01 · RegulatoryLink
FDA approves the first gene therapy for glycogen storage disease type Ia
US Food and Drug Administration ·
Genglycos (pariglasgene brecaparvovec-opnr) is a one-time, liver-directed AAV8 gene therapy delivering a functional G6PC gene, approved for patients aged 8 and above. Until now the condition has been managed by strict around-the-clock cornstarch supplementation and nothing else.
This is an accelerated approval on a surrogate endpoint. Over 48 weeks, treated patients reduced daily cornstarch intake by a mean of 31 per cent against placebo, roughly one dose a day. Confirmatory trials are required. Warnings cover anaphylaxis, liver toxicity, adrenal insufficiency and tumorigenicity.