Tecovirimat and mpox: A regulatory balancing act between hope, hurdles, and high-risk populations

Amid recent clinical trial results, the regulatory landscape for tecovirimat in the treatment of mpox has become increasingly complex. The US FDA's decision to not proceed with the approval of tecovirimat for mpox appears well-founded, given the lack of significant clinical benefits observed in large-scale trials. Meanwhile, Japan's broad approval of tecovirimat for all orthopoxviruses, including mpox, may be premature. This approval should be revisited to focus on high-risk populations who may derive the most benefit from the treatment. Similarly, the European Medicines Agency (EMA) and the U.K.’s Medicines & Healthcare products Regulatory Agency (MHRA) approval of tecovirimat for mpox should be reassessed, with consideration given to restricting its use to high-risk individuals. Moving forward, clinical trials must focus on the most vulnerable populations and assess the role of tecovirimat in those who receive treatment early. At this time, we recommend that the EA-IND not be discontinued, but rather be refined to provide access to patients who may benefit, while awaiting further evidence from future RCTs to clarify the drug's role and effectiveness.