Ultragenyx Pharmaceutical RARE has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $203 million. The company received the voucher after the FDA approved Fayuvi (rebisufligene etisparvovec-hopf), also known as UX111. Fayuvi is the first FDA-approved treatment for mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A.
The agreement marks Ultragenyx’s second PRV sale in one week. The company previously announced a $210 million sale of the voucher awarded following the approval of Genglycos (pariglasgene brecaparvovec-opnr), a gene therapy for glycogen storage disease type Ia. The Fayuvi transaction remains subject to customary and regulatory closing conditions.
Fayuvi Voucher Sale Adds Capital to RARE’s Growth Strategy
In September 2026, the FDA approved Fayuvi for the neurologic manifestations of MPS IIIA in pediatric patients with preserved neurodevelopmental function. Sanfilippo syndrome type A is a rare, inherited metabolic disorder. The approval gave eligible patients access to the first FDA-approved treatment for the condition and marked Ultragenyx’s sixth FDA approval overall and second gene therapy approval. The therapy addresses a disease area with substantial unmet medical need.
Beyond its potential clinical impact, the approval created a valuable financial asset for Ultragenyx. Under the FDA’s Rare Pediatric Disease PRV program, sponsors may receive a voucher after approval of an eligible treatment for a rare pediatric disease. A voucher can be redeemed to obtain priority review for a subsequent marketing application for a different product, or it can be sold or transferred. Selling the voucher allows a drugmaker to convert a regulatory milestone into funding without issuing additional shares.
Year to date, RARE shares have plunged 35.2% compared with the industry’s 0.3% decline.
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Per Ultragenyx, the $203 million transaction will deliver meaningful non-dilutive funding, supporting its efforts to develop treatments for rare and ultra-rare diseases and achieve profitability. The company also noted that Fayuvi itself benefited from capital generated through a previous PRV sale, underscoring how voucher monetization can help finance the development of therapies for conditions with limited treatment options. The company has not disclosed a closing date, and the proceeds depend on completion of the transaction.
The latest deal follows the Genglycos voucher agreement announced earlier in the week. Under the terms of that transaction, 20% of the $210 million proceeds, or $42 million, is payable to the National Institutes of Health under a prior patent license agreement, leaving approximately $168 million before other costs. This payment relates to the Genglycos voucher transaction. Ultragenyx has not announced a similar deduction for the Fayuvi sale.
Fayuvi and Genglycos expand Ultragenyx’s gene therapy presence within a broader rare-disease portfolio. Its marketed therapies also include Crysvita (burosumab-twza) for X-linked hypophosphatemia and tumor-induced osteomalacia, Mepsevii (vestronidase alfa-vjbk) for mucopolysaccharidosis type VII, and Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders. Ultragenyx also holds rights to develop, commercialize and distribute Evkeeza (evinacumab-dgnb) outside the United States, while Regeneron Pharmaceuticals commercializes the drug in the United States. Together, the two PRV transactions provide Ultragenyx with opportunities to monetize regulatory milestones and strengthen its financial resources as the company advances its rare-disease portfolio.
Ultragenyx Pharmaceutical Inc. Price and Consensus
Ultragenyx Pharmaceutical Inc. price-consensus-chart | Ultragenyx Pharmaceutical Inc. Quote
RARE’s Zacks Rank & Stocks to Consider
Ultragenyx currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Amarin AMRN and Corcept Therapeutics CORT, each currently sporting a Zacks Rank #1 (Strong Buy) and Aldeyra Therapeutics ALDX, carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, loss per share estimates for Amarin have narrowed from 65 cents to 39 cents for 2026. Over the same period, estimates for 2027 loss per share have narrowed from 51 cents to 50 cents. AMRN’s shares have lost 16.8% year to date.
Amarin’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 62.27%.
Over the past 60 days, earnings per share estimates for Corcept Therapeutics have increased from $1.13 to $1.86 for 2026. Over the same period, estimates for 2027 earnings per share have risen from $2.35 to $3.48. CORT’s shares have surged 234% year to date.
Corcept Therapeutics’ earnings missed estimates in two of the trailing four quarters, beat in one, while meeting in the remaining occasion, with the average surprise being 240.74%.
Over the past 60 days, loss per share estimates for Aldeyra Therapeutics have narrowed from 43 cents to 39 cents for 2026. Over the same period, estimates for 2027 loss per share have narrowed from 22 cents to 16 cents. ALDX’s shares have plunged 84.7% year to date.
Aldeyra Therapeutics’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 29.25%.
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