Clinical hold on Pompe disease drug brings new gene therapy woes for Astellas

The US Food and Drug Administration (FDA) has placed a clinical hold on the FORTIS PhI/II trial following a serious adverse event (SAE) of peripheral sensory neuropathy in one of the trial participants.

FORTIS is evaluating Astellas’ (TYO: 4503) AT845, an investigational adeno-associated virus (AAV) gene replacement therapy in adults with late-onset Pompe disease.

"We are working closely with the FDA to determine appropriate next steps"The FDA informed the Japanese drugmaker that it did not have sufficient information to assess the risks to subjects and requires more information about the recently reported SAE.