Ultragenyx Pharmaceutical Inc（RARE）資訊與事件RAREUltragenyx Pharmaceutical Inc

Ultragenyx Sells Rare Pediatric Disease PRV for $203M

Ultragenyx announced that it has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher, or PRV, for $203M. Ultragenyx received the PRV upon FDA approval of Fayuvi, also known as UX111, the first-ever FDA-approved treatment for mucopolysaccharidosis type IIIA. The closing of the PRV transaction is subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott Rodino Antitrust Improvements Act.

Ultragenyx Sells Rare Pediatric Disease PRV for $210M

Ultragenyx announced that it has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher, or PRV, for $210M. Ultragenyx received the PRV upon FDA approval of Genglycos, also known as DTX401, the first treatment designed to address the underlying cause of glycogen storage disease type Ia. The closing of the PRV transaction is subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott Rodino Antitrust Improvements Act.

Ultragenyx's Fayuvi Receives Full FDA Approval

Ultragenyx announced that the U.S. FDA granted standard full approval of Fayuvi, also known as UX111, for the treatment of pediatric patients with mucopolysaccharidosis type IIIA. Fayuvi is the first-ever FDA-approved treatment for Sanfilippo syndrome Type A, a progressive and fatal neurodegenerative disease, and the second gene therapy approval for Ultragenyx. The company received a Priority Review Voucher upon this approval.