The US Food and Drug Administration (FDA) has approved Ultragenyx Pharmaceutical's gene therapy Genglycos for glycogen storage disease type Ia (GSDIa), also known as Von Gierke disease. The therapy is approved for adults and children aged eight years and older.
GSDIa is a rare inherited disorder caused by a deficiency of the enzyme glucose-6-phosphatase, which is needed to release glucose from stored glycogen. The condition can cause dangerously low blood glucose levels.
Genglycos, also known as pariglasgene brecaparvovec, is designed to deliver a functional copy of the G6PC gene to liver cells. Genglycos is the first FDA-approved treatment for GSDIa and the first gene therapy approved by the FDA for the condition.
Reuters has more on this story.