US FDA pom zoo Livmarli: thawj cov tshuaj kho mob cholestatic pruritus txuam nrog Alagille syndrome (ALGS)!---1/2

Mirum Pharma yog lub tuam txhab biopharmaceutical mob siab rau tsim kho tshiab los kho cov kab mob siab tsawg. Tsis ntev los no, lub tuam txhab tshaj tawm tias US Food and Drug Administration (FDA) tau pom zoo Livmarli (maralixibat) qhov ncauj tshuaj, uas tau noj ib hnub ib zaug rau kev kho mob cholestasis rau cov neeg mob Alagille syndrome (ALGS) hnub nyoog ≥ 1 xyoo. Khaus khaus. ALGS yog ib yam kab mob uas tsis tshua muaj los ntawm daim siab uas cuam tshuam rau 2000-2500 tus menyuam yaus hauv Tebchaws Meskas.

ALGS yog tshwm sim los ntawm qhov txawv txav ntawm cov kua tsib, uas tuaj yeem ua rau muaj kab mob siab. Cov ducts txawv txav los yog nqaim tuaj yeem ua rau cholestasis, thiab cov kua tsib acids nyob hauv daim siab, uas tuaj yeem ua rau mob thiab ua rau lub siab puas tsuaj, thiab tiv thaiv lub siab ua haujlwm tsis zoo. Lub cholestasis ntawm ALGS yog txuam nrog khaus. Kev khaus khaus yog ib qho ntawm cov tsos mob tshwm sim ntau tshaj plaws uas cuam tshuam nrog ALGS, thiab nws kuj yog ib qho kev qhia ntau tshaj plaws rau kev hloov daim siab rau cov neeg mob ALGS.

Nws yog ib qho tsim nyog hais tias Livmarli yog thawj zaug thiab tsuas yog cov tshuaj pom zoo rau kev kho mob ntawm ALGS cholestatic pruritus, uas yuav ua rau muaj txiaj ntsig hloov pauv hauv cov qauv kev kho mob ntawm ALGS. Livmarli tau pom zoo los ntawm cov txheej txheem tshuaj xyuas qhov tseem ceeb. Yav dhau los, FDA tau tso cai Livmarli qhov tsis tshua muaj tus kab mob rau menyuam yaus (RPDD) thiab kev tsim tshuaj tiv thaiv (BTD) rau kev kho ALGS.

Thaum pom zoo Livmarli, FDA kuj tau tshaj tawm daim ntawv pov thawj ntawm cov kab mob me me (PRV) rau Mirum kom muab nqi zog rau lub tuam txhab rau nws cov txiaj ntsig zoo rau kev tsim cov tshuaj tshiab rau cov kab mob tsawg. Qhov PRV no tuaj yeem txhiv tau rau qhov tseem ceeb ntawm kev tshuaj xyuas cov ntawv thov tshuaj tshiab tom ntej, thiab tuaj yeem muag lossis xa mus.

Cov khoom siv tshuaj nquag ntawm Livmarli qhov ncauj tshuaj yog maralixibat, uas yog qhov ncauj tswj hwm ileal bile acid transporter (IBAT) inhibitor nrog kev nqus tsawg. ALGS yog ib hom kab mob siab tsawg, thiab tsis muaj kev lees paub kev kho mob, yog li muaj qhov xav tau kev kho mob tseem ceeb thiab ceev ceev. Kev pom zoo ntawm Livmarli ntawm kev ua lag luam yuav coj thawj qhov kev xaiv tshuaj kho mob rau tus kab mob loj heev no, yuav muab cov phiaj xwm kev kho mob muaj txiaj ntsig, thiab thaum kawg yuav txo qis qhov xav tau ntawm daim siab hloov pauv.

FDA pom zoo Livmarli, raws li cov kev tshawb fawb ICONIC tseem ceeb thiab 5-xyoo cov ntaub ntawv los ntawm kev txhawb nqa kev tshawb fawb. Cov ntaub ntawv no ua ke ua ib lub cev muaj zog pov thawj rau Livmarli los kho 86 ALGS cov neeg mob. Cov ntaub ntawv los ntawm ICONIC txoj kev tshawb fawb pom tau tias tom qab tau txais kev kho mob Livmarli, cov neeg mob pruritus tau txo qis (p< 0.0001),="" thiab="" lwm="" cov="" cim="" ntawm="" kab="" mob="" siab="" cholestatic="" tau="" txhim="">