Tsoom Fwv Teb Chaws Asmeskas FDA tso cai Sanofi rilzabrutinib khiav ceev kev sib tw!

Sanofi tsis ntev los no tshaj tawm tias Asmeskas Chaw Tswj Xyuas Khoom Noj thiab Tshuaj (FDA) tau tso cai Rilzabrutinib Ceev Nrov Mus Ncig (FTD) rau kev kho mob ntawm kev tiv thaiv kabmob thrombocytopenia (ITP). rilzabrutinib yog qhov ncauj Bruton' s tyrosine kinase (BTK) inhibitor nyob hauv kev tsim. Cov tshuaj muaj lub peev xwm los ua thawj BTK inhibitor rau kev kho mob ntawm ITP. Thaum Lub Kaum Hlis 2018, FDA tau tso cai rau me nyuam ntsuag tshuaj ntsuab (ODD) rau rilzabrutinib los kho ITP. Tom qab cov txiaj ntsig zoo ntawm Kev Tshawb Fawb Theem 1/2, Sanofi tau pib qib 3 qhov kev tshawb fawb soj ntsuam ntsuas rilzabrutinib rau ITP.

Orphan Tshuaj yog hais txog cov tshuaj siv rau kev tiv thaiv, kho mob, thiab kev kuaj mob ntawm cov kab mob uas tsis tshua muaj, thiab cov kab mob uas tsis tshua muaj yog cov lus dav dav rau cov chav kawm muaj cov kab mob uas muaj qhov tshwm sim tsawg kawg, tseem hu ua"" Hauv Tebchaws Meskas, muaj cov kab mob tsawg kawg yog hais txog cov kabmob muaj tsawg dua 200,000 tus neeg mob. Kev txhawb zog rau kev txhim kho cov tshuaj muaj mob tsis tshua muaj ntau yam kev txhawb zog hauv tsev kho mob, xws li cov se nqi ntsig txog nqi kho mob, FDA siv tus nqi txo, thiab kev soj ntsuam kuaj mob. FDA' s kev pabcuam hauv kev tsim cov qauv siv thiab 7-xyoo kev ua lag luam kev zam lub sijhawm rau cov kev pom zoo pom zoo tom qab cov tshuaj pib.

Kev khiav ceev ceev (FTD) lub hom phiaj ua kom nrawm nrawm txhim kho thiab tshuaj xyuas sai sai ntawm cov tshuaj rau cov kab mob loj los daws cov kev kho mob tsis txaus ntseeg nyob hauv thaj chaw tseem ceeb. Tau txais cov peev txheej nrawm nrawm rau cov tshuaj sim txhais tau tias cov tuam txhab kws kho mob tuaj yeem cuam tshuam nrog FDA ntau dua thaum lub sijhawm tshawb fawb thiab kev txhim kho. Tom qab xa tawm daim ntawv thov kev lag luam, lawv tsim nyog rau kev pom zoo nrawm thiab kev tshuaj xyuas ua ntej yog tias lawv ua tau raws cov qauv tsim nyog. Ib qho ntxiv, lawv kuj tsim nyog rau cov nqi tshuaj xyuas.

Tus qauv tshuaj lom neeg ntawm rilzabrutinib (duab los ntawm: pubchem)

Kev txhaj tshuaj tiv thaiv kab mob ntshav siab (ITP) yog ib qho mob tsis tshua muaj thiab txaus autoimmune los ntawm lub cev tiv thaiv kab mob platelet puas tsuaj thiab ua kom cov platelet tsis zoo, ua rau cov ntshav platelet tsawg hauv cov ntshav (thrombocytopenia) thiab yooj yim los ntshav. Thiab muaj qhov cuam tshuam tsis zoo rau lub neej zoo ntawm cov neeg mob. Rau cov neeg mob ITP uas tau txais kev kho mob corticosteroid rau kev rov huam dua los yog kev tawm tsam, tseem muaj qhov tsis tseem ceeb ntawm kev kho mob kom ua tiav tau sai thiab siv tau ntev.

Rilzabrutinib yog qhov ncauj, thim rov qab, covalent, BTK inhibitor uas nyob hauv kev kho mob hauv kev txhim kho rau kev kho kab mob-tiv thaiv kab mob. BTK koom nrog rau qhov tsis muaj zog thiab hloov kho lub cev tsis raug thiab yog lub teeb liab molecule rau cov kab mob tiv thaiv kab mob. Cov ntaub ntawv tshawb fawb qhia tau tias rilzabrutinib tuaj yeem tiv thaiv cov kabmob tiv thaiv kab mob, tshem tawm cov cim kev ua haujlwm ntawm autoantibodies, thiab tiv thaiv kev tsim khoom ntawm cov kab mob autoantibodies tshiab tsis tas yuav tshem tawm cov hlwb B. Rilzabrutinib muaj lub peev xwm los tua cov kab mob pathogenesis thiab tsis tau pom tias hloov pauv platelet sib sau ua ke. Cov chaw kho mob tseem ceeb hauv cov txheej txheem tam sim no tseem tab tom tshawb nrhiav, thiab lawv qhov kev nyab xeeb thiab ua haujlwm tseem tsis tau raug tshuaj xyuas los ntawm ib lub koom haum tswj hwm.

Ntxiv rau ITP, rilzabrutinib yog tam sim no nyob rau Theem 3 kho mob ntawm pemphigus, uas yog lub cev tiv thaiv kab mob sib kis ua rau cov hlwv ntawm cov nqaij mos thiab tawv nqaij. Ntxiv rau, rilzabrutinib kuj tseem tau pib theem 2 kev tshawb fawb soj ntsuam los kho tus kab mob autoimmune IgG4 kab mob.