01.09.25 09:19, Börse Global (en).
Clinical Program Pivot Following FDA Decision A significant development emerged from regulatory discussions with the U.S. Food and Drug Administration. The agency imposed a clinical hold on Design Therapeutics' planned expansion of its key RESTORE-FA study within the United States. This critical trial evaluates the promising drug candidate DT-216P2 as a treatment for Friedreich's ataxia, a progressive neurological disorder. While initial pharmacokinetic data had shown encouraging results, the FDA's decision has substantially delayed the program's advancement and raised questions about the regulatory path forward. Despite this setback in the U.S. market, the company continues to progress with the study outside American borders.
