I-Qfitlia (Fitusiran), inoveli yonyango olusekwe kwi-siRNA kwi-haemophilia ifumene imvume ye-FDA. Yona yi iRNA encinci ephazamisayo (siRNA) yonyango esekelwe ukuba ukuphazamisa i-anticoagulants yendalo efana ne-antithrombin (AT) kunye ne-tissue factor pathway inhibitor (TFPI). Ibophelela kwi-AT mRNA esibindi kwaye ivimbela ukuguqulelwa kwe-AT ngaloo ndlela inciphisa i-antithrombin kunye ukuphucula ukuveliswa kwe-thrombin. Isetyenziswa njengenaliti engaphantsi kwesikhumba eqala kanye kwiinyanga ezimbini. I-dose kunye nokuphindaphindiweyo kweenaliti zihlengahlengiswa kusetyenziswa i-INNOVANCE Antithrombin companion diagnostic eqinisekisa umsebenzi we-antithrombin kuluhlu olujoliswe kuyo. Idosi emiselweyo ayivunywanga. Unyango olutsha lubalulekile kwizigulane kuba lusetyenziswa kancinci kunezinye iindlela ezikhoyo.
I-Qfitlia (fitusiran) ivunyiwe e-USA (ngomhla we-28 kaMatshi 2025) kwiprophylaxis yesiqhelo ukukhusela okanye ukunciphisa ukuphindaphinda kweziganeko zokopha kubantu abadala kunye nabantwana abaneminyaka eyi-12 ubudala nangaphezulu kunye ne-hemophilia A okanye i-hemophilia B, kunye okanye ngaphandle kwe-factor VIII okanye i-IX inhibitors (i-antibodies engathathi hlangothi). Unyango olutsha lubalulekile kuba lusetyenziswa rhoqo (ukuqala kanye kwiinyanga ezimbini) kunezinye iindlela ezikhoyo.
Iziphazamiso zokopha kwi-haemophilia zibangelwa kukunganeli kwezinto zokujiyisa. IHaemophilia A yenziwa ngenxa yokunqongophala kwe-clotting factor VIII (FVIII), ngelixa i-haemophilia B ibangelwa kumanqanaba aphantsi e-factor IX (FIX). Ukunqongophala kwe-Factor factor XI inoxanduva lwe-haemophilia C. Ezi meko zinyangwa ngokufafaza i-clotting factor elungiselelwe urhwebo okanye i-non-factor product njenge-active replacement of the missing factor.
I-Octocog alfa (Advate), 'eyenziwe ngokwemfuza kusetyenziswa ubuchwepheshe be-DNA' inguqulelo ye-clotting factor VIII, idla ngokusetyenziselwa uthintelo kunye nonyango oluyimfuneko lwehaemophilia A. Kwi-haemophilia B, nonacog alfa (BeneFix), ethi luluguqulelo lobunjineli lwe-clotting factor IX eqhele ukusetyenziswa.
I-Hympavzi (i-marstacimab-hncq) yamkelwa e-USA (ngomhla we-11 ngo-Oktobha 2024) nakwi-EU (ngomhla we-19 Septemba 2024) njengechiza elitsha lokuthintela iziqephu zokopha kubantu abane-hemophilia A okanye i-hemophilia B. Yi-antibody ye-monoclonal yomntu ethintela iziqephu zokopha ngokujolisa kwi-protein ye-anticotisegu inhibitor” kwaye yehlisa umsebenzi wayo we-anticoagulation ngokwandisa umthamo we-thrombin. Olu lolokuqala, olungeyonto yonyango kunye nonyango lwe-hemophilia B kanye ngeveki.
Enye i-monoclonal antibody, i-Concizumab (Alhemo) yamkelwa e-USA (ngomhla wama-20 kuDisemba 2024) nakwi-EU (ngomhla we-16 kaDisemba 2024) ukuze kuthintelwe iziqendu ukopha kwizigulane ezine-hemophilia A ene-factor VIII inhibitors okanye i-hemophilia B ene-factor IX inhibitors. Ezinye izigulana ezinehaemophilia “kumayeza ee-clotting factor” kunyango lwemeko yazo yokopha ziye ziphuhlise izilwa-buhlungu (ezichasene namayeza e-clotting factor). Izilwa-buhlungu ezenziweyo zithintela isenzo “samayeza ee-clotting factor” ziwenza angasebenzi kakuhle. I-Concizumab (Alhemo), elawulwa yonke imihla njengenaliti engaphantsi kwesikhumba, yenzelwe ukunyanga le meko ebikhe yanyangwa ngokunyamezelwa kwamajoni omzimba ngokusebenzisa inaliti yemihla ngemihla yezinto zokujiyisa.
Ngelixa i-mpavzi (i-marstacimab-hncq) kunye ne-Concizumab (i-Alhemo) ziyi-antibodies ze-monoclonal, unyango olutsha lwe-Qfitlia (fitusiran) luyi-RNA ephazamisayo encinci (i-siRNA) esekelwe kunyango ephazamisana ne-anticoagulants yendalo efana ne-antithrombin (AT) kunye ne-tissue factor pathway inhibitor (TFPI). Ibophelela kwi-AT mRNA esibindini kwaye ivimba ukuguqulelwa kwe-AT ngaloo ndlela inciphisa i-antithrombin kunye nokuphucula ukuveliswa kwe-thrombin.
I-Qfitlia (fitusiran) isetyenziswa njengenaliti engaphantsi kwesikhumba eqala kanye kwiinyanga ezimbini.
I-dose kunye nokuphindaphindiweyo kweenaliti zihlengahlengiswa kusetyenziswa i-INNOVANCE Antithrombin companion diagnostic eqinisekisa umsebenzi we-antithrombin kuluhlu olujoliswe kuyo. Idosi emiselweyo ayivunywanga. Ngaphandle koku, unyango olutsha lubalulekile kwizigulane kuba lusetyenziswa kancinci kunezinye iindlela ezikhoyo.
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Iingxelo:
- Ukukhutshwa kweNdaba ye-FDA-i-FDA ivuma iNoveli yoNyango lweHemophilia A okanye i-B, ene-Factor Inhibitors okanye ngaphandle kwayo. Ithunyelwe nge-28 Matshi 2025. Ifumaneka apha https://www.fda.gov/news-events/press-announcements/fda-approves-novel-treatment-hemophilia-or-b-or-without-factor-inhibitors
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Inqaku elidibeneyo:
- Concizumab (Alhemo) yeHemophilia A okanye B ene-Inhibitors (Ngomhla we-29 kuDisemba 2024)
- I-Hympavzi (marstacimab): Unyango olutsha lwe-Hemophilia (12 Okthobha 2024)
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