Ultragenyx Gets A ‘Much-Needed Win,’ Says Cantor – Sees $400M Opportunity After FDA Approves Neurodegenerative Disease Therapy

• Cantor Fitzgerald raised its price target on Ultragenyx to $39 from $33, maintaining an ‘Overweight’ rating, according to The Fly.
• Citi increased RARE’s target to $32 from $31, pointing to a potentially meaningful commercial opportunity.
• The Fayuvi approval also earned Ultragenyx a Priority Review Voucher, enabling quicker FDA review for a future drug application.

Ultragenyx Pharmaceutical (RARE) was in the spotlight on Friday after a slew of price-target hikes following the U.S. Food and Drug Administration (FDA) approval of Fayuvi to treat children with Sanfilippo syndrome Type A.

The approval comes just weeks after the company suffered a major setback in its Angelman syndrome program.

At the time of writing, RARE stock was up 1%, adding to its 12.6% rally in the previous session.

Priority Review Voucher: An Added Bonus

Cantor Fitzgerald raised its price target on Ultragenyx to $39 from $33, maintaining an ‘Overweight’ rating, and called the approval a “much-needed win,” according to TheFly. This implies a 166% upside potential from current levels.

The Fayuvi approval also earned Ultragenyx a Priority Review Voucher (PRV). A PRV enables a quicker FDA review for a future drug application or allows the biotech firm to sell the voucher to another company for cash.

Cantor estimates that this voucher, together with another PRV previously received by the company, could potentially generate a combined $400 million in non-dilutive capital if monetized.

Citi increased RARE’s target to $32 from $31 and kept a ‘Buy’ rating, calling Fayuvi’s label “favorable” and highlighting a potentially meaningful commercial opportunity. Canaccord also raised the target to $39 from $37 and maintained a ‘Buy’ rating.

ABEO Gets A Price Target Hike

Separately, Cantor also raised Abeona Therapeutics (ABEO) price target to $30 from $28, keeping an ‘Overweight’ rating. The firm said Fayuvi approval could benefit Abeona through royalties of up to 10% on sales and up to $30 million in commercial milestone payments.

ABEO shares climbed 3% in pre-market.

First Treatment For A Devastating Disease

Fayuvi, also known as UX111, is a one-time gene therapy for certain pediatric patients with Sanfilippo syndrome Type A, an ultra-rare and fatal genetic disease that causes progressive damage to the brain.

Fayuvi is Ultragenyx’s second approved gene therapy and sixth FDA-approved treatment overall. The company expects commercial supplies to begin shipping to qualified U.S. treatment centers within 30 to 60 days.

The FDA had rejected the application last year over manufacturing issues.

Earlier this month, Ultragenyx’s Phase 3 Aspire study of GTX-102 failed to meet its primary goal in Angelman syndrome, another rare neurogenetic disorder, prompting the company to discontinue the program. The stock crashed more than 44% after the news.

Retail Bull Eyes $50

Retail sentiment for RARE on Stocktwits turned ‘extremely bullish’ from ‘bullish’ over the past 24 hours, amid ‘extremely high’ message volumes. RARE was also among the top trending tickers at the time of writing.

One user expects the stock to hit $50.

RARE has been under heavy selling pressure so far in 2026, declining 38%.

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