DTX401 / ANNUAL
No resting orders on this market.
Important information: 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 โ 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 โ glucose-6-phosphatase โ 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 60โ61% reductions.
Resolves Yes if the FDA's decision on DTX401 (pariglasgene brecaparvovec) by Ultragenyx Pharmaceutical in 2026 is a full approval or an accelerated approval, the market resolves to Yes.
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 โ 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 โ glucose-6-phosphatase โ 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 60โ61% reductions.