Ultragenyx recently received two separate FDA approvals for gene therapies in the span of a month, each regulatory nod accompanied by a voucher the company could use to speed up the future review of another rare disease drug. In the past week, Ultragenyx has reached separate deals to sell each regulatory fast pass.
The Novato, California-based biotech announced Wednesday a definitive agreement to sell a voucher for $203 million, following a deal last week to sell a voucher for $210 million. The identities of both voucher buyers were undisclosed and the purchases still need to meet closing conditions. But the deals put Ultragenyx in line for non-dilutive capital that the rare disease specialist can use to bolster its drug pipeline.
Congress created the voucher program to incentivize drug development in rare diseases. Companies awarded a regulatory approval in a rare pediatric disease can receive one of these vouchers, which cuts the standard review time of 10 months down to six. These vouchers may be sold, and many voucher recipients opt to sell them to big pharmaceutical companies. Demand for these regulatory fast passes has pushed prices higher; most of the vouchers sold this year fetched prices of $200 million or more.
Ultragenyx received a voucher alongside the late August approval of Gengylcos, a gene therapy for a rare inherited enzyme deficiency called glycogen storage disease type Ia. Another voucher came from the mid-September FDA nod for Fayuvi, a gene therapy for a different rare inherited enzyme deficiency called Sanfilippo syndrome Type A. Each product is the first approved therapy for its respective ultra-rare indication.
While the Ultragenyx gene therapies carry multi-million-dollar price tags, neither product is expected to become a big revenue driver. Much of the company’s future financial expectations had rested on apazunersen, which was in development for Angelman syndrome, a rare genetic neurological disorder with no FDA-approved therapies. Last month, Ultragenyx reported this antisense oligonucleotide drug failed its Phase 3 test. The company said the disappointing trial outcome would lead to a corporate restructuring and expense reductions.
The Angelman drug came from an acquisition. Ultragenyx’s two gene therapies also came from business deals. The capital from the voucher sales could be applied toward another deal for a different rare disease therapeutic candidate. While the company made clear its intent to sell both vouchers, it has not said how it will use the proceeds. As of the end of June, Ultragenyx reported its cash position was $436 million. The company is expected to report third quarter 2026 financial results in November.
Ultragenyx previously received vouchers for rare disease drug approvals in 2017 and 2018. In a prepared statement, Chief Financial Officer Howard Horn noted that the Sanfilippo gene therapy benefited from capital generated by a previous voucher sale, “demonstrating the important role the [priority review voucher] program plays in helping companies develop transformative therapies for rare disease patients.”
In a Wednesday research note, William Blair analyst Lachlan Hanbury-Brown said that even though Ultragenyx is launching two new gene therapies, the company’s pipeline lacks any clinical catalysts to drive value for the company’s stock in the next year. But the sale of the two vouchers significantly bolsters the biotech’s balance sheet with non-dilutive capital.
“We look forward to hearing how management plans to strategically deploy this additional capital, as well as details on the company’s expense reductions plans, which we believe will help achieve [generally accepted accounting principles] profitability in 2027,” Hanbury-Brown said.
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