Vutrisiran 18-hli Cov Ntaub Ntawv Tshiab: Kev Kho HATTR Amyloidosis Nrog Polyneuropathy!

Alnylam yog ib lub tuam txhab kev lag luam-kev kho RNAi. Tsis ntev los no, lub tuam txhab tshaj tawm 18-hli qhov txiaj ntsig zoo ntawm vutrisiran theem 3 HELIOS-A txoj kev kawm. Vutrisiran yog RNAi txoj kev kho nyob rau hauv txoj kev loj hlob, txhaj tshuaj subcutaneously txhua 3 lub hlis rau kev kho mob ntawm cov neeg laus cov neeg laus uas muaj keeb kwm ntawm transthyretin amyloidosis polyneuropathy (hATTR-PN). Yav dhau los, txoj kev tshawb no tau mus txog qhov kawg ntawm qhov kawg thiab tag nrho cov ntsiab lus thib ob ntawm lub sijhawm 9 lub hlis.

Cov txiaj ntsig tshiab tshaj tawm lub sijhawm no qhia tau hais tias txoj kev tshawb fawb tau mus txog tag nrho cov ntsiab lus thib ob ntsuas ntawm 18-hli lub sijhawm taw qhia: piv nrog cov ntaub ntawv placebo sab nraud ntawm RNAi tshuaj Onpattro (patisiran) Phase 3 APOLLO txoj kev tshawb fawb, kev kho vutrisiran ua rau muaj kev puas tsuaj rau neuropathy (mNIS. +7), lub neej zoo (QOL), kev nrawm nrawm, kev noj zaub mov zoo, thiab kev tsis taus tag nrho tau txheeb xyuas tau zoo dua. Nyob rau hauv cov nqe lus ntawm qhov kawg theem kawg kawg, raws li kev cia siab, pab pawg neeg kho mob vutrisiran tau pom tias tsis muaj qhov tsis zoo ntawm qhov txo qis hauv cov ntshav TTR piv nrog cov pab pawg kho mob Onpattro hauv txoj kev tshawb no.

Tsis tas li ntawd, ntawm 18 lub hlis, cov neeg mob kho nrog vutrisiran kuj tau pom tias muaj kev txhim kho hauv ntau qhov kev tshawb nrhiav qhov kawg. Piv nrog rau cov placebo, cov neeg mob kho nrog vutrisiran pom kev txhim kho hauv plawv biomarker endpoint NT-proBNP (ib qho kev ntsuas ntawm lub plawv siab). Tsis tas li ntawd, piv nrog cov placebo, cov neeg mob kho nrog vutrisiran kuj tau txhim kho nyob rau hauv qee yam echocardiographic tsis. Thaum kawg, nyob rau hauv kev npaj pab pawg ntawm 48 tus neeg mob, kev kho mob vutrisiran tau cuam tshuam nrog kev txhim kho hauv technetium (Technetium) uptake hauv plawv ntawm cov neeg mob feem ntau, muab pov thawj muaj peev xwm rau txo qis plawv amyloid load.

Hauv qhov kev tshawb fawb no, vutrisiran tseem qhia txog kev txhawb nqa kev nyab xeeb thiab kev zam. Alnylam npaj yuav tshaj tawm tag nrho 18-lub hlis cov txiaj ntsig ntawm lub rooj sib tham kho mob thaum ntxov 2022.

siRNA: Silencing cov noob tshwj xeeb, txo cov kev qhia ntau los ntawm 99%

Vutrisiran yog ib qho kev kho mob subcutaneous RNAi nyob rau hauv kev txhim kho rau kev kho mob ntawm ATTR amyloidosis, nrog rau cov tshuaj tua kab mob (hATTR) thiab cov tsiaj qus (wtATTR) amyloidosis. Vutrisiran tuaj yeem tsom thiab ntsiag to tshwj xeeb tus xa xov RNA, thaiv nws ua ntej cov tsiaj qus thiab mutant transthyretin (TTR) cov proteins raug tsim.

Vutrisiran tau txhaj tshuaj subcutaneously txhua lub hlis twg (3 lub hlis), uas tuaj yeem pab txo qis kev tso tawm ntawm TTR amyloid hauv cov ntaub so ntswg, txhawb kev tshem tawm TTR amyloid tso rau hauv cov ntaub so ntswg, thiab muaj peev xwm rov ua haujlwm ntawm cov ntaub so ntswg. vutrisiran siv Alnylam''s Enhanced Stable Chemistry (ESC)-GalNAc conjugated xa platform, uas lub hom phiaj los txhim kho potency thiab siab metabolic stability, tso cai rau tsis tshua muaj kev txhaj tshuaj subcutaneous.

Tam sim no, daim ntawv thov kev lag luam ntawm vutrisiran rau kev kho mob ntawm cov neeg laus nrog hATTR-PN yog nyob rau hauv kev tshuaj xyuas los ntawm US FDA thiab European Union EMA. Hauv Tebchaws Meskas thiab European Union, vutrisiran tau tso cai Orphan Drug Designation (ODD) rau kev kho mob ntawm ATTR amyloidosis, thiab hauv Tebchaws Meskas kuj tau txais txiaj ntsig Fast Track Designation (FTD) rau kev kho mob ntawm cov neeg laus hATTR-PN. Vutrisiran tab tom tab tom saib xyuas qhov tseem ceeb los ntawm US FDA, thiab lub hom phiaj hnub ntawm Tsab Cai Tshuaj Siv Tshuaj (PDUFA) yog lub Plaub Hlis 14, 2022.