Bristol-Myers Squibb Inhibitor Mavacamten Teb Chaws Asmeskas Kev Ntsuam Xyuas Txuas Ntxiv Los ntawm 3 Lub Hlis!

Bristol-Myers Squibb (BMS) tsis ntev los no tshaj tawm tias US Food and Drug Administration (FDA) tau txuas ntxiv mavacamten' Daim Ntawv Thov Tshuaj Tshiab (NDA) lub sijhawm tshuaj xyuas los ntawm 3 lub hlis. Tus tshiab" Tsab Cai Tus Neeg Siv Tshuaj Txhaum Cai" (PDUFA) hnub tim yog 2022 Lub Plaub Hlis 28th. Cov tshuaj yog ib qho tshiab, qhov ncauj, myocardial myosin allosteric modulator siv los kho cov tsos mob obstructive hypertrophic cardiomyopathy (oHCM), kab mob plawv uas muaj qhov tshwm sim siab.

Thaum Lub Xya Hli 2020, US FDA tau tso cai mavacamten ib qho kev xaiv tshuaj rau oHCM. Yog tias pom zoo, mavacamten yuav dhau los ua thawj myosin inhibitor los kho oHCM. Tam sim no, daim ntawv thov kev tso cai ua lag luam (MAA) ntawm mavacamten rau kev kho mob ntawm cov tsos mob oHCM kuj tseem raug tshuaj xyuas los ntawm European Medicines Agency (EMA).

Raws li BMS tshaj tawm, FDA tau ceeb toom rau lub tuam txhab thaum Lub Kaum Ib Hlis 18, 2021 kom txuas ntxiv PDUFA hnub kom tso cai sijhawm txaus los tshuaj xyuas cov ntaub ntawv tshiab ntsig txog cov phiaj xwm kev ntsuas kev pheej hmoo txo ​​qis (REMS). Qhov kev pabcuam REMS tau suav nrog hauv thawj daim ntawv thov ntawm mavacamten. FDA tsis tau thov cov ntaub ntawv ntxiv lossis kev tshawb fawb.

Samit Hirawat, MD, Tus Lwm Thawj Coj Thawj Coj thiab Tus Thawj Saib Xyuas Kev Kho Mob ntawm Bristol-Myers Squibb tau hais tias:" Peb muaj kev ntseeg siab hauv mavacamten. Hauv qhov pivotal EXPLORER-HCM kev sim, qhov no pioneering plawv myosin inhibitor tau siv los kho cov tsos mob oHCM cov neeg mob. , Cov xwm txheej ua haujlwm thiab lub neej zoo tau pom muaj kev txhim kho tseem ceeb hauv kev kho mob. Peb tos ntsoov yuav txuas ntxiv ua haujlwm nrog FDA kom coj cov tshuaj tseem ceeb no rau cov neeg mob."

mavacamten yog thawj-hauv-chav kawm, qhov ncauj, myosin allosteric inhibitor, siv los kho cov kab mob uas muaj ntau lub plawv contraction thiab impaired diastolic filling raws li sab hauv. Mavacamten ntseeg tau tias yuav txo qis myocardial contractility los ntawm inhibiting kev tsim ntawm ntau dhau myosin-actin cross-bridges. Kev tsim ntawm ntau dhau ntawm myosin-actin hla choj tuaj yeem ua rau ntau dhau myocardial contraction, sab laug ventricular hypertrophy thiab txo kev ua raws. Hauv kev tshawb fawb soj ntsuam thiab preclinical, mavacamten tseem qhia txog biomarkers uas txo cov phab ntsa hauv lub plawv, txo qis myocardial contraction ntau, thiab ua rau kev ua raws li diastolic.

mavacamten yog thawj zaug tsim los kho cov tsos mob obstructive hypertrophic cardiomyopathy (oHCM). Raws li nws cov txheej txheem ntawm kev ua thiab cov pov thawj ntawm kev kho mob, mavacamten tseem tab tom kawm kho mob rau kev kho mob ntawm cov tsos mob uas tsis yog-obstructive hypertrophic cardiomyopathy (HCM) thiab lub plawv tsis ua hauj lwm nrog khaws cia ejection feem (HFpEF).

mavacamten chemical structure

Ob leeg mavacamten NDA thiab MAA yog raws li cov txiaj ntsig ntawm Theem 3 EXPLORER-HCM tseem ceeb mus sib hais. Kev sim tau ua nyob rau hauv cov neeg mob oHCM cov tsos mob thiab piv mavacamten nrog cov placebo. Cov txiaj ntsig tau kuaj pom tau tias mavacamten tau qhia txog kev kho mob muaj zog, nrog rau kev kho mob tseem ceeb hauv cov tsos mob, kev ua haujlwm zoo thiab lub neej zoo, nrog rau nws lub peev xwm los daws cov kab mob sab laug ventricular outflow obstruction. Hauv txoj kev tshawb fawb EXPLORER-HCM, tag nrho cov ntsiab lus tseem ceeb thiab theem nrab tau mus txog qhov tseem ceeb ntawm cov ntaub ntawv.

mavacamten (MYK-461) yog tsim los ntawm MyoKardia. Thaum Lub Kaum Hli 5, 2020, Bristol-Myers Squibb tshaj tawm tias nws yuav tau MyoKardia rau USD 13.1 billion nyiaj ntsuab thiab 60% tus nqi them. Thaum Lub Kaum Ib Hlis 17, 2020, Bristol-Myers Squibb tshaj tawm tias qhov tau txais MyoKardia tau ua tiav tiav. Qhov kev nrhiav tau no yog Bristol-Myers Squibb' qhov kev lag luam loj thib ob tom qab US $ 74 billion tau los ntawm Celgene hauv 2019.

Nws yog ib nqi sau cia hais tias lub yim hli ntuj 11, 2020, LianBio, uas yog incubated los ntawm lub peev lub tuam txhab Perceptive Advisors, tau raug tsim. Nyob rau tib hnub, nws tshaj tawm ob txoj kev koom tes tseem ceeb. Ib qho yog los qhia BridgeBio Pharma' cov khoom xa mus rau Tuam Tshoj, thiab lwm qhov Qhov project yog los qhia myoKardia' s mavecamten rau Tuam Tshoj.

Bristol-Myers Squibb muaj kev cia siab rau mavacamten. Thaum nws tau txais MyoKardia, lub tuam txhab tshaj tawm tias mavacamten yuav dhau los ua cov tshuaj pioneering rau kev kho HCM. Kev lag luam kuj zoo siab heev txog kev lag luam zeem muag ntawm mavacamten. Thaum Lub Kaum Ob Hlis 2020, Ntsuam Xyuas Vantage, lub koom haum tshawb fawb kev lag luam tshuaj, tau tso tawm" Sab saum toj 10 Cov Tshuaj Tshiab nrog Cov Peev Xwm Ua Lag Luam hauv 2021". Nyob rau hauv daim ntawv no, mavacamten ranked peb. Ntsuam xyuas Vantage kwv yees tias mavacamten' lub ntiaj teb kev muag khoom hauv 2026 yuav ncav cuag US $ 2 billion.