Qfitlia (Fitusiran), itọju ti o da lori siRNA aramada fun haemophilia ti gba ifọwọsi FDA. O ni a RNA kekere interfering (siRNA) orisun mba pe dabaru pẹlu awọn anticoagulants adayeba gẹgẹbi antithrombin (AT) ati inhibitor ifosiwewe tissu (TFPI). O sopọ mọ AT mRNA ninu ẹdọ ati awọn bulọọki AT itumọ nitorina dinku antithrombin ati mu ilọsiwaju ti thrombin. O ti wa ni abojuto bi abẹrẹ subcutaneous ti o bere lẹẹkan gbogbo osu meji. Iwọn ati igbohunsafẹfẹ ti awọn abẹrẹ jẹ atunṣe nipa lilo iwadii ẹlẹgbẹ INNOVANCE Antithrombin ẹlẹgbẹ ti o rii daju iṣẹ antithrombin ni ibiti ibi-afẹde. Iwọn ti o wa titi ko fọwọsi. Itọju tuntun jẹ pataki fun awọn alaisan nitori pe o nṣakoso ni igbagbogbo ju awọn aṣayan miiran ti o wa tẹlẹ lọ.
Qfitlia (fitusiran) ti fọwọsi ni AMẸRIKA (ni Oṣu Kẹta Ọjọ 28, Ọdun 2025) fun prophylaxis igbagbogbo lati ṣe idiwọ tabi dinku igbohunsafẹfẹ ti awọn iṣẹlẹ ẹjẹ ninu agbalagba ati awọn ọmọde ti ọjọ-ori ọdun 12 ati agbalagba pẹlu hemophilia A tabi hemophilia B, pẹlu tabi laisi ifosiwewe VIII tabi IX inhibitors (aiṣedeede awọn ọlọjẹ). Itọju tuntun jẹ pataki nitori pe o nṣakoso ni igbagbogbo (bẹrẹ lẹẹkan ni gbogbo oṣu meji) ju awọn aṣayan miiran ti o wa tẹlẹ lọ.
Awọn rudurudu ẹjẹ ni haemophilia jẹ nitori aipe awọn ifosiwewe didi. Haemophilia A jẹ nitori aipe ifosiwewe didi VIII (FVIII), lakoko ti haemophilia B jẹ nitori awọn ipele kekere ti ifosiwewe IX (FIX). Aini ifosiwewe iṣẹ-ṣiṣe XI jẹ iduro fun haemophilia C. Awọn ipo wọnyi ni a ṣe itọju nipasẹ fifisilẹ ifosiwewe didi ti a pese sile ni iṣowo tabi ọja ti kii ṣe ifosiwewe bi rirọpo iṣẹ ṣiṣe ti ifosiwewe ti o padanu.
Octocog alfa (Advate), eyiti o jẹ ‘ẹda apilẹṣẹ nipa lilo imọ-ẹrọ DNA’ ẹya ti didi ifosiwewe VIII, ni a lo nigbagbogbo fun idena ati itọju ibeere ti haemophilia A. Fun haemophilia B, nonacog alfa (BeneFix), eyiti jẹ ẹya ẹlẹrọ version of didi ifosiwewe IX ti wa ni commonly lo.
Hympavzi (marstacimab-hncq) ni a fọwọsi ni AMẸRIKA (ni Oṣu Kẹwa Ọjọ 11, Ọdun 2024) ati ni EU (ni Oṣu Kẹsan Ọjọ 19, Ọdun 2024) gẹgẹbi oogun tuntun fun idena awọn iṣẹlẹ ẹjẹ ni awọn ẹni-kọọkan ti o ni hemophilia A tabi hemophilia B. O jẹ egboogi monoclonal ti eniyan ti o ṣe idiwọ awọn iṣẹlẹ ẹjẹ tabi idinamọ awọn iṣẹlẹ ẹjẹ nipa ibi-afẹde ipa ọna ọlọjẹ ti a npe ni “proteincosuuse factor” iṣẹ ṣiṣe anticoagulation rẹ nitorinaa n pọ si iye thrombin. Eyi jẹ akọkọ, ti kii ṣe ifosiwewe ati itọju lẹẹkan-ọsẹ fun hemophilia B.
Antibody monoclonal miiran, Concizumab (Alhemo) ni a fọwọsi ni AMẸRIKA (ni ọjọ 20 Oṣu kejila ọdun 2024) ati ni EU (ni ọjọ 16 Oṣu kejila ọdun 2024) fun idena awọn iṣẹlẹ ẹjẹ ni awọn alaisan ti o ni hemophilia A pẹlu awọn inhibitors VIII tabi hemophilia B pẹlu awọn inhibitors IX. Diẹ ninu awọn alaisan haemophilia lori “awọn oogun ifosiwewe didi” fun itọju ipo rudurudu ẹjẹ wọn ṣe agbekalẹ awọn aporo ara (lodi si awọn oogun ifosiwewe didi). Awọn apo-ara ti a ṣẹda ṣe dojuti iṣe ti “awọn oogun ifosiwewe didi” ti o jẹ ki wọn ko munadoko. Concizumab (Alhemo), ti a nṣakoso lojoojumọ gẹgẹbi abẹrẹ abẹlẹ, ni itumọ lati ṣe itọju ipo yii eyiti a ti ṣe itọju ni aṣa nipasẹ fifamọra ifarada ajẹsara nipasẹ awọn abẹrẹ ojoojumọ ti awọn okunfa didi.
Lakoko ti mpavzi (marstacimab-hncq) ati Concizumab (Alhemo) jẹ awọn ajẹsara monoclonal, itọju tuntun Qfitlia (fitusiran) jẹ itọju ailera kekere ti o da lori RNA (siRNA) ti o dabaru pẹlu awọn anticoagulants adayeba gẹgẹbi antithrombin (AT) ati oludena ipa ọna ti ara (TFPI). O sopọ mọ AT mRNA ninu ẹdọ ati awọn bulọọki AT itumọ nitorina o dinku antithrombin ati ilọsiwaju iran thrombin.
Qfitlia (fitusiran) ni a nṣakoso bi abẹrẹ abẹ-ara ti o bẹrẹ lẹẹkan ni gbogbo oṣu meji.
Iwọn ati igbohunsafẹfẹ ti awọn abẹrẹ jẹ atunṣe nipa lilo iwadii ẹlẹgbẹ INNOVANCE Antithrombin ẹlẹgbẹ ti o rii daju iṣẹ antithrombin ni ibiti ibi-afẹde. Iwọn ti o wa titi ko fọwọsi. Laibikita eyi, itọju titun jẹ pataki fun awọn alaisan nitori pe o nṣakoso ni igbagbogbo ju awọn aṣayan miiran ti o wa tẹlẹ lọ.
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To jo:
- Itusilẹ Awọn iroyin FDA – FDA fọwọsi Itọju aramada fun Hemophilia A tabi B, pẹlu tabi laisi Awọn inhibitors Factor. Pipa 28 Oṣù 2025. Wa ni https://www.fda.gov/news-events/press-announcements/fda-approves-novel-treatment-hemophilia-or-b-or-without-factor-inhibitors
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Oro ti o ni ibatan:
- Concizumab (Alhemo) fun Hemophilia A tabi B pẹlu awọn inhibitors (29 Oṣu kejila ọdun 2024)
- Hympavzi (marstacimab): Itọju Tuntun fun Hemophilia (12 Oṣu Kẹwa 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}