Shots:
- Ultragenyx has reported the P-III (Enh3ance) study data assessing DTX301 AAV8 gene therapy vs PBO for the treatment of ornithine transcarbamylase (OTC) deficiency
- DTX301 reduced 24hr. plasma ammonia by 18% vs PBO at Wk. 36, with ammonia levels maintained in the normal range, despite a 27% mean decrease in scavenger drugs & ~13% increase in protein intake; 8/9 pts with abnormal baseline ammonia normalized rapidly
- At Wk. 24, DTX301 showed improved PROs per PGIC, with 71% reporting much improved vs 0% (total n=15 reporting), while 64% reported moderate (21%) or much improvement (43%) in symptoms & daily living vs 19% (total n=30 reporting). 1EP evaluation is under evaluation, with data expected in H1’27
Ref: Globenewswire | Image: Ultragenyx | Press Release
Related News: Ultragenyx Completes FDA Rolling BLA Submission for DTX401 in Glycogen Storage Disease Type Ia (GSDIa)
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