Use of TPOXX® for Treatment of Monkeypox though Expanded Access Pathway

Image Credit
Cynthia S. Goldsmith, Russell Regnery
Digitally-colorized electron microscopic (EM) image depicting monkeypox virus particles. It is a thin section image from a human skin sample. On the left are mature, oval-shaped virus particles and on the right are spherical particles of immature virions.

On Thursday, 4 August 2022, a public health emergency was declared for the contagious disease known as monkeypox. Do you know how to access tecovirimat (brand name TPOXX® from Siga Techologies) an anti-viral to treat patients that have tested positive for monkeypox? Several players are involved in making this happen!

TPOXX® is approved for the treatment of human smallpox which is caused by the Variola virus, an orthopox virus. However, there are enough similarities across orthopoxviruses that TPOXX® confers some protection and treatment for non-variola orthopox infections such as monkeypox. A supply of TPOXX® is currently stored in the Strategic National Stockpile (SNS) for just such an outbreak. The SNS is positioned to distribute products such a TPOXX® in a rapid fashion from storage locations across the US. Since it is not approved for monkeypox, this treatment is considered investigational and must go through the FDA Expanded Access pathway.

The CDC and the FDA have made a streamlined path to follow utilizing the Expanded Access process for an investigational new drug. The CDC holds an Intermediate-Size Patient Population Expanded Access IND to allow for access to TPOXX® for treatment of monkeypox, and the CDC IRB is willing to be the IRB of record. This means that individual physicians will not need to submit anything to the FDA and can work with their IRB for an expedited submission. Physicians without an IRB have only a simple contract to sign for the CDC IRB to take this role. The treatment protocol and informed consent have already been vetted and are ready for use.

All of this preparation from the CDC and FDA is meant to minimize the burden on physicians, so they can focus on coordinating care with their local health department and treating their patients.

If you have a patient displaying symptoms of monkeypox, refer to the monkeypox guidance provided by the CDC for each of the needed steps. Interested in learning more about Expanded Access pathways? Check out the TEAMSS website where the steps for treatment use are outlined and templates provided.

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