FDA Approves First Smallpox Drug

In order to address concerns of the use of smallpox as a bioweapon, the US Food and Drug Administration (FDA) has approved TPOXX (tecovirimat)—the first drug with an indication for the treatment of smallpox.

The approval was granted under the FDA’s Animal Rule, allowing the findings from animal studies to support approval when it is not ethical to conduct efficacy trials in humans. Successful studies of TPOXX vs placebo were performed in animals infected with viruses closely related to the one that causes smallpox. In these studies, researchers found that a greater number of TPOXX-treated animals survived compared with placebo-treated animals.

The safety of TPOXX was subsequently assessed in 359 healthy human volunteers without smallpox.

Headache, nausea, and abdominal pain were the most common side effects with TPOXX use.

—Christina Vogt


FDA approves the first drug with an indication for treatment of smallpox [press release]. US Food and Drug Administration. July 13, 2018. approves first drug with an indication for treatment of smallpox Published on July 16, 2018.