Monkeypox Drug Approval Depends on Human-Based Clinical Trial Data
The peer-review New England Journal of Medicine recently published a decision to require human trial data to show that SIGA Technologies' antiviral medication TPOXX is safe and effective in treating people infected with the monkeypox virus (MPXV).
This Perspective was presented on August 3, 2022, by Adam Sherwat, M.D., John T. Brooks, M.D., Debra Birnkrant, M.D., and Peter Kim, M.D.
It stated: that there is a drug tecovirimat (TPOXX), available for clinical use under an expanded-access protocol that might conceivably speed the resolution of monkeypox illness and improve outcomes.
Yet it poses the conundrum: how to manage compassionate access to a drug whose safety and efficacy in humans have not been established.
Tecovirimat was approved for the treatment of smallpox disease under a regulation known as the "Animal Rule."
This U.S. FDA pathway allows for approval of drugs for serious or life-threatening conditions when it is not ethical to conduct efficacy studies in humans.
Since smallpox and monkeypox are caused by the same genus of viruses, to understand better the role that tecovirimat might play in our response to the monkeypox outbreak, it is essential to understand the basis for its approval by the FDA for the treatment of smallpox and the knowledge gaps that remain.
These doctors added: Before the onset of the current outbreak, the National Institutes of Health (NIH) had initiated planning for a randomized, controlled trial (RCT) in the Democratic Republic of Congo (DRC) to evaluate the safety and efficacy of tecovirimat in treating monkeypox.
However, the current worldwide outbreak involves a different clade of monkeypox virus than that which generally causes monkeypox infection in the DRC, and some of the clinical manifestations of the current outbreak and affected populations at this time differ from those in countries where monkeypox is endemic.
Therefore, the NIH is now also developing a U.S.-based RCT to assess the safety and efficacy of tecovirimat for the treatment of monkeypox disease.
The AIDS Clinical Trials Group will conduct this trial.
We anticipate that these trials will provide data needed for clinical and regulatory decision-making in the USA, concluded this NEJM Perspective.
Additional monkeypox treatment news is posted at MonkeypoxToday.org.
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