CDC, NIH, FDA lead­ers call for US-based clin­i­cal tri­al of small­pox drug in treat­ing mon­key­pox

With the ris­ing num­ber of mon­key­pox cas­es, lead­ing re­searchers at the CDC, FDA and NIH are call­ing on a ran­dom­ized clin­i­cal tri­al to see if an ap­proved small­pox drug is ef­fec­tive at treat­ing mon­key­pox.

No mon­key­pox treat­ments are ap­proved in the US, so pa­tients look­ing to get re­lief for their le­sions and oth­er symp­toms from the virus must go through a set of hur­dles to get the small­pox drug through a gov­ern­ment ex­pand­ed ac­cess pro­gram. Ap­proved for small­pox in 2018, the drug is mar­ket­ed as TPOXX by the biotech SIGA. The Eu­ro­pean Union ap­proved it for mon­key­pox in ad­di­tion to small­pox ear­li­er this year and the UK fol­lowed suit in Ju­ly.

In an opin­ion piece pub­lished in the New Eng­land Jour­nal of Med­i­cine on Wednes­day, gov­ern­ment re­searchers said the NIH’s Na­tion­al In­sti­tute of Al­ler­gy and In­fec­tious Dis­eases is work­ing with the AIDS Clin­i­cal Tri­als Group, which has over­seen stud­ies of HIV meds since the 1980s, to set up a clin­i­cal tri­al to test TPOXX for mon­key­pox.

But while NI­AID, ACTG and SIGA all con­firmed to End­points News via email that a US-based clin­i­cal tri­al of the drug is be­ing planned, they de­clined to say when it would start. Ac­tivists ar­gue the tri­al should have tak­en place be­fore mon­key­pox be­came a glob­al out­break be­cause the virus has been en­dem­ic in some coun­tries in Africa for years.

“Adults with mon­key­pox in­fec­tion, in­clud­ing peo­ple liv­ing with HIV, would be el­i­gi­ble to en­roll. NI­AID will share more in­for­ma­tion as it be­comes avail­able,” the NI­AID said in an emailed state­ment.

“Those tri­als should have been ob­vi­ous­ly planned for well be­fore a cur­rent out­break. This is what the job of pan­dem­ic pre­pared­ness is. We ob­vi­ous­ly had mil­lions of dos­es stock­piled for this pur­pose and it is in­sane that the fed­er­al gov­ern­ment didn’t ac­tu­al­ly have ef­fi­ca­cy tri­als planned for it,” James Krel­len­stein, strat­e­gy & pol­i­cy man­ag­ing di­rec­tor for the or­ga­ni­za­tion PrEP4All, told End­points in a phone in­ter­view.

The re­searchers analo­gize the lack of ap­proved meds for the cur­rent mon­key­pox out­break to the ear­ly years of the HIV epi­dem­ic in the 1980s, when ther­a­pies had been de­vel­oped, but the process for ap­prov­ing their use in hu­mans was “painful­ly slow.” An eth­i­cal bal­ance was need­ed to en­sure pa­tients had ac­cess while al­so “up­hold­ing the re­spon­si­bil­i­ty” of en­sur­ing they were safe and ef­fec­tive, the re­searchers wrote Wednes­day in NE­JM.

“Now, 34 years lat­er, we are faced with an un­can­ni­ly sim­i­lar sit­u­a­tion,” they wrote. The au­thors of the piece in­clude John Brooks, CMO for the CDC’s Di­vi­sion of HIV Pre­ven­tion; De­bra Birnkrant, di­rec­tor of the Di­vi­sion of An­tivi­rals with­in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search; and Pe­ter Kim, deputy di­rec­tor of the Ther­a­peu­tic Re­search Pro­gram in the Di­vi­sion of AIDS at NI­AID.

Or­ga­ni­za­tions such as PrEP4All have called for the re­moval of re­stric­tions to the drug so pa­tients can get the med quick­er and more eas­i­ly as mon­key­pox case counts climb past 25,000 across 83 coun­tries. The US is one of the biggest out­break re­gions and three states — Illi­nois, New York and Cal­i­for­nia — have de­clared it a pub­lic health emer­gency.

Pri­or to the cur­rent glob­al out­break, the NIH had be­gun plan­ning for a ran­dom­ized clin­i­cal tri­al of the drug in the De­mo­c­ra­t­ic Re­pub­lic of the Con­go, but in their NE­JM piece, the re­searchers cite the need for a US-based tri­al since cur­rent world­wide cas­es are of a dif­fer­ent clade than what is seen in the DRC.

The US has ac­cess to about 1.7 mil­lion cours­es of the drug through the Strate­gic Na­tion­al Stock­pile, and BAR­DA has a con­tract with SIGA to re­plen­ish the cours­es when they ex­pire, ac­cord­ing to the com­pa­ny. As of last week, 10,000 cours­es of the drug had been sent to states and cities up­on re­quest, the De­part­ment of Health and Hu­man Ser­vices told NPR.

A SIGA spokesper­son said the com­pa­ny is “aware of the plans for a clin­i­cal tri­al and is work­ing with NI­IAD [sic] to ad­vance a place­bo-con­trolled study of tecovir­i­mat in the treat­ment of mon­key­pox.” SIGA re­ports quar­ter­ly earn­ings on Thurs­day.

SIGA can­celed a sched­uled in­ter­view be­tween sci­ence chief Den­nis Hru­by, CEO Phillip Gomez and End­points, say­ing that it “would pre­fer to wait for pub­li­ca­tion of the full ar­ti­cle in NE­JM be­fore com­ment­ing much fur­ther on any specifics.”

Hru­by told NBC News last month that plans for large clin­i­cal tri­als were in ad­vanced stages in Cana­da, the UK and the EU. Hru­by told the news out­let that SIGA might not re­ly on US tri­al da­ta to sub­mit to the FDA, but rather look to com­bined find­ings in oth­er coun­tries and safe­ty da­ta from glob­al com­pas­sion­ate-use pro­grams.

In their NE­JM piece, the CDC, FDA and NIH re­searchers wrote that they “an­tic­i­pate that these tri­als will pro­vide da­ta need­ed for clin­i­cal and reg­u­la­to­ry de­ci­sion mak­ing in the Unit­ed States.”

SIGA’s drug was ap­proved in the US in 2018 un­der the “An­i­mal Rule,” which al­lows drugs for se­ri­ous or life-threat­en­ing con­di­tions to be green­lit when hu­man stud­ies are deemed un­eth­i­cal. Ef­fi­ca­cy is es­tab­lished in an­i­mal mod­els of the hu­man dis­ease. The drug was ap­proved based on ef­fi­ca­cy da­ta gath­ered from in­fect­ing non­hu­man pri­mates with mon­key­pox virus and rab­bits with rab­bit­pox virus, as well as safe­ty da­ta from more than 400 healthy adult vol­un­teers.

As the mon­key­pox out­break spreads to more than 6,000 re­port­ed cas­es in the US, peo­ple at high­est risk have been scram­bling to get vac­ci­nat­ed against the virus. There is one jab ap­proved in the US, Bavar­i­an Nordic’s Jyn­neos, which is in lim­it­ed sup­plies in the US af­ter a na­tion­al stock­pile large­ly evap­o­rat­ed in re­cent years. A small­pox vac­cine, ACAM2000, is al­so be­ing used in cer­tain in­stances.

Mean­while, Mod­er­na lead­ers said Wednes­day that the pan­dem­ic-in­fa­mous biotech is look­ing in­to a po­ten­tial mon­key­pox pro­gram.

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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

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Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

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