Tsoom Fwv Teb Chaws Asmeskas FDA pom zoo Fintepla (fenfluramine qhov ncauj daws) los kho tus mob Dravet!

Zogenix yog lub tuam txhab ua lag luam tshuaj rau kev txhim kho cov tshuaj rau kev kho mob ntawm cov kabmob tsis tshua muaj. Tsis ntev los no, lub tuam txhab tshaj tawm tias Asmeskas Chaw Tswj Xyuas Khoom Noj thiab Tshuaj (FDA) tau pom zoo Fintepla (fenfluramine) qhov ncauj tshuaj CIV rau kev kho mob vwm mob cuam tshuam nrog Dravet syndrome rau cov neeg mob ≥2 xyoos. Cov tshuaj tau pom zoo los ntawm kev tshuaj xyuas tus txheej txheem ua ntej. Ua ntej no, FDA tau tso cai rau Fintepla chaw tu menyuam yaus tsim nyog txog yeeb tshuaj (ODD) thiab kev tsim txom cov tshuaj muaj peev xwm (BTD) los kho Dravet syndrome-cuam tshuam nrog kev mob vwm.

Cov tsos mob Dravet yog ib qho mob sib kis mob rau me nyuam yaus tsawg kawg uas pom los ntawm kev qaug dab peg thiab siv tshuaj tiv thaiv mob hnyav, uas cuam tshuam txog tsev kho mob thiab kev kho mob maj nrawm, kev loj hlob loj thiab txav mus los, thiab kev tuag sai sai (SUDEP) muaj kev pheej hmoo ntau.

Fintepla yog cov kua tsim ntawm fenfluramine koob tshuaj, tuaj yeem txo qhov nquag ntawm qaug dab peg los ntawm kev hloov kho cov kev ua ntawm serotonin receptors thiab sigma-1 receptors (saib kev siv: Fenfluramine ploj zuj zus NMDA receptor-mediated qaug dab peg los ntawm nws cov kev sib xyaw ntawm serotonin 5HT2A thiab hom 1 sigma receptors). Cov ntaub ntawv los ntawm ob qhov kev tso tshuaj rau cov theem III soj ntsuam pom tau hais tias nyob hauv cov neeg mob uas tsis muaj peev xwm tswj tau qhov qaug dab peg zoo nrog lwm cov tshuaj, Fintepla txo qis qhov zaus ntawm qhov mob chua leeg tsawg dua piv rau cov placebo.

Joseph Sullivan, MD, tus kws tshawb nrhiav soj ntsuam kev tshawb fawb ntawm Fintepla thiab tus thawj coj ntawm Center for Excellence in Pediatric Epilepsy ntawm Benioff Cov Tsev Kho Mob Tsev Kho Mob ntawm University of California, San Francisco, tau hais tias: Tsis muaj qhov xav tau, txawm hais tias lawv Tom qab noj ib lossis ntau cov tshuaj muaj kab mob siab, cov qaug dab peg tseem muaj heev. Muab hais tias Fintepla tau txo qis ntau zaus ntawm qhov mob chua leeg hauv qhov kev sim tshuaj, txuas nrog kev saib xyuas tas mus li thiab muaj kev nyab xeeb, Kuv xav tias Fintepla Nws yuav muab txoj kev kho mob tseem ceeb heev rau cov neeg mob Dravet syndrome."

Fenfluramine-cov qauv lus qauv (qauv duab: Wikipedia.org)

FDA tau pom zoo Fintepla rau kev kho mob ntawm Dravet mob ntsig txog kev mob vwm raws li cov ntaub ntawv los ntawm ob qho kev sib tw, ob qhov muag tsis pom kev, cov tshuaj tiv thaiv kab mob qib III tau soj ntsuam (hauv cov neeg mob hnub nyoog 2-18 xyoo, cov txiaj ntsig tau tshaj tawm hauv Lancet thiab JAMA Neurology, feem ), thiab Kev Nyab Xeeb cov ntaub ntawv ntawm qhib daim ntawv lo ncua kev sim (ntau tus neeg mob tau txais Fintepla txog 3 xyoos). Cov ntaub ntawv qhia tau hais tias hauv kev tshawb nrhiav cov neeg mob uas tau noj ib lossis ntau cov tshuaj tiv thaiv kev mob vwm uas tsis tau tswj kev qaug dab peg, Fintepla tau txo cov mob qaug dab peg txhua lub hlis piv rau cov placebo, raws li cov kev kho mob uas twb muaj lawm. chua leeg, CS) zaus. Ib qho ntxiv, feem ntau ntawm cov neeg mob tshawb nrhiav hauv 3-4 lub lis piam pib pib kev kho Fintepla thiab kho zoo ib yam li cov sijhawm kho mob.

Nws yog tsim nyog sau cia tias Fintepla' s daim ntawv qhia cov tshuaj suav nrog lub npov dub ceeb toom tias cov tshuaj cuam tshuam nrog lub plawv mob ntsws (VHD) thiab mob ntsws ntsig (PAH). Vim tias cov kev pheej hmoo no, cov neeg mob yuav tsum muaj kev saib xyuas echocardiographic txhua txhua rau lub hlis ua ntej thiab thaum kho, thiab kev soj ntsuam echocardiographic peb mus rau rau lub hlis tom qab kho tas. Yog tias qhov kev kho mob echocardiogram qhia txog cov tsos mob ntawm lub plawv, PAH, lossis lwm yam kev mob plawv, cov kws kho mob yuav tsum xav txog cov txiaj ntsig thiab kev pheej hmoo ntawm kev txuas ntxiv siv Fintepla los kho cov neeg mob.

Vim tias muaj VHD thiab PAH txaus ntshai, Fintepla tsuas yog siv raws li kev tshuaj ntsuam xyuas kev pheej hmoo thiab txo kev siv tswv yim (REMS) los ntawm cov phiaj xwm phiaj xwm muag tshuaj txwv. Fintepla REMS xav kom cov kws kho mob uas hais txog Fintepla thiab cov chaw muag tshuaj uas faib Fintepla tau lees paub tshwj xeeb hauv Fintepla REMS thiab xav kom cov neeg mob mus rau npe hauv REMS. Ua ib feem ntawm REMS qhov yuav tsum tau ua, kev muab tshuaj rau cov kws kho mob thiab cov neeg mob yuav tsum ua raws li qhov yuav tsum tau kuaj xyuas lub plawv echocardiographic kom tau txais Fintepla kho mob.

Hauv cov kev tshawb fawb soj ntsuam, qhov tshwm sim tsis zoo tshaj plaws ntawm Fintepla (tshwm sim ≥10% thiab siab dua cov placebo) suav nrog: tsis nco qab, tsis tsaug zog, pw tsaug zog thiab qaug zog, raws plab, cem quav, txawv txav echocardiography, nkees lossis qaug zog, tag nrho Ataxia, tshuav kev tsis txaus, gait tsis meej, nce ntshav siab, tshem tawm, ntau dhau ntawm salivation, kub taub hau, mob siab rau txoj hlab pas, ntuav, poob phaus, ntog, muaj xwm txheej epilepticus.

Ntxiv rau Dravet syndrome, Zogenix tseem tab tom txhim kho Fintepla rau kev kho mob qaug dab peg cuam tshuam nrog Lennox-Gastaut syndrome (LGS). Dravet syndrome thiab LGS yog ob qho tsis tshua muaj thiab feem ntau muaj kev puas tsuaj rau thaum menyuam qaug dab peg, uas muaj cov yam ntxwv ntawm lub hnub nyoog thaum pib, muaj ntau yam mob chua leeg, ntau zaus ntawm qaug dab peg, kev puas tsuaj loj rau kev txawj ntse, thiab kev kho mob nyuaj. Hauv Tebchaws Meskas, Fintepla tau txais txiaj ntsig ntawm Kev Tshuaj Tiv Thaiv Breakthrough Drug (BTD) rau kev kho mob ntawm Dravet Syndrome, Orphan Drug Qualification (ODD) rau kev kho mob ntawm Dravet Syndrome thiab LGS.