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FDA Approves First Gene Therapy for Rare Metabolic Disorder
20 Aug
Summary
- Ultragenyx's gene therapy, Genglycos, received FDA approval.
- It treats glycogen storage disease type Ia (GSDIa), also known as Von Gierke disease.
- This is the first therapy to address the underlying cause of the disorder.
The U.S. Food and Drug Administration has granted approval for Ultragenyx Pharmaceutical's innovative gene therapy, Genglycos. This significant development targets glycogen storage disease type Ia (GSDIa), a rare metabolic disorder also recognized as Von Gierke disease. GSDIa is characterized by the body's inability to release glucose from stored glycogen, leading to dangerously low blood sugar levels.
This newly approved therapy marks a pivotal moment as it is the first treatment to address the underlying genetic cause of GSDIa. The disorder affects an estimated 1 in 100,000 infants in the United States. While other experimental treatments are in development by companies like Moderna and Beam Therapeutics, they are still in the early stages of clinical testing.
