RARE Stock Slumps On Multiple Wall Street Price Target Slashes, Clinical Pipeline Uncertainty

Ultragenyx said on Thursday that the FDA has again refused approval for UX111 in the treatment of Sanfilippo syndrome type A.

• Ultragenyx on Thursday did not detail FDA’s concerns for the repeated rejection of UX111.
• TD Cowen lowered the firm's price target on Ultragenyx to $49 from $75 and maintained a ‘Buy’ rating on the shares.
• Goldman Sachs and Baird also slashed their price targets on the company on Friday.

Shares of Ultragenyx Pharmaceutical Inc. (RARE) slumped 9% on Friday after multiple Wall Street analysts slashed price targets on the company after it divulged new regulatory hurdles for its UX111 gene therapy.

The company submitted an application seeking approval for UX111 for Sanfilippo syndrome type A in January after it was denied approval in 2025.

However, on Thursday, the company said that the U.S. Food and Drug Administration (FDA) has again refused approval for the gene therapy and sought additional supportive documentation which the company expects to provide in a resubmission.

Regulatory Concerns Around UX111

The FDA first refused to approve the company’s application for the drug in July, and issued a letter requesting Ultragenyx to provide more information and improvements related to specific aspects of chemistry, manufacturing, and controls as well as observations from inspections of its manufacturing facility.

Ultragenyx on Thursday did not detail the FDA’s concerns for the repeated rejection. The company had said in January that it expects potential approval for the therapy by the third quarter of 2026, but that will likely be delayed owing to the fresh rejection.

Ultragenyx also said that it is continuing to evaluate the study data for UX143. In December, the company said that its late-stage studies of Setrusumab (UX143) in osteogenesis imperfecta did not meet the primary endpoint of reducing annualized fracture rates, though both trials showed improvements in bone mineral density. On Thursday, the company said that it is continuing to study the data, without providing more details.

Q4 Earnings And FY26 Outlook

Ultragenyx also announced fourth-quarter (Q4) revenue of $207 million on Thursday, driven by its products Crysvita and Dojolvi, and earnings per share of $1.29.

For FY26, the company expects total revenue in the range of $730 million to $760 million, an increase of 8% to 13% compared to 2025, excluding revenue from potential launches. This, however, trails an average analyst estimate of $794.83 million.

The company also announced a 10% reduction in workforce on Thursday, impacting about 130 employees, as part of its restructuring plan aimed at touch profitability in 2027.

Wall Street Consensus

TD Cowen lowered the firm's price target on Ultragenyx to $49 from $75 and maintained a ‘Buy’ rating on the shares. Given growing uncertainty about the FDA's stance on bio-marker based approvals for drugs treating rare diseases, the firm sees regulatory risk having increased for UX111 and it also notes that the company's 2026 revenue forecast fell short of consensus estimates.

Goldman Sachs, meanwhile, lowered the firm's price target on Ultragenyx to $61 from $71 and kept a ‘Buy’ rating on the shares.

Management is focused on cost reductions to maintain a path to 2027 profitability and upcoming clinical catalysts, including late-stage GTX-102 data in Angelman syndrome, as well as gene therapy programs UX701, DTX401, and UX111, which will shape the company's pipeline execution and strategic outlook, the firm said.

Baird, simultaneously, lowered the firm's price target on Ultragenyx to $40 from $47 and kept an ‘Outperform’ rating on the shares.

How Did Stocktwits Users React?

On Stocktwits, retail sentiment around RARE stock rose from ‘bearish’ to ‘bullish’ territory over the past 24 hours, while message volume increased from ‘low’ to ‘high’ levels.

A Stocktwits user dismissed the selloff as an “overreaction.”

Another highlighted the company’s hardships with Setrusumab and opined that the company will eventually terminate the program.

Yet another said that the market is punishing uncertainty around the company’s pipeline. “Profitability is contingent on the pipeline blossoming into commercialized products,” they said.

RARE stock has declined 51% over the past 12 months. 

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