Ultragenyx’ Genglycos wins FDA accelerated approval
US rare diseases specialist Ultragenyx Pharmaceutical (Nasdaq: RARE) revealed that the US Food and Drug Administration (FDA) has granted accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and pediatric patients eight years and older with glycogen storage disease type Ia (GSDIa).
The company also received a Priority Review Voucher. The approval provides a long-awaited first-ever option to reduce the burden of care associated with GSDIa. Ultragenyx’s shares jumped 11.3% to $26.24 in after-hours trading on Wednesday following the announcement.
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