The FDA granted accelerated approval to Ultragenyx Pharmaceutical’s Genglycos, the first approved treatment for glycogen storage disease type Ia.
The one-time AAV8-based gene therapy is approved to reduce daily cornstarch intake as an adjunct to nutritional management in adults and pediatric patients 8 years of age and older with the rare inherited genetic disorder, according to an Aug. 19 news release. The therapy delivers a functional G6PC gene to the liver.
In a randomized, double-blind, placebo-controlled study, patients receiving Genglycos had a statistically significant mean reduction from baseline in daily cornstarch intake of 31% compared with placebo over 48 weeks after dosing. The manufacturer must complete additional clinical trials to confirm the therapy’s effectiveness.
Across two clinical studies, serious adverse reactions included anaphylaxis, adrenal insufficiency, high lactate levels and hypoglycemia. Genglycos-treated patients had a higher rate of hypertriglyceridemia than placebo-treated patients, 29% vs 8%.
