This market will resolve to No if the FDA's decision is a denial (CRL issued), a withdrawal by the sponsor, or a conditional approval — or if no decision is issued.

DTX401 (pariglasgene brecaparvovec) is a one-time gene therapy developed by Ultragenyx Pharmaceutical to treat Glycogen Storage Disease Type Ia (GSDIa), a rare inherited disorder in which the liver cannot properly release stored sugar into the bloodstream. Think of it like installing a corrected instruction manual inside liver cells: after a single infusion, the therapy is designed to restore the missing enzyme activity — glucose-6-phosphatase — so the liver can once again regulate blood sugar on its own. In the Phase 3 GlucoGene trial , DTX401 produced a statistically significant 41.3% mean reduction in daily cornstarch intake at Week 48 versus 10.3% for placebo (p<0.0001), with durability data at 96 weeks showing approximately 60–61% reductions.