Ultragenyx Pharmaceutical (RARE Quick QuoteRARE - Free Report) has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $210 million. The transaction is expected to provide the rare-disease drugmaker with a meaningful influx of non-dilutive cash, strengthening its financial position as it advances therapies for rare and ultra-rare diseases. The proceeds could also support Ultragenyx’s efforts to move toward profitability without requiring an equity financing. The deal remains subject to the satisfaction of certain regulatory and customary closing conditions.

The PRV was awarded to Ultragenyx following the FDA approval of Genglycos (pariglasgene brecaparvovec-opnr), also known as DTX401. Under the agency’s Rare Pediatric Disease PRV program, an eligible sponsor can receive a voucher after gaining approval for a qualifying rare pediatric disease therapy. The voucher can then be redeemed to obtain priority review for a future marketing application for a different eligible product. Importantly, the voucher can also be sold or transferred, allowing drugmakers to monetize the regulatory asset and generate capital.

Recent FDA Approval of Genglycos Earns RARE a Valuable PRV

In August, the FDA granted accelerated approval to Ultragenyx’s Genglycos to treat glycogen storage disease type Ia (GSDIa), making it the first treatment designed to address the underlying cause of the rare metabolic disorder. The therapy is indicated as an adjunct to nutritional management to reduce daily cornstarch intake in adults and pediatric patients aged eight years and older. The approval represents another important regulatory milestone for Ultragenyx as the company expands its portfolio of therapies targeting serious rare diseases.

Year to date, RARE shares have plunged 35.3% against the industry’s 1.3% growth.

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Ultragenyx’s Genglycos is an AAV8 gene therapy developed to target the underlying disease biology in GSDIa. By focusing on the root cause rather than solely managing symptoms through dietary measures, the therapy broadens treatment options for patients with a condition associated with significant unmet medical need. The approval marked Ultragenyx’s first gene therapy product and fifth FDA approval overall, strengthening its rare-disease franchise and expanding its presence in genetic and metabolic disorders.

The approval was strategically important because the resulting PRV gives Ultragenyx immediate financial flexibility beyond Genglycos’ commercial opportunity. The $210 million sale will provide non-dilutive capital to support research, development and commercialization. However, 20% ($42 million) of the proceeds is payable to the NIH under a prior patent license agreement, leaving about $168 million before other costs. Ultragenyx has previously benefited from PRV monetization to fund Genglycos’ development, highlighting the program’s role as a source of non-dilutive funding for rare-disease innovation.

Ultragenyx’s marketed portfolio spans several rare metabolic and genetic disorders. Crysvita (burosumab-twza) is approved for X-linked hypophosphatemia in adults and pediatric patients and for tumor-induced osteomalacia, while Mepsevii (vestronidase alfa-vjbk) treats mucopolysaccharidosis type VII. Dojolvi (triheptanoin) is indicated for long-chain fatty acid oxidation disorders, and Evkeeza (evinacumab-dgnb) is approved for homozygous familial hypercholesterolemia. Evkeeza is a partnered drug, with Ultragenyx holding rights to develop, commercialize and distribute it outside the United States, while Regeneron Pharmaceuticals commercializes it in the United States.

In September, the FDA also approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time gene therapy for the neurologic manifestations of mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome Type A, in pediatric patients with preserved neurodevelopmental function. The approval marked Ultragenyx’s sixth FDA approval overall and second gene therapy approval, further expanding its presence in rare and ultra-rare diseases.

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 Alnylam Pharmaceuticals (ALNY Quick QuoteALNY - Free Report) , currently sporting a Zacks Rank #1 (Strong Buy), and CRISPR Therapeutics (CRSP Quick QuoteCRSP - Free Report) and Aldeyra Therapeutics (ALDX Quick QuoteALDX - Free Report) , each 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, estimates for Alnylam’s 2026 earnings per share have decreased from $8.65 to $8.63. Over the same period, earnings estimates for 2027 have decreased from $12.13 to $12.04. ALNY shares have plunged 43.5% year to date.

Alnylam’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 27.58%.

Over the past 60 days, estimates for CRISPR Therapeutics’ 2026 loss per share have narrowed from $4.53 to $4.47. Over the same period, earnings estimates for 2027 have narrowed from $3.63 to $3.61. CRSP shares have lost 0.1% year to date.

CRISPR Therapeutics’ earnings beat estimates in two of the trailing four quarters and missed on the remaining two occasions, with the average negative surprise being 1.38%.

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 shares have plunged 81.5% year to date.

Aldeyra Therapeutics’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 29.25%.