Ultragenyx Wins First FDA Nod for GSDIa Gene Therapy
Ultragenyx has secured the first FDA approval for its gene therapy targeting the rare metabolic disorder glycogen storage disease type Ia. Priced at 2.7 million dollars, the treatment aims to reduce patient reliance on daily cornstarch regimens.
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ULTRAGENYX PHARMACEUTICAL IN secured U.S. Food and Drug Administration approval for its gene therapy, sending shares up 7.5% in after-hours trading Wednesday afternoon. The treatment, named Genglycos, is the first authorized therapy for glycogen storage disease type Ia. The ultra-rare genetic disorder affects up to 2,500 patients in the United States, according to company estimates.
