Reblozyl siv rau kev nthuav qhia hauv Tebchaws Meskas thiab Tebchaws Europe: kev kho mob ntawm kev tso ntshav-ywj siab β-thalassemia!

Bristol-Myers Squibb (BMS) tsis ntev los no tau tshaj tawm tias US Food and Drug Administration (FDA) tau lees txais Reblozyl's (luspatercept-aamt) supplementary biological product license application (sBLA) thiab tau tso cai saib xyuas qhov tseem ceeb: cov tshuaj yog ib tug thawj-hauv-chav kawm) Red blood cell maturation agent (EMA), siv rau non-transfusion dependent (NTD) β-thalassemia (β-thalassemia) cov neeg laus los kho anemia. FDA tau teeb tsa lub hom phiaj hnub rau sBLA's Prescription Drug User Fee Act (PDUFA) raws li lub Peb Hlis 27, 2022. Tsis tas li ntawd, European Medicines Agency (EMA) tau txais daim ntawv thov hloov Class II rau Reblozyl rau kev kho mob ntshav qab zib hauv cov neeg laus. Cov neeg mob uas muaj NTDβ thalassemia.

NTD beta thalassemia yog ib lo lus siv los piav qhia cov neeg mob uas tsis tas yuav tsum tau txhaj tshuaj rau lub neej ntev ntawm cov qe ntshav liab (RBC) kom muaj sia nyob, txawm hais tias lawv yuav xav tau qee zaus lossis me ntsis, feem ntau nyob rau lub sijhawm teem tseg. Cov neeg mob uas muaj NTD beta thalassemia yuav muaj kev mob ntshav qab zib thiab cov hlau ntau dhau, uas tuaj yeem ua rau muaj teeb meem hauv chaw kho mob, thiab xav tau kev kho mob sai. Cov ntaub ntawv soj ntsuam kuaj pom tau hais tias nyob rau hauv cov neeg laus uas muaj NTD β thalassemia, tsis hais txog theem pib hemoglobin, Reblozyl tuaj yeem nce tus neeg mob' qib hemoglobin thiab txhim kho tus neeg mob' lub neej zoo.

Reblozyl yog Ameslikas tsim los ntawm BMS thiab Acceleron Pharmaceuticals. Tom qab Merck tsis ntev los no ua tiav $ 11.5 nphom kev tau txais Acceleron, Reblozyl tam sim no koom ua ke tsim thiab ua lag luam los ntawm BMS thiab Merck. Reblozyl yog thawj tus neeg sawv cev ntawm cov ntshav liab kom loj hlob (EMA) los siv rau kev kho mob β-thalassemia thiab qis heev / qis / nruab nrab muaj feem pheej hmoo myelodysplastic syndrome (MSD) ntsig txog ntshav qab zib nrog kev tswj hwm kev pom zoo. Ntawm cov pab pawg neeg mob tsim nyog, Reblozyl sawv cev rau pawg kho mob tseem ceeb. Nws yuav tsum tau muab sau tseg tias nyob rau hauv cov neeg mob uas yuav tsum tau kho anemia tam sim ntawd, Reblozyl tsis haum raws li ib tug hloov rau cov ntshav liab.

Cov khoom siv tshuaj nquag ntawm Reblozyl yog luspatercept, uas yog thawj-hauv-chav qe ntshav liab maturation tus neeg sawv cev (EMA) uas tuaj yeem tswj cov kev loj hlob ntawm cov qe ntshav liab lig. Luspatercept yog ib qho fusion fusion protein, uas yog tsim los ntawm fusion ntawm Fc domain ntawm tib neeg IgG1 thiab extracellular domain ntawm Activin IIB receptor (ActRIIB). Raws li ib tug ligand ntxiab, nws muaj peev xwm tswj tau lig RBC maturation los ntawm lub hom phiaj khi. Kev hloov pauv kev loj hlob (TGF)-β superfamily ntawm cov ligands tshwj xeeb txo qhov kev ua haujlwm ntawm Smad2/3 signaling pathway, txhim kho cov cim ntawm RBC tsis raug, txhawb kev loj hlob ntawm cov qe ntshav liab lig RBC, thiab nce qib hemoglobin.

Reblozyl sBLA thiab Class II hloov daim ntawv thov yog ua raws li kev nyab xeeb thiab kev ua tau zoo ntawm qhov pivotal Phase 2 BEYOND txoj kev kawm. Txoj kev tshawb no tau ua nyob rau hauv cov neeg laus nrog NTD beta thalassemia thiab soj ntsuam Reblozyl ua ke nrog Kev Pabcuam Zoo Tshaj Plaws (BSC). Cov ntaub ntawv tshaj tawm thaum Lub Rau Hli 2021 tau pom tias 77.1% ntawm cov neeg mob hauv Reblozyl+BSC pab pawg kho mob tau ua tiav qib hemoglobin nce siab (≥1.0g/dL), thaum cov placebo+BSC pawg yog 0%. Tsis tas li ntawd, piv nrog cov placebo pab pawg, cov neeg mob hauv pawg Reblozyl kuj tau tshaj tawm cov txiaj ntsig tau zoo dua.

Dr. Noah Berkowitz, tus lwm thawj coj loj ntawm kev txhim kho hematology ntawm Bristol-Myers Squibb, tau hais tias: "Cov neeg mob uas tsis muaj kev hloov pauv-dependent beta thalassemia yuav tsis xav tau kev txhaj tshuaj rau lub neej kom muaj sia nyob, tab sis lawv xav tau kev kho mob zoo vim tias lawv ntsib ib tus mob. series ntawm chronic anemia thiab hlau overload. Cov teeb meem kho mob tshwm sim los ntawm Reblozyl. Reblozyl yog ib qho kev kho mob tseem ceeb uas tau pom zoo los ntawm ntau lub teb chaws (xws li Tebchaws Meskas thiab European Union) rau kev kho mob ntshav qab zib muaj feem xyuam rau β-thalassemia thiab kev pheej hmoo tsawg myelodysplastic syndrome. Peb thiab Merck tau cog lus tias yuav txuas ntxiv mus rau kev ua haujlwm kho mob ntawm Reblozyl Project, thiab tos ntsoov yuav ua haujlwm ze nrog FDA thaum lub sijhawm tshuaj xyuas kom coj tau cov kev kho mob tshiab rau pawg neeg mob uas tsis tau txais kev pab no."