Ultragenyx Pharmaceutical (NASDAQ:RARE) Trading Up 11.5% – Here’s Why

Posted by Francis Steltz on Sep 19th, 2026

Shares of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE – Get Free Report) were up 11.5% on Thursday . The stock traded as high as $14.24 and last traded at $14.3560. Approximately 4,669,826 shares changed hands during mid-day trading, an increase of 83% from the average daily volume of 2,545,771 shares. The stock had previously closed at $12.88.

Ultragenyx Pharmaceutical News Summary

Here are the key news stories impacting Ultragenyx Pharmaceutical this week:

Positive Sentiment: The FDA granted full approval to FAYUVI for pediatric patients with Sanfilippo syndrome Type A, also known as MPS IIIA. It is the first approved treatment for the fatal neurodegenerative disease, creating a new commercial opportunity for Ultragenyx where no prior therapy existed. Clinical data showed durable benefit, including a 23.5-point cognitive advantage versus natural history in the pivotal analysis. Ultragenyx Announces Approval of FAYUVI Gene Therapy
Positive Sentiment: FAYUVI carries a reported $3.95 million list price, potentially making it a significant revenue contributor despite the disease’s small patient population. Ultragenyx expects shipments to qualified treatment centers within 30–60 days and will provide insurance and treatment-access support through its UltraCare program. Ultragenyx Sets $3.95m List Price for Fatal Rare Disease Therapy
Positive Sentiment: The approval is Ultragenyx’s second gene-therapy approval and sixth FDA approval overall. The company also received a Priority Review Voucher, which could accelerate development or potentially have financial value. US FDA Approves Ultragenyx’s Gene Therapy
Positive Sentiment: Analysts raised targets after the approval: Cantor Fitzgerald and Canaccord Genuity moved to $39, Citi to $32, and Morgan Stanley to $20. Citi and Canaccord maintained bullish ratings, while Morgan Stanley remained equal weight. Ultragenyx Stock Jumps on Fayuvi Approval and Analyst Target
Neutral Sentiment: The commercial opportunity is promising but limited by the ultra-rare population. Successful sales will depend on identifying eligible children, establishing treatment centers, securing insurer reimbursement, and managing the complexity of one-time gene-therapy administration.
Negative Sentiment: FAYUVI’s label includes risks such as liver toxicity, low platelet counts, infusion reactions, thrombotic microangiopathy, and potential malignancy. Long-term safety and real-world adoption remain important uncertainties.
Negative Sentiment: A Pomerantz law-firm investigation adds an overhang, while the earlier failed Angelman syndrome trial remains a reminder of Ultragenyx’s clinical-development risk. Pomerantz Investor Alert

Analyst Ratings Changes

Several equities analysts recently weighed in on RARE shares. William Blair lowered shares of Ultragenyx Pharmaceutical from an “outperform” rating to a “market perform” rating in a research report on Thursday, September 3rd. The Goldman Sachs Group lowered their price target on shares of Ultragenyx Pharmaceutical from $28.00 to $19.00 and set a “neutral” rating on the stock in a report on Friday, September 4th. TD Cowen decreased their target price on Ultragenyx Pharmaceutical from $49.00 to $18.00 and set a “buy” rating for the company in a research report on Thursday, September 3rd. Canaccord Genuity Group increased their price target on Ultragenyx Pharmaceutical from $37.00 to $39.00 and gave the stock a “buy” rating in a research note on Friday. Finally, Robert W. Baird lowered shares of Ultragenyx Pharmaceutical from an “outperform” rating to a “neutral” rating and cut their target price for the stock from $40.00 to $16.00 in a research report on Thursday, September 3rd. One analyst has rated the stock with a Strong Buy rating, nine have given a Buy rating, ten have issued a Hold rating and one has issued a Sell rating to the company’s stock. According to data from MarketBeat.com, Ultragenyx Pharmaceutical has an average rating of “Hold” and a consensus price target of $34.42.

Read Our Latest Research Report on Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical Trading Up 0.1%

The firm has a market cap of $1.43 billion, a P/E ratio of -2.48 and a beta of 0.31. The stock’s fifty day moving average is $23.88 and its two-hundred day moving average is $24.42.

Ultragenyx Pharmaceutical (NASDAQ:RARE – Get Free Report) last announced its quarterly earnings results on Tuesday, August 4th. The biopharmaceutical company reported ($0.90) EPS for the quarter, beating the consensus estimate of ($1.22) by $0.32. The firm had revenue of $214.00 million during the quarter, compared to the consensus estimate of $183.08 million. Ultragenyx Pharmaceutical had a negative net margin of 81.79% and a negative return on equity of 1,024.42%. The business’s quarterly revenue was up 28.5% compared to the same quarter last year. During the same period last year, the firm earned ($1.17) EPS. On average, analysts forecast that Ultragenyx Pharmaceutical Inc. will post -3.98 EPS for the current year.

Hedge Funds Weigh In On Ultragenyx Pharmaceutical

A number of institutional investors have recently added to or reduced their stakes in the business. Integrated Wealth Concepts LLC bought a new position in shares of Ultragenyx Pharmaceutical in the second quarter worth approximately $311,000. IFP Advisors Inc lifted its position in Ultragenyx Pharmaceutical by 191.7% during the second quarter. IFP Advisors Inc now owns 2,313 shares of the biopharmaceutical company’s stock valued at $77,000 after purchasing an additional 1,520 shares during the last quarter. Allworth Financial LP grew its stake in Ultragenyx Pharmaceutical by 130.9% in the 2nd quarter. Allworth Financial LP now owns 4,699 shares of the biopharmaceutical company’s stock worth $157,000 after buying an additional 2,664 shares in the last quarter. Nykredit A S purchased a new position in Ultragenyx Pharmaceutical in the 2nd quarter worth approximately $154,000. Finally, Corient Private Wealth LP increased its holdings in shares of Ultragenyx Pharmaceutical by 7.3% in the 2nd quarter. Corient Private Wealth LP now owns 146,493 shares of the biopharmaceutical company’s stock worth $4,891,000 after buying an additional 9,949 shares during the last quarter. 97.67% of the stock is currently owned by institutional investors.

About Ultragenyx Pharmaceutical

(Get Free Report)

Ultragenyx Pharmaceutical Inc is a biopharmaceutical company focused on developing and commercializing treatments for rare and ultra-rare diseases. The company’s work includes therapies for genetic, metabolic and skeletal disorders, with an emphasis on conditions that have limited or no effective treatment options.

Ultragenyx’s marketed products include Crysvita (burosumab), used to treat certain forms of X-linked hypophosphatemia and tumor-induced osteomalacia; Dojolvi (triheptanoin), a medical food for long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab), a treatment for homozygous familial hypercholesterolemia.