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Getting the latest healthcare news for you
Getting the latest healthcare news for you
A one-time gene therapy just changed the game for a rare metabolic disease. The FDA granted accelerated approval to Genglycos (pariglasgene brecaparvovec-opnr) for glycogen storage disease type Ia (GSD Ia) in patients aged 8 and older — the first therapy of its kind for this condition. In a phase 3 trial, it cut patients' daily cornstarch requirements by 31% compared to placebo.
A one-time gene therapy just changed the game for a rare metabolic disease. The FDA has granted accelerated approval to Genglycos (pariglasgene brecaparvovec-opnr, Ultragenyx) for glycogen storage disease type Ia (GSD Ia) in patients aged 8 and older — marking the first-ever FDA-approved therapy to reduce the burden of care for this condition. GSD Ia is a rare genetic disorder where a deficiency of the glucose-6-phosphatase enzyme prevents the liver from regulating blood sugar, forcing patients to consume raw cornstarch multiple times daily to avoid life-threatening hypoglycemia.
The approval is based on the phase 3 GlucoGene study, a double-blind trial of 46 patients. Those receiving Genglycos saw a 31% greater reduction in cornstarch requirements at 48 weeks versus placebo, along with a reduction of one cornstarch dose per day. Serious adverse events included anaphylaxis, adrenal insufficiency, high lactate levels, and hypoglycemia, while the most common side effects were elevated liver enzymes, nausea, headache, constipation, and hyperglycemia.
By the Numbers:
Why it matters: For patients and families, GSD Ia demands near-perfect daily management — any missed cornstarch dose can trigger seizures or death. A one-time gene therapy that meaningfully reduces that burden represents nearly 30 years of scientific progress and a meaningful quality-of-life advance for this community.
