Ultragenyx rises after securing FDA nod for rare metabolic disorder gene therapy
BUZZ-Ultragenyx rises after securing FDA nod for rare metabolic disorder gene therapy ** Shares of drugmaker Ultragenyx Pharmaceutical RARE.O rise 11.4% to $29.23 premarket
** RARE late on Wednesday said it has secured US FDA approval for its gene therapy Genglycos to treat an ultra-rare metabolic disorder known as glycogen storage disease type Ia (GSDIa), also known as Von Gierke disease, in adults and pediatric patients
** The disease reduces the liver's ability to control glucose levels and is associated with potentially life-threatening low blood sugar levels
** Day-to-day management of the condition requires a relentless regimen of raw cornstarch as an oral glucose replacement therapy
** Genglycos will have a U.S. per-patient list price of $2.7 million and be available through qualified treatment centers within 30 to 60 days, the company said
** As of last close, RARE stock up 14% YTD
(Reporting by Padmanabhan Ananthan)
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