Ultragenyx Pharmaceutical... (RARE)

Ultragenyx stock rises as Genglycos wins FDA's accelerated approval as the first treatment targeting the underlying cause of GSDIa, boosting its rare-disease portfolio.

Analysts raised their price targets on RARE stock after the FDA granted accelerated regulatory approval for its rare metabolic disease therapy.

The clearance of Genglycos for a rare glycogen storage disease gives Ultragenyx a sellable voucher and momentum ahead of an eagerly anticipated study readout.

Ultragenyx Pharmaceutical (NASDAQ:RARE) said the U.S. Food and Drug Administration has approved GENGLYCOS, also known as DTX401, for glycogen storage disease type 1A, or GSD1A. The approval marks the ...

The FDA granted accelerated approval to GENGLYCOS, also called DTX401, for patients aged eight and older with glycogen storage disease type Ia (GSDIa).