Sanofi Cablivi se apwouve pa Kanada pou tretman an nan akeri thrombotic tronbositopenik purpurea (aTTP)!

Sanofi denyeman te anonse ke sante Kanada te apwouve Nanobody Dwog Cablivi la (caplacizumab) nan yon pwosesis revizyon priyorite, konbine avek echanj plasma ak terapi immunosuppressive pou akeri thrombotic tronbositopenik purpurea (aTTP) tretman nan pasyan granmoun. aTTP se yon maladi san ra ki lakoz tronbozi nan veso san nan tout ko a, ki ka lakoz domaj nan ogan vital epi yo ka mennen nan konplikasyon fatal. Cablivi se premye Dwog la ki apwouve pa Kanada a espesyalman trete aTTP.

Cablivi se premye a apwouve Nanobody Dwog ak premye Dwog la espesyalman trete aTTP. Cablivi ka anpeche fomasyon an nan microthrombi (san boul) nan ko a, fe pasyan refe pi vit, redwi kek nan konplikasyon ki asosye ak aTTP, epi redwi pousantaj la nan repetition maladi.

Cablivi te devlope pa Beljik biotechnologie Ablynx, ak Sanofi achte Ablynx pou $4.800.000.000 nan mwa Janvye 2018. Cablivi te apwouve nan Inyon Ewopeyen an nan mwa Out 2018 ak nan Etazini nan mwa fevriye 2019. Nan Etazini ak Inyon Ewopeyen an, Cablivi te akode ofelen kalifikasyon Dwog pou tretman an nan aTTP, e li te akode kalifikasyon vit-tras ak kalifikasyon revizyon priyorite nan Etazini yo. Cablivi se kounye a yon pati nan Mondyal la nan biznis enfimye pwofesyonel nan Sanofi nan Sanofi maladi ki ra Genzan nan ak tout franchiz.

Dokte Katerina Pavenski, yon hematologist nan lopital St. Michael a, te di: "aTTP se yon maladi devaste ke, si ou pa trete efektivman, ka mennen nan lanmo. Malgre ke opsyon tretman aktyel yo te amelyore pronostik pasyan an, konplikasyon toujou rive mennen nan lanmo. Nan syans klinik, Cablivi ka akselere fe gerizon ak soulajman maladi, epi yo gen yon enpak enpotan sou konplikasyon maladi. Apwobasyon sa a ap ba nou yon tretman enpotan opsyon pou ede amelyore pronostik pasyan yo avek aTTP. "

aTTP se yon bagay ki menase, otoiminite ki baze sou maladi coagulation ki karakterize pa fomasyon an nan gwo kantite lajan nan boul san nan veso nan ti nan tout ko a, ki mennen nan grav thrombocytopenia (ki ba konte segonde) ak microangiopathy emoliz anemi (pet nan globil wouj yo akoz destriksyon emoliz), tisi ischemia (limite san nan kek oganis) ak domaj ogan vaste, espesyalman sevo a ak ke Malgre li resevwa aktyel swen estanda regimens, ki gen ladan yon sel fwa-chak jou plasma (PEX) ak immunosuppressive terapi, pasyan toujou fe fas a gwo risk konplikasyon thrombotic, repetition, ak lanmo, ak aTTP epizod yo toujou asosye ak jiska 20% motalite a ki gen rapo, ki pi lanmo rive nan jou 30 nan dyagnostik.

Aktif engredyan pharmaceutique la nan Cablivi se caplacizumab, ki se yon bivalent ki pisan ak Selektif anti-an dlo fakte (vWF) nanobody, ki ka bloke enteraksyon an ant ultra gwo vWF multise (ULvWF) ak plaket, vize plaket agrégation yo ak fomasyon ki vin apre ak akimilasyon nan microclots gen yon Efe imedya. Nan pasyan aTTP, boul sa a ti san ka lakoz thrombocytopenia grav, tisi ischemia, ak malfonksyonman ogan. Efe imedyat Cablivi a (Efe imedyat), pandan yo ap demantelman pwosesis la maladi kache, ka pwoteje pasyan aTTP soti nan manifestasyon klinik nan maladi.

Apwobasyon nan Cablivi ki baze sou done ki soti nan faz III etid klinik HERCULES (NCT02553317). Etid la se te yon randomized, doub-aveg, etid plasebo-kontwole, ak 145 pasyan granmoun ak aTTP te enskri. Nan etid la, pasyan yo te owaza asiyen nan Cablivi oswa plasebo pandan yo ap resevwa estanda regimens swen (plasma echanj ak immunosuppressive terapi).

Rezilta yo nan etid la te montre ke: (1) an tem de endpoint prensipal la, Cablivi konbine ak estanda swen regimens siyifikativman pi kout tan an pou fe yon konte de nomalizasyon konpare ak plasebo + swen estanda regimens (p = 0,01); nan nenpot pwen tan espesifik pandan peryod la etid, gwoup tretman Cablivi te 1,55 fwa plis chans reyalize nomal tandans konte pase gwoup plasebo. (2) pandan peryod la etid tout antye, Cablivi konbine avek estanda swen regimens siyifikativman redwi aTTP ki gen rapo ak lanmo, aTTP repetition, oswa omwen yon gwo evenman thromboembolic nan 74% (p<0.001) compared="" to="" placebo="" +="" standard="" care="" regimens.="" (3)="" during="" the="" entire="" study="" period,="" compared="" with="" placebo="" +="" standard="" care="" regimen,="" cablivi="" combined="" with="" standard="" care="" regimen="" significantly="" reduced="" the="" number="" of="" attp="" recurrences="" by="" 67%="" (p=""><0.001). (4)="" during="" the="" entire="" study="" period,="" 0="" refractory="" diseases="" occurred="" in="" the="" cablivi="" treatment="" group="" and="" 3="" refractory="" diseases="" occurred="" in="" the="" placebo="" group,="" although="" no="" statistically="" significant="" difference="" was="" reached="" (p="0.06)." (5)="" compared="" with="" the="" placebo="" group,="" the="" patients="" in="" the="" cablivi="" treatment="" group="" normalized="" the="" three="" organ="" damage="" markers="" (lactate="" dehydrogenase,="" cardiac="" troponin="" i,="" and="" serum="" creatinine)="" earlier="" (due="" to="" the="" stratified="" statistical="" test,="" p="" no="" significance="" was="" detected).="" (6)="" compared="" with="" the="" placebo="" group,="" the="" use="" of="" plasma="" exchange="" in="" patients="" in="" the="" cablivi="" treatment="" group="" has="" a="" clinically="" significant="" reduction="" (mean="" 5.8="" days="" vs="" 9.4="" days,="" a="" 38%="" reduction),="" and="" in="" the="" intensive="" care="" unit="" (a="" 65%="" reduction)="" and="" hospitals="" (a="" reduction="" 31%)="" the="" residence="" time="" is="" shorter.="" (7)="" the="" safety="" of="" cablivi="" is="" consistent="" with="" previous="" reports="" and="" is="" consistent="" with="" its="" mechanism="" of="" action,="" including="" increased="" risk="" of="" bleeding;="" the="" most="" common="" bleeding-related="" adverse="" events="" are="" nose="" bleeding="" and="" gum="">