I-CSL Behring namuhla imemezele ukuthi i-European Medicines Agency (EMA) yamukele Isicelo Sokugunyaza Ukumaketha (MAA) se-etranacogene dezaparvovec (EtranaDez) ngaphansi kwenqubo yayo yokuhlola esheshisiwe. I-Etranacogene dezaparvovec iwuhlobo lokwelapha olusekelwe ku-adeno-associated virus five (AAV5)-based gene therapy olulawulwa kanye nje kuphela ezigulini ezine-hemophilia B ezopha kakhulu. Uma kuvunyiwe, i-etranacogene dezaparvovec izohlinzeka abantu abaphila ne-hemophilia B e-European Union (EU) kanye ne-European Economic Area (EEA) ngenketho yokwelapha yokwelapha ngofuzo yokuqala ngqa enciphisa kakhulu izinga lokopha konyaka ngemva kokufakwa kanye. Ukuhlola okusheshisiwe kungase kunciphise umugqa wesikhathi uma i-MAA yamukelwe ukuthi ibuyekezwe futhi ihlinzekwe ngomkhiqizo wokwelapha lapho ukwelapha kulindeleke ukuthi kube nentshisekelo enkulu yezempilo yomphakathi, ikakhulukazi ephathelene nokuqanjwa kabusha kokwelapha.
"Njengekhandidethi lokuqala lokwelapha isakhi sofuzo le-hemophilia B, lesi sinyathelo esibalulekile sokulawula sisondeza i-CSL Behring ekuletheni isithembiso sokwelashwa ngofuzo emphakathini onokuphazamiseka okopha," kusho u-Emmanuelle Lecomte Brisset, Inhloko Yezindaba Zokulawulwa Komhlaba e-CSL Behring. "Sibheke ngabomvu ukusebenzisana neziphathimandla ukuze silethe amandla okuguqula izakhi zofuzo kubantu abaphila nalesi simo esicindezelayo, esihlala isikhathi eside."
I-MAA isekelwa yimiphumela emihle evela ocwaningweni olubalulekile lwe-HOPE-B, uhlolo olukhulu kakhulu lokwelashwa ngofuzo ku-hemophilia B kuze kube manje. Iziguli ezine-Hemophilia B ezichazwa njenge-phenotype yokopha kakhulu ephathwe nge-etranacogene dezaparvovec zibonise izinga lokopha elilungisiwe lonyaka (ABR) elincishisiwe ngo-64% futhi zabonisa ukuphakama kunokwelashwa kwe-prophylaxis ezinyangeni ezingu-18 ngemva kokwelashwa uma kuqhathaniswa nokugijima kwezinyanga ezingu-6 esikhathini. Ukwengeza, kube nokukhuphuka okuzinzile nokuqinile kumazinga omsebenzi we-Factor IX (FIX). I-Etranacogene dezaparvovec yayiklanyelwe ngokukhethekile ukwenza ikhono lokujiyisa igazi eliseduze nelivamile libe nokwenzeka ngokubhekana nembangela eyinhloko yalesi simo: isakhi sofuzo se-F9 esinephutha esidala ukuntuleka kokucwilisa i-Factor IX (FIX).
"Ukwamukelwa kwe-etranacogene dezaparvovec ukuze kubuyekezwe yi-EMA kuqhubekisela phambili ukuphishekela kwethu okuqhubekayo ukuthuthukisa izimpilo nokuphila kahle kwalabo abaphila ne-hemophilia B nezinye izifo ezingavamile nezibucayi," kusho uBill Mezzanotte, iPhini Likamongameli Omkhulu, iNhloko ye-R&D kanye Umphathi Omkhulu Wezokwelapha we-CSL Limited. “Siyaziqhenya ngokusebenzisana ne-uniQure ukuba phambili kule ntuthuko yesayensi okuhloswe ngayo ukwenza i-hemophilia B ingxenye yesibili yempilo yesiguli esikhundleni sokukhathazeka okuqhubekayo.”
Ukuthuthukiswa komtholampilo kweminyaka eminingi bekuholwa yi-uniQure (Nasdaq: QURE) futhi uxhaso lwezilingo zomtholampilo e-United States lushintshele ku-CSL Behring ngemuva kokuthola kwayo amalungelo omhlaba okudayisa i-etranacogene dezaparvovec. I-CSL Behring isemkhankasweni wokushintsha uxhaso lwezilingo zomtholampilo e-European Union.
LOKHO ONGAKUTHATHE KULESI SIHLOKO:
- “We are proud to work with uniQure to be at the forefront of this scientific advancement which aims to make hemophilia B a secondary part of a patient’s life instead of a constant concern.
- If approved, etranacogene dezaparvovec will provide people living with hemophilia B in the European Union (EU) and European Economic Area (EEA) with the first-ever gene therapy treatment option that significantly reduces the rate of annual bleeds after a single infusion.
- Accelerated assessment potentially reduces the timeline once the MAA is accepted for review and is provided for a medicinal product when the therapy is expected to be of major public health interest, particularly pertaining to therapeutic innovation.
