Ultragenyx Pharmaceutical has secured €203 million from the sale of one rare disease voucher and €210 million from the sale of another, which the company can use to bolster its drug pipeline. The vouchers were awarded by the FDA for the approval of two gene therapies, Gengylcos and Fayuvi, in late August and mid-September, respectively.
The vouchers can be used to speed up the review of another rare disease drug, cutting the standard review time from 10 months to six. The demand for these vouchers has driven prices higher, with most selling for $200 million or more this year.
Congress created the rare disease voucher program to incentivize drug development in rare diseases. Companies that receive a regulatory approval for a rare pediatric disease can receive a voucher, which can be sold to other companies. Ultragenyx received two vouchers, one for Gengylcos and one for Fayuvi, which are the first approved therapies for their respective indications.
The gene therapies, however, are not expected to be big revenue drivers due to their price tags in the millions. Ultragenyx’s future financial expectations had rested on apazunersen, a drug for Angelman syndrome, but it failed its Phase 3 test last month.
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The company has announced a corporate restructuring and expense reductions following the disappointing trial outcome. The capital from the voucher sales could be applied toward another deal for a different rare disease therapeutic candidate. As of the end of June, Ultragenyx reported a cash position of $436 million.
Ultragenyx previously received vouchers for rare disease drug approvals in 2017 and 2018. The company’s Chief Financial Officer, Howard Horn, noted that the Sanfilippo gene therapy benefited from capital generated by a previous voucher sale, demonstrating the importance of the voucher program in developing transformative therapies for rare disease patients.
William Blair analyst Lachlan Hanbury-Brown said that the sale of the two vouchers significantly bolsters Ultragenyx’s balance sheet with non-dilutive capital. He added that the company’s pipeline lacks clinical catalysts to drive value for the company’s stock in the next year, but looks forward to hearing how management plans to deploy the additional capital and details on the company’s expense reductions plans, which he believes will help achieve generally accepted accounting principles profitability in 2027.
