BUZZ-Ultragenyx shares rise after FDA approves rare disease treatment drug

September 18 - ** Shares of pharmaceutical company Ultragenyx Pharmaceutical (RARE.O) rose 1% in premarket trading to $14.65 after the company closed up 12.58% on Thursday

** The U.S. Food and Drug Administration (FDA) on Thursday night (link) approved Ultragenyx's gene therapy Fayuvi as the first treatment for children with Sanfilippo syndrome type A

** Sanfilippo syndrome type A is a genetic disorder that gradually damages the brain and nervous system

** RARE set the U.S. recommended retail price for Fayuvi at $3.95 million. This one-time therapy for a rare and deadly childhood disease becomes one of the most expensive drugs in the world

** Abeona Therapeutics (ABEO.O) licensed Fayuvi to Ultragenyx in 2022, granting it global development and commercialization rights for the therapy

** ABEO shares rose 3.5% in premarket trading

** ABEO is eligible to receive certain commercial milestone payments and royalties tied to future product sales

** As of the previous trading session close, RARE shares had declined approximately 37% year-to-date

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