Ultragenyx has entered a definitive agreement to sell a rare pediatric disease priority review voucher for $210 million, the company announced October 7. Ultragenyx received the voucher upon FDA approval of Genglycos (pariglasgene brecaparvovec-opnr, also known as DTX401), the first treatment designed to address the underlying cause of glycogen storage disease type Ia, which won accelerated approval on August 19. The closing is subject to customary conditions including expiration of the Hart-Scott-Rodino waiting period; the buyer was not named. Jefferies is serving as exclusive financial advisor.
Monetizing the voucher provides significant non-dilutive capital and supports the company’s path to profitability, chief financial officer Howard Horn said. The sale is an incrementally positive development that will significantly strengthen the balance sheet, William Blair analyst Lachlan Hanbury-Brown wrote in a client note. It comes a month after Ultragenyx said it would consider cost cuts following the failure of a key experimental treatment for Angelman syndrome.
Ultragenyx received a second voucher upon the approval of the Sanfilippo gene therapy Fayuvi, leaving room for a repeat transaction. Priority review vouchers, which let the buyer secure a six-month FDA review instead of the standard ten months, have become a standard financing lever for rare-disease developers: they convert regulatory success in one program into capital for the rest of the pipeline, and Genglycos itself benefited from an earlier voucher sale during its development.