FDA approves Ultragenyx gene therapy for glycogen storage disease
The US Food and Drug Administration approved a gene therapy from Ultragenyx Pharmaceutical for treating glycogen storage disease type Ia, also known as Von Gierke disease, in adults and children. The drug, branded as Genglycos, is the first approved gene therapy for the rare metabolic disorder, which affects 1,500 to 2,500 patients in the US. The deficiency reduces the liver's ability to control glucose levels and is associated with potentially life-threatening low blood sugar levels. Genglycos will carry a US per-patient list price of $2.7 million. Separately, Regeneron Pharmaceuticals won regulatory approval for Pasatru, a medicine for fibrodysplasia ossificans progressiva. A Phase 3 trial showed the drug led to a 94 percent reduction in new lesions that form bone, while a high dose led to a 90 percent reduction.
Patients with glycogen storage disease type Ia now have access to the first gene therapy approved for their condition.
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