Ultragenyx Pharmaceutical Gets FDA Agreement to Expand GTX-102 Study

By Chris Wack

Ultragenyx Pharmaceutical said Wednesday that the U.S. Food and Drug Administration has reviewed and agreed to a protocol amendment to a Phase 1/2 study evaluating the safety and tolerability of GTX-102 in pediatric Angelman syndrome patients with a genetically confirmed diagnosis of full maternal UBE3A gene deletion.

The Novato, Calif.-based biopharmaceutical company said the amendment enables it to harmonize dose ranges in the U.S. with dose ranges used in international cohorts for the open-label, dose-escalating Phase 1/2 study, which is also looking at clinical responses measured by a panel of efficacy assessments for functional domains impacted in Angelman syndrome.

Outside the U.S., the Phase 1/2 study has been actively enrolling and administering GTX-102 to expansion patient cohorts to the dosing and treatment regimen that will be used in the Phase 3 program.

Write to Chris Wack at chris.wack@wsj.com

(END) Dow Jones Newswires

May 17, 2023 08:41 ET (12:41 GMT)

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