Ultragenyx to secure $413M through sale of two rare disease priority review vouchers

Ultragenyx has entered into agreements to sell two priority review vouchers (PRVs) for a combined total of approximately $413 million. The company secured these vouchers following recent FDA approvals for two gene therapies targeting ultra-rare diseases.

The first voucher, valued at $210 million, was awarded following the approval of Genglycos (pariglasgene brecaparvovec-opnr), a treatment for glycogen storage disease type Ia. A second voucher, valued at $203 million, follows the mid-September approval of Fayuvi, a therapy for Sanfilippo syndrome Type A.

The FDA’s Rare Pediatric Disease PRV program incentivizes the development of treatments for rare conditions by granting a voucher that can reduce the standard drug review time from 10 months to six. These vouchers can be used for subsequent applications or sold to other companies. Ultragenyx intends to use the resulting non-dilutive capital to bolster its drug pipeline and support its path to profitability.