As monkeypox cases continue to rise in the US and around the world, many of those most affected by the virus say they do not have access to effective treatment.
The current monkeypox outbreak has led to more than 21,100 confirmed cases worldwide, including more than 4,900 in the US. The virus has infected people in Central and West Africa for decades, but recently started circulating abroad, so far mainly among men who have sex with men. It spreads through close skin-to-skin contact, including during sex. The virus causes painful, blister-like lesions — in the current outbreak, these are commonly found on the genitals and anus — and sometimes flu-like symptoms. At least five people have been killed, according to the World Health Organization.
An antiviral drug called tecovirimat, or TPOXX, has already been approved by the U.S. Food and Drug Administration for the treatment of smallpox and may also be used in monkeypox, a related virus. But the only U.S. stockpile of this drug is in the nation’s strategic national stockpile, and the Centers for Disease Control and Prevention require health care providers to submit an application for a new investigational drug, or IND, to access their patients. . Critics say the process to obtain an IND for TPOXX is cumbersome, however, and the drug — which was originally tested on animals with monkeypox — should be more readily available for treating the disease in humans.
“The TPOXX issues don’t make sense from a scientific standpoint,” said gay rights activist James Krellenstein, co-founder and director for strategy and policy at PrEP4All, an organization working to increase access to HIV medications. “TPOXX is approved based on animal data [for monkeypox],” he says. But the CDC’s stance is that “you can’t prescribe” [the] drug…for monkeypox because the FDA label says, ‘Only smallpox.’”
Krellenstein and a group of infectious disease experts wrote a letter to the heads of the U.S. Department of Health and Human Services, CDC and FDA detailing the barriers to accessing TPOXX for the treatment of monkeypox. The letter, dated June 15 and shared with Scientific Americanargued that the IND process was too burdensome for healthcare providers, who she said had to review a protocol of more than 100 pages to access the drug and collect comprehensive information about each patient who received it, as well as photos of lesions and other data.
The CDC then clarified its protocol for obtaining TPOXX, which allows the forms to be submitted after treatment has begun and makes the submission of photos and samples of lesions optional. “CDC posted a clarification on the process for obtaining TPOXX after hearing confusion from clinicians experiencing issues,” said Kevin Griffis, a spokesperson for the agency. “The post and a subsequent email to clinicians [make] clear that providers can obtain the medication directly from CDC or through their state health departments and fill out the required forms after treatment has begun.
The FDA was also involved in the process. “The FDA worked closely with the CDC to streamline the protocol to reduce data collection and reporting requirements. The revised protocol is now available for use,” said FDA spokesman Chanapa Tantibanchachai.
The letter from Krellenstein and others also pointed out that TPOXX was approved by the FDA in 2018 for the treatment of smallpox. There was no data on the efficacy of TPOXX in humans, as it would be clearly unethical to infect humans with smallpox for a trial. Instead, the drug was approved under the FDA’s so-called Animal Rule, based on data for two related viruses: monkeypox in non-human primates and rabbit pox in rabbits. But despite this, the CDC still states that an IND is needed to provide TPOXX for monkeypox, as it is only FDA-approved for smallpox. The letter called this “illogical.”
Krellenstein and his colleagues have called for one of two solutions to make TPOXX more readily available to people with monkeypox: In one, the CDC would withdraw the requirement for an IND to access the drug from the national supply. Otherwise, the FDA would make TPOXX available for monkeypox through an emergency use authorization (EUA) — a process that allows unapproved medical products, or unapproved uses of those products, to diagnose, treat, or prevent serious disease. In order for the FDA to issue an EUA, the Secretary of HHS must first declare an emergency, under Section 564 of the Federal Food, Drug, and Cosmetic Act. Emergencies have been declared for COVID-19 and the opioid crisis. But so far, HHS Secretary Xavier Becerra has declared no such emergency for monkeypox.
“The buck has to stop with him,” says Krellenstein. “He now has to do his job and figure out how to solve this problem. It’s not up to us to do his job anymore. This [letter] is addressed to Secretary Becerra for a reason, and Secretary Becerra must do this.”
In a press briefing this week, Becerra told reporters he would individually weigh an emergency declaration for monkeypox and other public health issues based on the criteria for such a declaration. “We continue to monitor the response to monkeypox across the country,” he said. “We have made vaccines, tests and treatments – far in excess of the numbers currently needed – available to all jurisdictions managing their public health systems, and are willing to work with clinicians to make all three – tests, treatments and vaccines – available. to make . And we will weigh any decision about declaring a public health emergency based on the response we see across the country.”
During the briefing, Becerra also noted that the government had secured an additional 786,000 doses of the JYNNEOS monkeypox vaccine, in addition to the more than 330,000 already distributed in jurisdictions across the country.
“There is no report of a public health emergency at this time,” Bill Hall, a spokesperson for HHS, wrote in an email to Scientific American. “If such a statement is made in the future, it will be posted here.”
The US has also struggled with its response to monkeypox testing and vaccination, with some experts saying it may already be too late to prevent the disease from becoming endemic worldwide. But Garrett Wilkinson, government relations and policy officer at the nonprofit Partners In Health and a signer of the letter with Krellenstein, thinks assessment is counterproductive. “I would hate it if the message is ‘It’s too late,'” Wilkinson says.
Krellenstein agrees. “We are now at a decision point. There are two forks in the road where we can either try to get it under control,” he says, “or we can’t.”
.