Unyango olutsha lofuzo lwezigulane ezine-hemophilia B

A BAMBA isiKhululo sasimahla 8 | eTurboNews | eTN
IAvatar kaLinda Hohnholz
Ibhalwe ngu Linda Hohnholz

I-CSL Behring namhlanje ibhengeze ukuba i-Arhente yeMithi yaseYurophu (EMA) yamkele isicelo sokuGunyanyiswa kweNtengiso (MAA) ye-etranacogene dezaparvovec (EtranaDez) phantsi kwenkqubo yokuvavanya ngokukhawuleza. I-Etranacogene dezaparvovec yintsholongwane yophando enxulumene ne-adeno-associated (AAV5)-based gene therapy elawulwa njengonyango lwexesha elinye kwizigulane ezine-hemophilia B ezopha kakhulu. Ukuba ivunyiwe, i-etranacogene dezaparvovec iya kubonelela abantu abaphila ne-hemophilia B kwi-European Union (EU) kunye ne-European Economic Area (EEA) ngokhetho lokuqala lonyango lonyango lwemfuza olunciphisa kakhulu izinga lokopha konyaka emva kokutofelwa okukodwa. Uvavanyo olukhawulezileyo lunokunciphisa umda wexesha emva kokuba i-MAA yamkelwe ukuba ihlaziywe kwaye ibonelelwe ngemveliso yeyeza xa unyango lulindeleke ukuba lube lunomdla omkhulu kwimpilo yoluntu, ngokukodwa ngokubhekiselele ekusungulweni konyango.

"Njengomgqatswa wokuqala wonyango lwemfuza ye-hemophilia B, lo mba ubalulekileyo wolawulo uzisa i-CSL Behring inyathelo elinye kufutshane nokubonelela ngesithembiso sonyango lwemfuza kuluntu olunengxaki yokopha," utshilo u-Emmanuelle Lecomte Brisset, iNtloko yeMicimbi yoLawulo lweHlabathi kwi-CSL Behring. "Sijonge phambili ekusebenzisaneni nabasemagunyeni ukuzisa amandla enguqu kunyango lwemfuza kubantu abaphila nale meko inqobileyo, ubomi bonke."

I-MAA ixhaswa ziziphumo ezilungileyo ezivela kulingo olubalulekileyo lwe-HOPE-B, olonalingo lonyango lwemfuza lukhulu kwi-hemophilia B ukuza kuthi ga ngoku. Izigulane ze-Hemophilia B ezichazwe njenge-phenotype yokopha kakhulu enyangwa nge-etranacogene dezaparvovec ibonise ukuncitshiswa kweqondo lokuphuma kwegazi ngonyaka (ABR) ngama-64% kwaye yabonisa ukongama kunyango lwe-prophylaxis kwiinyanga ezili-18 emva konyango xa kuthelekiswa neenyanga ezi-6 eziqhutywe kwixesha. Ukongezelela, kukho ukunyuka okuzinzile kunye nokuzinzileyo kumanqanaba omsebenzi we-Factor IX (FIX). I-Etranacogene dezaparvovec yenzelwe ngokukodwa ukwenza isakhono sokuvala igazi esikufutshane-esiqhelekileyo ngokujongana nonobangela osisiseko wale meko: i-F9 gene ephosakeleyo ebangela ukusilela kwi-clotting Factor IX (FIX).

"Ukwamkelwa kwe-etranacogene dezaparvovec ukuba ihlaziywe yi-EMA iqhubela phambili ukuphishekela kwethu ngokuqhubekayo ukuphucula ubomi kunye nentlalontle yabo baphila ne-hemophilia B kunye nezinye izifo ezinqabileyo neziyingozi," utshilo uBill Mezzanotte, uSekela Mongameli oLawulayo, iNtloko ye-R & D kunye IGosa eliPhezulu lezoNyango le-CSL Limited. “Siyazingca ngokusebenza ne-uniQure ukuba phambili kule nkqubela phambili yezesayensi ejolise ekwenzeni i-hemophilia B ibe yinxalenye yesibini yobomi besigulana endaweni yokuba isoloko ixhalabile.”

Uphuhliso lwezonyango lweminyaka emininzi lukhokelwa yi-uniQure (Nasdaq: QURE) kunye nenkxaso-mali yolingo lwezonyango e-United States iye yatshintshela kwi-CSL Behring emva kokufumana kwayo amalungelo ehlabathi okurhweba nge-etranacogene dezaparvovec. I-CSL Behring ikwinkqubo yotshintsho lwenkxaso-mali yolingo lwezonyango kwiManyano yaseYurophu.

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IAvatar kaLinda Hohnholz

Linda Hohnholz

Umhleli oyintloko we eTurboNews esekwe kwi-eTN HQ.

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