Shots:
- Ultragenyx reported the US FDA accelerated approval to GENGLYCOS (pariglasgene brecaparvovec-opnr; DTX401) for adults and pediatric patients (≥8yrs., n=46) with glycogen storage disease type Ia (GSDIa)
- The approval was supported by the P-III (GlucoGene) study, in which GENGLYCOS (n=20) demonstrated a statistically significant reduction in cornstarch requirements at 48wks. vs PBO (n=24) (p<0.001). The therapy is designed to restore the liver’s ability to release glucose during fasting or metabolic stress, potentially reducing the burden of intensive dietary management.
- The company also received a Priority Review Voucher with the approval. Furthermore, as part of the accelerated approval, Ultragenyx will conduct post-marketing monitoring through its GSDIa Disease Monitoring Program, providing additional safety and efficacy data on cornstarch requirements, fasting tolerance, and other measures.
Ref: Company Release | Image: Ultragenyx | Press Release
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