Fennec's Pedmark (sodium Thiosulfate) Tau Txais Kev Ntsuam Xyuas Ua Ntej Los Ntawm US FDA!

Fennec Pharmaceuticals Inc yog cov kws tshaj lij kev lag luam tau mob siab rau kev txhim kho Pedmark (ib qho kev sib txawv ntawm sodium thiosulfate) rau cov neeg mob kom tiv thaiv ototoxicity tshwm sim los ntawm platinum-muaj tshuaj kho. Tsis ntev los no, lub tuam txhab tshaj tawm tias Asmeskas Chaw Tswj Xyuas Khoom Noj thiab Tshuaj (FDA) tau lees txais Pedmark' s daim ntawv thov kev siv tshuaj tshiab (NDA) thiab tau tso cai kuaj xyuas qhov muaj feem thib tseem ceeb, thaum qhia meej txog cov nqi siv tshuaj rau tus qauv siv (PDUFA) hom phiaj. hnub yog Lub Yim Hli 10, 2020.

Pedmark yog cov tshuaj tshawb nrhiav, uas yog dej-soluble thiol compound, siv los ua cov tshuaj txo cov tshuaj lom neeg, ua haujlwm ntev (4-8 teev) ntawm koob tshuaj Pedmark (16 - 2 0g / m {{4} }) tuaj yeem tiv thaiv cov tsiaj qauv thiab cov neeg mob los ntawm Ototoxicity ntxias los ntawm platinum.

Daim ntawv thov NDA pom zoo Pedmark rau cov neeg mob hauv cheeb tsam, tsis mob ua cov qog nqaij hlav los ntawm 1 lub hlis mus rau tsawg dua ntawm 18 xyoos kom tiv thaiv ototoxicity tshwm sim los ntawm kev kho mob cisplatin. Yog tias tau txais kev pom zoo, Pedmark yuav yog thawj qhov khoom siv txhawm rau tiv thaiv kev hnov ​​lus tsis zoo ntawm cov menyuam yaus hauv cov menyuam yaus. Yav dhau los, FDA tau tso cai rau cov txau yeeb yaj kiab kev ua yeeb tshuaj rau menyuam ntsuag, kev tsim nyog sai, thiab kev muaj peev xwm sib haum tshuaj. Tam sim no, Pedmark tseem tab tom tshuaj xyuas los ntawm European Medicines Agency (EMA).

Cisplatin thiab lwm cov tshuaj pleev platinum yog cov tshuaj siv tshuaj rau me nyuam yaus muaj qhov tsis zoo. Hmoov tsis zoo, kev kho mob platinum tuaj yeem ua rau ototoxicity lossis hnov ​​lus tsis zoo, uas yog tas mus li thiab tsis cuam tshuam, thiab muaj kev phom sij tshwj xeeb rau cov neeg muaj mob cancer thaum yau.

Hauv Tebchaws Asmeskas thiab Tebchaws Europe, kwv yees tias ntau tshaj li 10, 000 cov menyuam yaus tau txais tshuaj pleev platinum-based tshuaj txhua xyoo. Ntawm lawv, 40% -90% ntawm cov menyuam yaus yuav muaj qhov mob tsis zoo, tsis muaj kev cuam tshuam. Qhov tshwm sim ntawm ototoxicity hauv cov menyuam no nyob ntawm qhov koob tshuaj thiab lub sijhawm siv tshuaj kho mob, thiab ntau ntawm lawv yuav tsum muaj lub suab hnov ​​lus ntev ntev. Tam sim no tsis muaj cov tshuaj uas tuaj yeem tiv thaiv qhov tsis hnov ​​lus zoo no, thiab tsuas yog kim, ua haujlwm nyuaj, thiab kev cog lus cochlear kev cog hniav (sab hauv pob ntseg) tau muab pov thawj los muab qee yam txiaj ntsig. Ntawm cov neeg mob ototoxicity ntawm cov theem tseem ceeb ntawm txoj kev loj hlob, cov menyuam mos thiab cov menyuam yaus tsis muaj kev hais lus thiab kev kawm paub, thiab cov menyuam loj dua thiab cov tub ntxhais hluas tsis muaj kev sib raug zoo thiab kev xav thiab kev kawm tiav.

Pedmark tau soj ntsuam los ntawm pab pawg sib koom tes hauv ob theem III kev tshawb fawb soj ntsuam los tiv thaiv thiab txo ototoxicity, suav nrog COG ACCL 0431 kawm thiab SIOPEL 6 kev kawm. Ob txoj kev tshawb fawb tau ua tiav tiav. Txoj kev tshawb COG ACCL 0431 suav txog tsib yam kev mob qog nqaij hlav menyuam yaus uas feem ntau tau txais kev kho mob cisplatin siv tshuaj kho mob hauv zos thiab qhia tawm cov kabmob, suav nrog kev kho kab mob hepatoblastoma, kab mob rau cov qog nqaij hlav, mob ua pob txha pob txha, mob pob txha mos, mob hlwb hlav thiab mob hlwb. Kev soj ntsuam SIOPEL 6 tsuas suav nrog cov neeg mob hepatoblastoma nrog cov qog hauv zos xwb.

Pedmark tuaj yeem tiv thaiv cov hlwb los ntawm tshuaj lom neeg mob lom rau ntau yam. Cov txheej txheem tshuaj tiv thaiv no tsis yog qhov tshwj xeeb. Pedmark tuaj yeem inactivate platinum complex thiab ua rau nws covalently khi rau thiol yam tsis muaj cytotoxicity. Txawm hais tias platinum hauv cov ntshav ncig ploj mus rau qhov muaj txiaj ntsig ntau tom qab 4 teev ntawm platinum tswj, Pedmark' kev tiv thaiv platinum-hais txog kev hnov ​​lus tsis tau zoo tseem tom qab 8 teev ntawm platinum kev tswj hwm. Qhov no qhia tau tias Pedmark tuaj yeem khi rau platinum / protein complex, yog li ua kom nws muaj tshuaj lom tsawg, thiab muaj peev xwm ua rau platinum ua rau hnov ​​lus tsis hnov ​​qab yog vim qhov tso cov protein-khi platinum hauv cochlea.

Lwm cov kev ua haujlwm los ntawm kev ua haujlwm uas Pedmark tiv thaiv ototoxicity yog los ntawm kev tshem tawm ntawm cov pa oxygen reactive (ROS) thiab qhov nce ntawm cov kab mob txo qis (xws li GSH). Hauv pob ntseg sab hauv, lub cochlea tuaj yeem mloog Pedmark hauv perilymph lossis endolymph, thiab tuaj yeem nyob rau hauv zos txhim kho cov tshuaj tiv thaiv ntawm Pedmark tiv thaiv ototoxicity. Yog li, cisplatin-mediated (ntawm ROS) plaub hau cell apoptosis yog qhov cuam tshuam los ntawm cov khoom txo qis ntawm sodium thiosulfate (STS).