FDA lifts hold on PTC Huntington’s disease trial

BioPharma Dive June 20, 2024
Jonathan Gardner

One-year study data showing PTC’s pill suppressed a key protein helped persuade the regulator to remove a partial clinical hold it had put in place in 2022.

Dive Brief:

• PTC Therapeutics said Thursday it can resume a Phase 2 trial of its Huntington’s disease drug in the U.S., following the Food and Drug Administration’s review of data showing the pill reduced a mutant protein linked to the disorder.
• The drug, called PTC518, suppressed levels of huntingtin protein in both the blood and the cerebrospinal fluid by 43% in patients who took a once-daily 10 milligram dose over 12 months, PTC said. The company had previously disclosed data showing that dose reduced blood levels by around 30% through 12 weeks.
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