RARE - Ultragenyx gets FDA nod to amend dosing ranges in trial of neuro disorder drug

2023-05-17 08:46:05 ET

• Ultragenyx Pharmaceutical ( NASDAQ: RARE ) said the U.S. Food and Drug Administration (FDA) agreed to a protocol amendment to a phase 1/2 trial of GTX-102 in children with Angelman syndrome.
• The amendment allows the company to harmonize dose ranges in the U.S. with those being used in ex-U.S. groups of the trial.
• Outside of the U.S., trial has been enrolling and dosing patients in the expansion groups to verify the GTX-102 dose and treatment regimen to be used in a phase 3 program.
• "We have begun working urgently to activate multiple study sites in the U.S. and plan to begin enrollment as quickly as possible," said Scott Stromatt, SVP and chief medical officer of neurology clinical development, Ultragenyx.
• Angelman syndrome is a rare neurogenetic disorder which causes developmental delay or intellectual disability, speech and movements issues, and also seizures among other symptoms. People with the disorder often smile and laugh frequently, and have a happy demeanor.

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