Qfitlia (Fitusiran), sabon magani na tushen siRNA don haemophilia ya sami amincewar FDA. Yana ne mai ƙananan RNA mai shiga tsakani (siRNA) tushen therapeutic cewa tsoma baki tare da maganin rigakafi na halitta irin su antithrombin (AT) da mai hana hanyar ƙwayar nama (TFPI). Yana ɗaure zuwa AT mRNA a cikin hanta kuma yana toshe fassarar AT don haka rage antithrombin kuma inganta thrombin tsara. Ana gudanar da shi azaman allurar subcutaneous farawa sau ɗaya kowane wata biyu. Ana daidaita kashi da mitar allura ta amfani da binciken abokin hulɗa na INNOVANCE Antithrombin wanda ke tabbatar da aikin antithrombin a cikin kewayon manufa. Ba a yarda da ƙayyadadden kashi ba. Sabuwar magani yana da mahimmanci ga marasa lafiya saboda ana gudanar da shi ƙasa da yawa fiye da sauran zaɓuɓɓukan da ake dasu.
An amince da Qfitlia (fitusiran) a cikin Amurka (a kan 28 Maris 2025) don rigakafin yau da kullun don hanawa ko rage yawan yawan zubar jini a cikin manya da yara masu shekaru 12 da haihuwa tare da hemophilia A ko hemophilia B, tare da ko ba tare da factor VIII ko IX inhibitors (tsakanin rigakafi). Sabuwar maganin yana da mahimmanci saboda ana gudanar da shi ƙasa da ƙasa (farawa sau ɗaya kowane wata biyu) fiye da sauran zaɓuɓɓukan da ake dasu.
Rashin zubar jini a cikin haemophilia yana faruwa ne saboda rashin isassun abubuwan da ke damun jini. Haemophilia A yana faruwa ne saboda rashi na nau'in clotting factor VIII (FVIII), yayin da haemophilia B ya kasance saboda ƙananan matakan factor IX (FIX). Rashin aikin factor XI yana da alhakin haemophilia C. Ana kula da waɗannan yanayi ta hanyar shigar da kayan aikin clotting na kasuwanci ko wani samfurin da ba shi da mahimmanci a matsayin maye gurbin aikin da ya ɓace.
Octocog alfa (Advate), wanda shine 'wanda aka kirkira ta hanyar amfani da fasahar DNA' nau'in nau'in clotting factor VIII, ana amfani dashi akai-akai don rigakafi da kuma kan buƙatar magani na haemophilia A. Don haemophilia B, nonacog alfa (BeneFix), wanda sigar injiniya ce ta clotting factor IX da ake yawan amfani da ita.
An amince da Hympavzi (marstacimab-hncq) a cikin Amurka (a kan 11 Oktoba 2024) kuma a cikin EU (a kan 19 Satumba 2024) a matsayin sabon magani don rigakafin cututtukan jini a cikin mutanen da ke da hemophilia A ko hemophilia B. Yana da rigakafin mutum monoclonal wanda ke hana ɓarna ko zubar da jini ta hanyar yin niyya ta hanyar rage ƙwayar cuta ta hanyar rage ƙwayar cuta ta hanyar rage ƙwayar cuta ta hanyar hana ƙwayoyin cuta. Ayyukan anticoagulation ta haka yana ƙara yawan thrombin. Wannan shi ne na farko, marasa dalili kuma magani sau ɗaya a mako-mako don hemophilia B.
Wani antibody monoclonal, Concizumab (Alhemo) an yarda da shi a cikin Amurka (a ranar 20 ga Disamba 2024) kuma a cikin EU (a ranar 16 ga Disamba 2024) don rigakafin cututtukan jini a cikin marasa lafiya tare da hemophilia A tare da masu hana VIII ko hemophilia B tare da masu hana IX factor. Wasu marasa lafiyar haemophilia a kan "magungunan abubuwan da ke haifar da clotting" don kula da yanayin rashin jinin jini suna haifar da ƙwayoyin rigakafi (a kan magungunan clotting factor). Kwayoyin rigakafin da aka kafa suna hana aikin "magungunan abubuwan da ke haifar da jini" yana sa su zama marasa tasiri. Concizumab (Alhemo), wanda ake yi wa yau da kullun azaman allurar subcutaneous, ana nufin magance wannan yanayin da aka saba yi ta hanyar haifar da juriya na rigakafi ta hanyar alluran yau da kullun na abubuwan clotting.
Duk da yake mpavzi (marstacimab-hncq) da Concizumab (Alhemo) sune ƙwayoyin rigakafi na monoclonal, sabon jiyya Qfitlia (fitusiran) ƙaramin maganin RNA ne (siRNA) mai tsangwama wanda ke tsoma baki tare da magungunan ƙwayoyin cuta na halitta irin su antithrombin (AT) da mai hana hanyar hanyar nama (TFPI). Yana ɗaure zuwa AT mRNA a cikin hanta kuma yana toshe fassarar AT don haka rage antithrombin kuma inganta haɓakar thrombin.
Ana yin Qfitlia (fitusiran) azaman allurar subcutaneous farawa sau ɗaya kowane wata biyu.
Ana daidaita kashi da mitar allura ta amfani da binciken abokin hulɗa na INNOVANCE Antithrombin wanda ke tabbatar da aikin antithrombin a cikin kewayon manufa. Ba a yarda da ƙayyadadden kashi ba. Duk da haka, sabon magani yana da mahimmanci ga marasa lafiya saboda ana gudanar da shi ƙasa da yawa fiye da sauran zaɓuɓɓukan da ake da su.
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References:
- Sakin Labaran FDA - FDA ta Amince da Maganin Novel don Hemophilia A ko B, tare da ko ba tare da Masu hana Factor ba. An buga 28 Maris 2025. Akwai a https://www.fda.gov/news-events/press-announcements/fda-approves-novel-treatment-hemophilia-or-b-or-without-factor-inhibitors
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Related article:
- Concizumab (Alhemo) don Hemophilia A ko B tare da Masu hanawa (29 Disamba 2024)
- Hympavzi (marstacimab): Sabon Magani ga Hemophilia (12 Oktoba 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}