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Getting the latest healthcare news for you
Getting the latest healthcare news for you
The FDA has approved Ultragenyx's Genglycos, the first gene therapy for glycogen storage disease type Ia (GSDIa), a rare metabolic disorder affecting 1,500–2,500 Americans. The one-time treatment targets the root cause of the disease, reducing patients' dependence on a grueling daily cornstarch regimen. It comes with a $2.7 million per-patient list price.
The FDA has given the green light to Ultragenyx's Genglycos — making it the first-ever gene therapy approved for glycogen storage disease type Ia (GSDIa), also known as Von Gierke disease. The ultra-rare genetic condition impairs the liver's ability to release glucose into the bloodstream, leading to potentially life-threatening low blood sugar. Until now, patients have had to manage the disease through a relentless daily regimen of raw cornstarch as an oral glucose substitute.
Genglycos works by addressing the underlying genetic defect, restoring the normal breakdown of glycogen to produce glucose — reducing or eliminating the need for cornstarch. The FDA's approval was based on a 48-week late-stage trial showing reduced cornstarch requirements versus placebo, though the agency noted this is a surrogate endpoint and has required Ultragenyx to complete additional trials to confirm long-term effectiveness.
By the Numbers:
Why it matters: For a patient population with no previously approved gene therapy options, Genglycos represents a landmark shift from daily disease management to a potential one-time treatment. However, its steep price tag raises important questions about access and affordability in the rare disease space.
