Ultragenyx aligns with FDA on Phase 3 design for GTX-102 in Angelman syndrome

Ultragenyx Pharmaceutical Inc.

FDA alignment on EoP2 meeting for GTX-102; Phase 3 trial to enroll ~120 UBE3A deletion patients.
Primary endpoint: Bayley-4 cognitive raw score; 48-week double-blind, sham-controlled design.
Patients in control arm eligible to roll over to treatment after double-blind period.
EMA accepted Phase 3 design under PRIME; PMDA meeting expected in coming weeks.
Plans for open-label study to evaluate GTX-102 in other Angelman genotypes and age groups.

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