Qfitlia (Fitusiran), ib qho tshiab siRNA-raws li kev kho mob haemophilia tau txais kev pom zoo los ntawm FDA. Nws yog ib tug me me cuam tshuam RNA (siRNA) raws li kev kho mob uas cuam tshuam nrog ntuj anticoagulants xws li antithrombin (AT) thiab cov ntaub so ntswg txoj kev inhibitor (TFPI). Nws khi rau AT mRNA hauv daim siab thiab thaiv AT txhais lus yog li txo cov antithrombin thiab txhim kho thrombin tiam. Nws yog siv raws li kev txhaj tshuaj subcutaneous pib ib zaug txhua ob lub hlis. Cov koob tshuaj thiab zaus ntawm kev txhaj tshuaj yog hloov kho siv INNOVANCE Antithrombin tus khub kuaj mob uas ua kom muaj kev ua haujlwm antithrombin hauv lub hom phiaj. Qhov koob tshuaj tas tsis pom zoo. Txoj kev kho tshiab no tseem ceeb rau cov neeg mob vim tias nws tau siv tsawg dua li lwm cov kev xaiv uas twb muaj lawm.
Qfitlia (fitusiran) tau txais kev pom zoo hauv Tebchaws Meskas (ntawm 28 Lub Peb Hlis 2025) rau kev tiv thaiv niaj hnub los tiv thaiv lossis txo qhov zaus ntawm cov ntshav tawm hauv cov neeg laus thiab menyuam yaus hnub nyoog 12 xyoos thiab laus dua nrog hemophilia A lossis hemophilia B, nrog lossis tsis muaj qhov VIII lossis IX inhibitors (neutralizing antibodies). Txoj kev kho tshiab yog qhov tseem ceeb vim tias nws tau siv tsawg dua (pib ib zaug txhua ob lub hlis) dua li lwm cov kev xaiv uas twb muaj lawm.
Kev mob ntshav los ntshav hauv haemophilia yog tshwm sim los ntawm qhov tsis txaus ntawm cov ntshav txhaws. Haemophilia A yog tshwm sim los ntawm qhov tsis muaj peev xwm ntawm cov ntshav txhaws VIII (FVIII), thaum haemophilia B yog vim qhov qis ntawm qhov IX (FIX). Tsis muaj qhov ua tau zoo XI yog lub luag haujlwm rau haemophilia C. Cov xwm txheej no tau kho los ntawm kev ua lag luam npaj cov khoom lag luam los yog cov khoom tsis yog khoom raws li kev hloov pauv ntawm qhov ploj lawm.
Octocog alfa (Advate), uas yog 'genetically engineered siv DNA technology' version of clotting factor VIII, feem ntau yog siv rau kev tiv thaiv raws li qhov xav tau kev kho mob ntawm haemophilia A. Rau haemophilia B, nonacog alfa (BeneFix), uas. yog ib qho kev tsim qauv ntawm cov khoom siv IX feem ntau siv.
Hympavzi (marstacimab-hncq) tau pom zoo hauv Tebchaws Meskas (Lub Kaum Hli 11 2024) thiab hauv EU (hnub tim 19 Lub Cuaj Hli 2024) ua cov tshuaj tshiab rau kev tiv thaiv los ntshav hauv cov tib neeg uas muaj hemophilia A lossis hemophilia B. Nws yog tib neeg monoclonal antibody uas tiv thaiv "cov ntshav tawm qhov tshwm sim los ntawm cov protein ntau tshwm sim los ntawm cov tshuaj tiv thaiv kab mob. pathway inhibitor" thiab txo nws cov kev ua haujlwm anticoagulation yog li ua kom cov thrombin nce ntxiv. Qhov no yog thawj zaug, tsis yog qhov tseem ceeb thiab kev kho mob ib zaug ib lim piam rau hemophilia B.
Lwm cov tshuaj tiv thaiv kab mob monoclonal, Concizumab (Alhemo) tau pom zoo hauv Tebchaws Meskas (ntawm 20 Lub Kaum Ob Hlis 2024) thiab hauv EU (hnub tim 16 Lub Kaum Ob Hlis 2024) rau kev tiv thaiv los ntshav hauv cov neeg mob hemophilia A nrog cov txiaj ntsig VIII inhibitors lossis hemophilia B nrog IX inhibitors. Qee cov neeg mob haemophilia ntawm "cov tshuaj tiv thaiv kab mob ntshav" rau kev kho lawv cov kev mob los ntshav tsim cov tshuaj tiv thaiv (tawm tsam cov tshuaj tiv thaiv kab mob). Cov tshuaj tiv thaiv tau tsim inhibit qhov kev ua ntawm "cov tshuaj tiv thaiv kab mob" ua rau lawv tsis zoo. Concizumab (Alhemo), muab tshuaj txhua hnub raws li kev txhaj tshuaj subcutaneous, yog txhais tau tias kho tus mob no uas ib txwm tau kho los ntawm kev tiv thaiv kab mob los ntawm kev txhaj tshuaj txhua hnub ntawm cov ntshav txhaws.
Thaum mpavzi (marstacimab-hncq) thiab Concizumab (Alhemo) yog monoclonal antibodies, kev kho tshiab Qfitlia (fitusiran) yog ib qho me me cuam tshuam RNA (siRNA) raws li kev kho mob uas cuam tshuam nrog ntuj anticoagulants xws li antithrombin (AT) thiab cov ntaub so ntswg txoj kev inhibitor (TFPI). Nws khi rau AT mRNA hauv daim siab thiab thaiv AT txhais lus yog li txo cov tshuaj antithrombin thiab txhim kho thrombin tiam.
Qfitlia (fitusiran) yog siv raws li kev txhaj tshuaj subcutaneous pib ib zaug txhua ob lub hlis.
Cov koob tshuaj thiab zaus ntawm kev txhaj tshuaj yog hloov kho siv INNOVANCE Antithrombin tus khub kuaj mob uas ua kom muaj kev ua haujlwm antithrombin hauv lub hom phiaj. Qhov koob tshuaj tas tsis pom zoo. Txawm li cas los xij, qhov kev kho tshiab no tseem ceeb rau cov neeg mob vim tias nws tau siv tsawg dua li lwm cov kev xaiv uas twb muaj lawm.
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References:
- FDA Xov Xwm Tshaj Tawm - FDA pom zoo kho tshiab rau Hemophilia A lossis B, nrog lossis tsis muaj Factor Inhibitors. Tshaj tawm 28 Lub Peb Hlis 2025. Muaj nyob ntawm https://www.fda.gov/news-events/press-announcements/fda-approves-novel-treatment-hemophilia-or-b-or-without-factor-inhibitors
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Tshooj cai:
- Concizumab (Alhemo) rau Hemophilia A lossis B nrog Inhibitors (29 Hlis ntuj nqeg 2024)
- Hympavzi (marstacimab): Kev Kho Tshiab rau Hemophilia (12 Lub Kaum Hli 2024)
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, a novel siRNA-based treatment for haemophilia has received FDA approval. It is a small interfering RNA (siRNA) based therapeutic that interfere with natural anticoagulants such as antithrombin (AT) and tissue factor pathway inhibitor (TFPI). It binds to AT mRNA in the liver and blocks AT translation thereby reduce antithrombin and improve thrombin generation. It is administered as subcutaneous injection starting once every two months. The dose and frequency of injections are adjusted using the INNOVANCE Antithrombin companion diagnostic that ensure antithrombin activity in the target range. The fixed dose is not approved. The new treatment is significant for patients because it is administered less frequently than other existing options.){kind=link}