Fa'afouina I'uga o Su'ega Falema'i i Togafitiga o Fa'ama'i Fabry

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Protalix BioTherapeutics, Inc. i aso nei na faʻasalalau faʻaiʻuga mulimuli mai le BRIGHT Phase III faʻataʻitaʻiga faʻataʻitaʻiga suʻesuʻeina pegunigalsidase alfa (PRX 102) mo togafitiga faʻamaʻi Fabry. O faʻaiʻuga o loʻo faʻaalia ai o togafitiga faʻatasi ma le 2 mg / kg o le PRX-102 o loʻo faʻatinoina e le intravenous (IV) infusion i vaiaso uma e fa na faʻafeiloaʻi lelei, ma o le faʻamaʻi Fabry na suʻesuʻeina e ala i le faʻatusatusaina o le glomerular filtration rate (eGFR) slope ma le plasma lyso-Gb3 concentration sa mautu.  

"Matou te fiafia e faʻasoa faʻamatalaga mulimuli mai le suʻesuʻega BRIGHT, o se faʻailoga taua i le alualu i luma o la matou polokalame faʻapitoa PRX-102," o le tala lea a Dror Bashan, le Peresetene ma le Ofisa Sili o le Protalix. "O le maua o nei faʻamatalaga mo le toe iloiloina e le US Food and Drug Administration, le European Medicines Agency ma isi tulafono faatonutonu o se isi laʻasaga agai i luma i le faʻatagaina o le PRX-102 e avea o se suiga lelei mo tagata matutua Fabry gasegase i le 1 mg masani. \kg ta'i lua vaiaso fa'apea fo'i ma le 2 mg\kg fa'atonuga i vaiaso ta'i fa.”

PRX-102 o se laau toto-fa'aalia toe tu'ufa'atasia, PEGylated, α galactosidase feso'ota'i feso'ota'i O se sui fa'atau oloa. O le BRIGHT Phase III faʻataʻitaʻiga faʻataʻitaʻiga (NCT03180840) o se multicenter, multinational open-label, suʻesuʻega suiga ua fuafuaina e iloilo ai le saogalemu, aoga ma pharmacokinetics o togafitiga faʻatasi ma le 2 mg / kg o PRX-102 faʻatautaia i vaiaso taʻitasi mo 52 vaiaso ( e tusa ma le 14 infusions). O le suʻesuʻega na lesitalaina ai le 30 tagata matutua maʻi faʻamaʻi Fabry (24 tane ma le 6 fafine) faʻatasi ma le umi (SD) tausaga o 40.5 (11.3) tausaga, e amata mai i le 19 i le 58 tausaga, oe na mauaina muamua se faʻataʻitaʻiga o le enzyme replacement therapy (ERT) mo le itiiti ifo. tolu tausaga i luga o se fua faʻamautu e faia i vaiaso uma e lua (agalsidase alfa - Replagal® poʻo agalsidase beta - Fabrazyme®). O fa'ailoga masani fa'ama'i Fabry i le fa'asologa o le acroparesthesia, le fa'apalepale o le vevela, angiokeratomas ma le hypohydrosis.

O tagata gasegase uma na auai i le suʻesuʻega na maua a itiiti ifo ma le tasi le fua o le PRX-102, ma 29 tagata gasegase na maeʻa le suʻesuʻega. Mai nei 29 maʻi, 28 na maua le faʻatonuga o le 2 mg / kg o PRX-102 i vaiaso uma e fa i le suʻesuʻega atoa, ae tasi le tagata maʻi na suia i le 1 mg / kg o PRX-102 i vaiaso uma e lua i le faʻasalalauga i le 11th infusion. . O le tasi tagata maʻi na alu ese mai le suʻesuʻega ina ua maeʻa le tui muamua ona o se faʻalavelave tau taavale.

Muamua infusions o PRX-102 na faʻatinoina i lalo o tulaga faʻatonutonu i le nofoaga suʻesuʻe. Faʻavae i luga o faʻataʻitaʻiga muamua i le suʻesuʻega suʻesuʻega, na mafai e tagata gasegase ona maua a latou PRX-102 infusions i se fale tausi fale ina ua malilie le Suʻesuʻega ma le Sponsor Medical Monitor e saogalemu le faia.

I le aotelega, o le 33 o le 182 le aofaʻi o togafitiga-faʻalavelave faʻalavelave faʻafuaseʻi (TEAEs) lipotia i le iva (30.0%) maʻi na manatu e fesoʻotaʻi ma togafitiga; sa agamalu uma pe feololo i le mamafa ma o le tele na foia i le faaiuga o le suʻesuʻega. E leai ni TEAE tuga pe ogaoga e feso'ota'i ma togafitiga ma e leai ni TEAE na i'u i le oti po'o le fa'amavaeina o su'esu'ega. O TEAE e feso'ota'i ma togafitiga, 27 na fa'aoso fa'a'ai (IRRs) ma o le vaega na totoe o mea e tasi o le manava tatā, erythema, vaivai, fa'ama'i pei o le influenza, fa'atuputeleina o le urine protein/creatinine ratio, ma le urine lelei mo sela pa'epa'e. O le 27 IRRs na lipotia i le lima (16.7%) maʻi, tane uma. O IRR uma na tutupu i le taimi o le faʻamaʻiina poʻo totonu o le lua itula i le maeʻa ai o le tui; e leai ni mea na tutupu na fa'amauina i le va o le lua ma le 24 itula talu ona tu'uina. E leai se tasi o tagata mama'i e leai ni vaila'au fa'ama'i (ADAs) i le su'esu'ega na atia'e ADA fa'aoso togafitiga ina ua mae'a le suiga ile PRX-102 togafitiga.

O suʻesuʻega o suʻesuʻega e faʻaalia ai o plasma lyso Gb3 concentrations na tumau pea i le taimi o suʻesuʻega ma se suiga (± SE) o le 3.01 nM (0.94) mai le laina faʻavae (19.36 nM ± 3.35) i le Vaiaso 52 (22.23 ± 3.60 nM). Ole fua ole eGFR atoa na mautu ile vaitaimi ole 52 vaiaso ole togafitiga, fa'atasi ai ma se suiga mai le laina ole 1.27 mL/min/1.73 m2(1.39). Mean (SE) eGFR slope, i le faaiuga o le suʻesuʻega, mo le faitau aofaʻi atoa, e 2.92 (1.05) mL / min / 1.73m2 / tausaga e faʻaalia ai le mautu.

"Matou te fiafia e faʻasalalau faʻaiʻuga mulimuli mai le BRIGHT Phase III faʻataʻitaʻiga faʻataʻitaʻiga ma e fia faʻafetai i tagata suʻesuʻe suʻesuʻe, Fabry faʻamaʻi, ma o latou aiga na tuutoina o latou taimi ma taumafaiga i lenei suʻesuʻega taua," o le tala lea a Giacomo Chiesi, ulu o Chiesi Fa'ama'i Fa'alelalolagi. "E faʻavae i luga o nei faʻamatalaga ma suʻesuʻega faʻapitoa faʻapitoa, matou te talitonu PRX-102 atonu o se togafitiga fou taua mo tagata mamaʻi o loʻo mauaina nei faʻamaʻi ERT i vaiaso uma, ma matou te tulimatai atu i le faʻalauteleina o la matou galuega i le lalolagi atoa ina ia maua faʻatagaga faʻatulafonoina. vave i le mafai ma tuʻuina atu avanoa i le faʻamaʻi Fabry community."

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Avatar a Linda Hohnholz

Linda Hohnholz

Faatonu sili mo eTurboNews fa'avae ile eTN HQ.

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