In quest to eliminate tropical diseases, Merck KGaA will submit anti-worming drug for approval in young children

As Mer­ck KGaA makes moves to treat the ne­glect­ed trop­i­cal dis­ease schis­to­so­mi­a­sis, it has an­nounced that its treat­ment arpraz­i­quan­tel has yield­ed pos­i­tive Phase III tri­al re­sults in chil­dren be­tween the ages of 3 months and 6 years old, and the com­pa­ny will now seek reg­u­la­to­ry ap­proval.

A pe­di­atric ver­sion of the stan­dard drug praz­i­quan­tel, arpraz­i­quan­tel is an an­ti-worm med­ica­tion that pre­vents new­ly hatched, par­a­sitic worms from grow­ing or mul­ti­ply­ing in­side a pa­tient. The tri­al could of­fer a cure for mil­lions.

In tri­als in Cote d’Ivoire and Kenya, more than 90% of par­tic­i­pants had no more par­a­site eggs in their stool or urine af­ter up to three weeks of treat­ment, Mer­ck KGaA said. The com­pa­ny will ap­ply for a sci­en­tif­ic opin­ion from EMA through the EU’s high-pri­or­i­ty med­i­cine pro­ce­dure, in­tend­ed for use out­side of Eu­rope. Mer­ck KGaA has got­ten sup­port from the Glob­al Health In­no­v­a­tive Tech­nol­o­gy Fund, Far­man­guin­hos in Brazil and Uni­ver­sal Cor­po­ra­tion in Kenya to ex­tend lo­cal pro­duc­tion ca­pac­i­ties. ‘With this mile­stone, we con­tin­ue our com­mit­ment to elim­i­nat­ing schis­to­so­mi­a­sis and en­sur­ing all peo­ple af­fect­ed by this ne­glect­ed trop­i­cal dis­ease have ac­cess to a life-sav­ing ther­a­py. To­geth­er with our con­sor­tium part­ners, we are stead­fast in our vi­sion to bring new hope to the world’s most vul­ner­a­ble pop­u­la­tions,’ Mer­ck KGaA’s health­care CEO Pe­ter Guenter said in a press re­lease.

The com­pa­ny is ad­dress­ing key re­quire­ments of the WHO’s 2021-2030 roadmap for ne­glect­ed trop­i­cal dis­eases through its Schis­to­so­mi­a­sis Elim­i­na­tion Pro­gram.

The pri­ma­ry end­point of the tri­al was de­fined as no par­a­site eggs in stool 17 to 21 days af­ter treat­ment, or in urine 35 to 40 days af­ter treat­ment. No new po­ten­tial risks or safe­ty con­cerns were iden­ti­fied, the com­pa­ny said in a press re­lease.

Arpraz­i­quan­tel is de­liv­ered through an oral tablet. There are an es­ti­mat­ed 50 mil­lion preschool-aged chil­dren with schis­to­so­mi­a­sis.

Kio Yam­abe, the act­ing CEO of the GHIT Fund, said in a state­ment:

Hav­ing part­nered with the Pe­di­atric Praz­i­quan­tel Con­sor­tium since 2013, we be­lieve that in­ter­na­tion­al col­lab­o­ra­tions like this are key to ad­dress­ing the bur­den of ma­jor in­fec­tious dis­eases in the de­vel­op­ing world. The suc­cess­ful joint de­vel­op­ment of praz­i­quan­tel by Con­sor­tium part­ners, Astel­las, Mer­ck and Far­man­guin­hos em­bod­ies our un­wa­ver­ing com­mit­ment to dri­ve Japan­ese in­no­va­tion and tech­nol­o­gy through glob­al part­ner­ships.

The dis­ease is al­so known as bil­harzia. Fa­mous­ly, Is­raeli pro­fes­sion­al cy­clist Chris Froome has bat­tled the dis­ease since 2010, and test­ed pos­i­tive for an­oth­er flare-up ear­li­er this year. Doc­tors told him that the worm could have been dor­mant for near­ly a decade be­fore flar­ing up again. Froome learned of the flare-up af­ter hav­ing di­ges­tive is­sues af­ter the Tour de France.

The drug works by caus­ing se­vere spasms and paral­y­sis of the worms’ mus­cles. Of­ten, those are passed in the pa­tient’s stool, but some­times, they are com­plete­ly de­stroyed in the bow­els, ac­cord­ing to the Mayo Clin­ic.

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