Ultragenyx’s Gene-Therapy Win Could Unlock A ‘Long Revenue Tailwind,’ Says Wells Fargo — This Analyst Sees 265% Upside For RARE Stock

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

• Wells Fargo lifted the stock’s price target to $46 and kept an ‘Overweight’ rating, according to The Fly.
• Ultragenyx also received a priority review voucher from the FDA, which will allow it to request priority review for a different drug application in the future.
• Cantor Fitzgerald estimated that the priority review voucher could be worth more than $100 million to the company.

Ultragenyx Pharmaceutical (RARE) drew bullish Wall Street action on Thursday after the U.S. Food and Drug Administration approved Genglycos, its first gene therapy and fifth approved medicine overall.

RARE shares were trading 6% higher in pre-market.

What Did The FDA Approve?

On Wednesday, Genglycos received accelerated FDA approval for patients aged eight and older with glycogen storage disease type Ia (GSDIa), a rare metabolic disease. The genetic disorder prevents the liver from properly releasing stored sugar into the blood, creating a risk of severe or life-threatening low blood sugar. Patients currently depend on strict diets and raw cornstarch around the clock to maintain glucose levels.

The one-time therapy helps the liver produce the missing enzyme. Approval was based on a Phase 3 study in which treated patients required significantly less cornstarch than those receiving a placebo

Analysts See Revenue And Voucher Upside

Wells Fargo lifted the stock’s price target to $46 with an ‘Overweight’ rating, saying Genglycos could create a ‘long revenue tailwind,’ while also giving Ultragenyx a valuable priority review voucher, according to The Fly.  

The voucher will allow Ultragenyx to request priority review for a different drug application in the future, potentially shortening the FDA’s target review period.

Cantor Fitzgerald estimated that the priority review voucher could be worth more than $100 million to the company. The firm raised its price target to $103 from $96, implying about 265% upside from current levels, and maintained an ‘Overweight’ rating.

The firm called the approval an important step toward Ultragenyx’s goal of reaching profitability in 2027.

Earlier this month, Ultragenyx reaffirmed its 2026 revenue outlook of $730 million to $760 million and said it had $436 million in cash and securities. Its next major catalyst is the FDA’s September 19 decision on UX111 for Sanfilippo syndrome Type A, another rare genetic metabolic disorder.

Retail Remains ‘Extremely Bullish’

Retail sentiment surrounding RARE on Stocktwits remained ‘extremely bullish’ over the past 24 hours, amid a 457% surge in message volumes.

RARE shares have gained more than 12% so far in 2026.

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