Next-gen mpox treatments: FDA calls for diverse, randomized controlled trials
For companies looking to develop the next mpox (formerly known as monkeypox) antivirals, the FDA came out with new draft guidance yesterday spelling out the details of what such development programs should include.
One of the major points in the draft is the FDA is calling for any new mpox treatments to run randomized controlled trials with wide racial and ethnic diversity and those with HIV, as mpox disproportionately affects these populations.
“Sponsors should ensure that clinical trial sites include geographic locations with a higher concentration of racial and ethnic minorities to recruit a diverse study population,” FDA said. “Current data suggest that, in the United States, approximately 40 percent of mpox cases have occurred in individuals living with human immunodeficiency virus (HIV) infection. Individuals living with HIV infection should not be excluded from clinical trials.”
In September 2022, NIAID started a clinical trial of tecovirimat in the US in collaboration with the AIDS Clinical Trials Group (ACTG). The trial is enrolling more than 500 adults and children with mpox at clinical research sites nationwide.
Timothy Wilkin, who specializes in infectious disease at Weill Cornell Medicine and is working on that tecovirimat trial, told Endpoints News that the guidance:
also encapsulates what we developed with FDA guidance for the current tecovirimat study. I think it is important that the FDA specifically called for people living with HIV to be included. In developing STOMP (the phase 3 trial of tecovirimat), the FDA encouraged us to maximize inclusivity. We are enrolling children of all ages and pregnant people.
Tecovirimat (also known as Tpoxx or ST-246) is FDA-approved for the treatment of human smallpox disease caused by Variola virus in adults and children, and is manufactured by the pharmaceutical company SIGA Technologies in New York City.
“The FDA may be releasing this to provide clarity for other drugs being considered such as brincidofovir,” Wilkin added.
The seven-page draft also includes pharmacology, toxicology, virology, as well as other clinical recommendations.
“The primary endpoint for phase 2 or phase 3 clinical trials should be clinically meaningful and should demonstrate the clinical benefit with a favorable effect on a meaningful aspect of how a participant with mpox feels, functions, or survives,” FDA said.