it another sellable voucher. Elsewhere, Roche declared success in a blood cancer trial and a cell therapy maker is cutting costs.

Abeona stands to collect mid-single-digit royalties on sales of Ultragenyx’s Fayuvi plus up to $30 million in commercial milestone payments as part of a licensing deal.

The Food and Drug Administration (FDA) granted full approval of Fayuvi, also known as UX111, for pediatric patients.

The biopharmaceutical company received FDA approval for a first-of-its-kind treatment for a rare neurodevelopmental disease.

Ultragenyx Pharmaceuticals won approval for its newest gene therapy on Thursday, helping shares partially reverse a catastrophic dive.

RARE investors are now awaiting an FDA decision on UX111, the company’s one-time gene therapy for Sanfilippo syndrome type A.

Large-cap healthcare outperformed this week: XLV gained 1.4% through Tuesday, while XBI fell 1.4% and IBB declined 0.5%.

In early September 2026, Ultragenyx Pharmaceutical announced that its Phase 3 Aspire trial of apazunersen (GTX-102) in Angelman syndrome failed to meet its primary and key secondary cognitive and functional endpoints, prompting a review of the program and planned significant expense reductions. On the same day, the FDA confirmed that Ultragenyx’s newly approved GENGLYCOS gene therapy for glycogen storage disease type Ia qualifies for a rare pediatric disease priority review voucher,...

Ultragenyx Pharmaceutical stock is under pressure after a multi year slide in returns, yet current market multiples still screen as expensive and the broader valuation checks send a mixed message. With fresh clinical setbacks alongside some positive data elsewhere in the pipeline, investors face conflicting signals about what the current price really reflects. Over the past 5 years the share price has fallen about 85%, which points to a sharp reset in market expectations for Ultragenyx...

Wall Street removed the Angelman program from valuation models, slashing price targets by as much as 76% and issuing multiple downgrades.

NIO, Ultragenyx Pharmaceutical and McDonald's slipped to annual lows on Thursday amid company-related pressures and Wall Street pessimism around the stocks.

Zanvastro is now approved for children and adults with Alexander disease, a rare and often fatal brain condition that affects fewer than one in a million people.

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Ultragenyx stock crashed Thursday, losing almost half its value, after its experimental Angelman treatment failed in final-phase testing.

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Ultragenyx Pharmaceuticals looks to cut costs following the trial failure, while attention remains squared on its burgeoning commercial business.

Ultragenyx (RARE) reported earnings 30 days ago. What's next for the stock? We take a look at earnings estimates for some clues.

Ultragenyx plunges 46% after its phase III Angelman syndrome study fails to achieve key efficacy goals, putting apazunersen's future in question.

Ultragenyx said the late-stage Aspire study of apazunersen, also known as GTX-102, did not meet its primary goal or key backup goal in a late-stage trial.

Ultragenyx Pharmaceutical (RARE) recently received accelerated approval from the U.S. Food and Drug Administration for GENGLYCOS, a gene therapy for glycogen storage disease type Ia. This milestone expands its portfolio of treatments for rare diseases. Ultragenyx Pharmaceutical’s share price has moved to US$25.81, with a 90-day share price return of 15.17% and a 30-day share price return of 3.61%. This suggests some momentum building around news such as the GENGLYCOS approval and the recent...

ALNY, BMRN, RARE and BBIO offer rare-disease growth potential through strong products, pipeline catalysts and high unmet medical needs.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) was up 7.5% in after-hours trading as of approximately 5:33 p.m. ET Wednesday after the FDA approved Genglycos for glycogen storage disease type Ia, or GSDIa. The one-time gene therapy is the first approved treatment designed to address the disorder’s underlying cause. It will carry a U.S. list price of approximately […]

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.