Astellas Pauses XLMTM Gene Therapy Trial After Another Safety Scare

Less than a year after the US Food and Drug Administration allowed its Phase I/II study of AT132 for X-linked myotubular myopathy (XLMTM) to resume following a months-long clinical hold over adverse events, Astellas Pharma, Inc. has hit the pause button on the gene therapy trial again due to a safety issue. The hold emphasizes the growing concern about the safety of adeno-associated viral (AAV) vector-based gene therapies in general on the eve of an FDA advisory committee on the topic.

The Tokyo-based drug maker said 1 September that it had paused screening and enrollment into the ASPIRO trial after a patient dosed during the summer experienced a severe adverse event of abnormal liver function tests (LFTs) in the weeks after receiving a lower dose – 1.3x10^14vg/kg – of the gene therapy