Astellas Halts Gene Therapy Trial Following Serious Adverse Event

September 3, 2021

Astellas Pharma has stopped enrolling and dosing in a phase 1/2 clinical trial of its genetic therapy AT132 for X-linked myotubular myopathy, a rare neuromuscular disease, after a subject suffered a serious adverse event.

The Tokyo-based company said that a study participant had abnormal liver function test results weeks after receiving a low dose of AT132. Astellas said it has been communicating with regulatory agencies, which have not yet placed a clinical hold on the trial.

Of the 24 study participants, three had previously developed hepatitis and later died. The FDA imposed a clinical hold on the trial as a result, but lifted it in December 2020 after reviewing changes to the trial protocol.

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