Ultragenyx Raises Significant Capital from Priority Review Voucher Sales

Ultragenyx received FDA approvals for two gene therapies in a month, and each approval came with a priority review voucher that can speed up future drug reviews. The therapies are Gengylcos, for glycogen storage disease type Ia, and Fayuvi, for Sanfilippo syndrome type A, and both are the first approved treatments for their ultra‑rare conditions.

The company announced the sale of each voucher, one for $203 million and another for $210 million. Buyers were not named, and the deals still need to meet closing conditions. The voucher program was created by Congress to encourage development of treatments for rare diseases; a voucher cuts the standard review time from 10 to 6 months, and recent prices have risen above $200 million as demand grows.

The company can use the money to fund other rare‑disease projects, especially after its antisense drug apazunersen for Angelman syndrome failed a phase‑3 trial and prompted restructuring and expense cuts.

Ultragenyx has received vouchers before, in 2017 and 2018. Chief Financial Officer Howard Horn said the Sanfilippo gene therapy benefited from capital generated by a previous voucher sale, showing how the program helps companies develop therapies for rare disease patients.

Analysts note that the voucher proceeds give Ultragenyx a stronger balance sheet and expect the company to deploy the funds strategically, aiming for profitability in 2027.