FDA approves Genglycos for GSDIa in adults and children 8 and older
The FDA says Genglycos is the first approved treatment for glycogen storage disease type Ia and was cleared under the accelerated approval pathway for patients 8 years and older.
The U.S. Food and Drug Administration has granted accelerated approval to Genglycos, or pariglasgene brecaparvovec-opnr, for adults and pediatric patients 8 years of age and older with glycogen storage disease type Ia, or GSDIa. The agency said the one-time gene therapy is intended to reduce daily cornstarch intake alongside nutritional management.
The FDA said Genglycos is the first approved treatment for the condition. GSDIa is a rare inherited disorder that can cause dangerously low blood sugar when a person goes too long without eating. Over time, the disease can lead to metabolic complications that affect normal function in certain organs and tissues, according to the agency.
People with GSDIa are typically managed with careful medical monitoring, frequent meals, avoidance of certain foods containing simple sugars, and daily uncooked or specially formulated cornstarch. The FDA said the approval was based on clinical trial data showing that Genglycos reduced daily cornstarch intake, which the agency described as a surrogate endpoint reasonably likely to predict clinical benefit.
In a randomized, double-blind, placebo-controlled study followed for 48 weeks after dosing, patients treated with Genglycos had a statistically significant mean reduction in daily cornstarch intake of 31% compared with placebo, the FDA said. The agency also said the treatment group showed a mean reduction of one cornstarch dose per day versus placebo. The release noted a numerical mean 3% increase in glucose values in the hypoglycemic range, defined as less than 70 mg/dL, in the Genglycos group versus placebo.
The approval was granted to Ultragenyx Pharmaceutical, Inc. The FDA said the therapy is a one-time AAV8-based gene therapy designed to deliver a functional G6PC gene to the liver, with the aim of restoring the deficient enzyme and helping stabilize blood sugar during fasting.
The agency said accelerated approval requires the manufacturer to complete additional clinical trials to confirm effectiveness. The application also received a Rare Pediatric Disease Priority Review Voucher, and the therapy had received regenerative medicine advanced therapy and Fast Track designations, the FDA said.
The FDA release also listed safety concerns. Serious adverse reactions reported in clinical studies included anaphylaxis, adrenal insufficiency, high lactate levels and hypoglycemia. Common adverse reactions included increased transaminases, nausea, headache, constipation and hyperglycemia. The agency said Genglycos-treated patients had a higher rate of hypertriglyceridemia than placebo, at 29% versus 8%.
Prescribing information carries warnings about anaphylaxis, liver toxicity, adrenal insufficiency and tumorigenicity, according to the FDA. The agency also said the treatment should not be used during pregnancy.
FDA officials quoted in the release said the approval is an advance for patients living with the condition. Karim Mikhail, acting director of the Center for Biologics Evaluation and Research, said the approval is a milestone in using gene therapy to treat the disease and improve quality of life. Megha Kaushal, acting deputy director of the CBER Office of Therapeutic Products, said the approval reflects confidence in the evidence to date while data continue to be gathered to confirm long-term benefit.
Ultragenyx must complete additional trials to verify effectiveness under the accelerated approval pathway, the FDA said.
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Editorial note: This article was prepared with AI assistance and includes source attribution for reader review.
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