Ultragenyx shares rise after FDA approval for Genglycos
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.
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