Ayvakyt (avapritinib) tau pom zoo los ntawm European Union thiab nkag ua ntej kev tshuaj xyuas hauv Suav teb!

CStone' s tus khub Blueprint Tshuaj yog lub tuam txhab ua lag luam tshuaj tsom tsom rau kev kho mob, ua kom tsis tshua muaj kab mob thiab tshuaj tiv thaiv kab mob cancer. Tsis ntev los no, Blueprint Tshuaj tau tshaj tawm tias Cov European Commission (EC) tau tso cai rau kev lag luam tawm rau cov tshuaj anticancer ntawm lub hom phiaj Ayvakyt (avapritinib) raws li kev ntsuas tshuaj rau kev kho platelet-derived growth factor receptor alpha (PDGFRA) cov noob neeg laus uas muaj kev tiv thaiv tsis tau lossis metastatic hloov plab coj khaub ncaws qog (GIST) nrog D842V hloov.

Nws tsim nyog teev tias Ayvakyt yog thawj qhov kev kho mob tsom rau hauv European Union rau Gist cov neeg mob nrog PDGFRA D842V hloov chaw. Cov tshuaj ua haujlwm ntawm Ayvakyt yog avapritinib, kinase inhibitor uas tau pom zoo los ntawm Asmeskas FDA rau Lub Ib Hlis xyoo no nyob rau hauv lub npe Ayvakit rau kev kho mob tsis muaj kev hloov pauv hauv exon 18 ntawm PDGFRA noob (suav nrog PDGFRA D842V hloov) Cov neeg laus cov neeg mob nrog kev sib deev lossis kev sib deev GIST. Nws tsim nyog teev tias avapritinib yog thawj qhov kev kho kom raug pom zoo rau GIST thiab thawj cov tshuaj nrog cov haujlwm siab tiv thaiv GIST nrog kev hloov pauv hauv exon 18 ntawm PDGFRA noob. Hauv nruab nrab lub Tsib Hlis xyoo no, avapritinib' s txoj kev kho plaub rau GIST raug tsis lees paub los ntawm FDA.

Thaum Lub Rau Hli 2018, CStone Cov Tshuaj Kho Mob tau mus txog kev sib koom tes tshwj xeeb thiab kev pom zoo nrog Blueprint Medicines, thiab tau txais kev txhim kho thiab ua lag luam txoj cai ntawm peb cov neeg sib tw nrog cov avapritinib hauv Greater China (Mainland, Hong Kong, Macau, Taiwan). Thaum Lub Peb Hlis Ntuj xyoo no, CStone Cov Tshuaj Kho Mob tau tshaj tawm qhov kev xa daim ntawv thov tshuaj tshiab rau avapritinib hauv Taiwan, Suav.

Lub Plaub Hlis xyoo no, CStone Cov Tshuaj Kho Mob tau tshaj tawm tias Lub Tsev Haujlwm Saib Xyuas Kev Kho Mob Hauv Tebchaws (NMPA) yuav lees txais cov ntawv thov tshuaj tshiab avapritinib, suav txog 2 qhov ntsuas, uas yog: (1) Rau kev kho mob ntawm cov neeg mob nqa platelet-derived growth factor receptor alpha ( PDGFRA) Cov neeg mob loj uas tsis tuaj yeem tiv thaiv lossis metastatic GIST nrog kev hloov pauv hauv exon 18 (suav nrog PDGFRA D842V kev hloov pauv); (2) Cov neeg laus nrog cov plaub-kab tsis tuaj yeem pom lossis metastatic GIST. Nyob rau hauv Lub Xya Hli xyoo no, avapritinib tau pom zoo kev soj ntsuam los ntawm NMPA.

GIST (Cov duab los ntawm: southfloridasurgicaloncology.com)

Kev pom zoo los ntawm EU yog saib raws li cov ntaub ntawv siv tau los ntawm Theem I NAVIGATOR kev soj ntsuam sim, thiab kev nyab xeeb los ntawm NAVIGATOR thiab Theem III VOYAGER txoj kev sim. Cov ntaub ntawv qhia tau hais tias nyob rau hauv cov neeg mob PDGFRA D842V mutant GIST uas tau txais yav tas los lossis tsis tau txais kev kho mob, kev kho avapritinib tau qhia cov lus teb tob thiab siv tau ntev. Hauv 38 cov neeg mob nrog PDGFRA D842V mutant GIST, qhov pib koob yog 300mg lossis 400mg ib zaug ib hnub, qhov lus teb tag nrho (ORR) ntawm kev kho mob Ayvakyt yog 95% (95CI: 82.3-99.4), thiab tus lej teb tiav (CR) yog 13% Qhov nruab nrab ntawm kev teb rov qab (DOR) yog 22.1 lub hlis (95% CI: 14.1-tsis pom tseeb), qhov nruab nrab ntawm kev vam meej tsis muaj sia nyob (PFS) yog 24 lub hlis, thiab qhov nruab nrab muaj sia nyob tag nrho (OS) tseem tsis tau mus txog. Cov ntaub ntawv ua tau zoo no tau tshaj tawm hauv ESMO lub rooj sib tham rau lub hlis no. Hauv kev sim tshuaj, qhov tshwm sim tsis zoo tshaj plaws (≥20%) yog xeev siab, qaug zog, ua kom yuag, ua kom tsis muaj zog, txhawm rau ntsej muag, mob ntshav qab zib, raws plab, zawv plab, ntuav, peripheral edema, muaj kua muag ntau dua, tsis qab los noj mov thiab nco qab.

GIST yog qhov muaj caj ces kho lub plab zom mov plab, thiab nws qhov PDGFRA D842V hloov tau cuam tshuam nrog tus neeg mob tsis tshua muaj kab mob. Kev rov qab kawm txog PDGFRA D842V mutant GIST cov neeg mob tau qhia tias ORR ntawm kev kho mob nrog imatinib (imatinib, tus qauv thawj-kab GIST txoj kev kho) yog 0%. ESMO cov lus qhia pom zoo suav nrog kev hloov pauv kom paub zoo raws li kev coj ua hauv kev txheeb xyuas GIST.

Sebastian Bauer, tus neeg tshawb nrhiav txog NAVIGATOR txoj kev tshawb nrhiav thiab kws kho mob ntawm lub plawv Essenside Cancer Center tau hais tias: "Keeb kwm, tsis muaj kev kho tau muab kev cia siab rau cov neeg mob PDGFRA D842V mutant GIST. Ayvakyt yog rau cov neeg mob GIST nrog kev hloov pauv no. Lub NAVIGATOR mus sib hais paub tseeb tias yuav luag txhua tus neeg mob uas muaj PDGFRA D842V mutant GIST tau txo cov qog thiab cov lus teb siv tshuaj tau ntev. Cov neeg mob no muaj lub sijhawm ntev dua li xav tau raws li cov txiaj ntsig yav dhau los. Thiab feem ntau tus neeg mob tuaj yeem tiv taus cov kev mob tshwm sim zoo. Nrog kev pom zoo no, kev hloov mus kuaj ua ntej kev kho mob thawj zaug yog qhov tseem ceeb tshaj li yav dhau los, kom cov neeg mob PDGFRA D842V mutant GIST tuaj yeem pib kho nrog AYVAKYT, uas yog txoj kev kho kom zoo rau hom mob qog xwb."

Avapritinib tuaj yeem xaiv thiab tawm tsam KIT thiab PDGFRA mutant kinases. Cov tshuaj yog ib hom kuv inhibitor tsim los tsom rau kev ua si lub zog kinase conformation; tag nrho oncogenic kinases teeb liab los ntawm qhov kev hloov pauv no. Avapritinib tau pom tias muaj ntau yam kev cuam tshuam los tiv thaiv kev cuam tshuam rau GIST-cuam tshuam rau KIT thiab PDGFRA kev hloov pauv, suav nrog kev ua haujlwm muaj zog tiv thaiv kev voj kev hloov pauv ntsig txog kev tawm tsam kev pom zoo tam sim no kev kho mob.

Piv nrog kev pom zoo ntau lub kinase inhibitors, avapritinib yog qhov muaj kev xaiv ntau rau KIT thiab PDGFRA dua li lwm cov kinases. Ib qho ntxiv, avapritinib yog qhov tsim tshwj xeeb los xaiv khi thiab khi D816 hloov pauv KIT, uas yog ib tus neeg tsav tsheb muaj kabmob feem ntau kwv yees li 95% ntawm cov neeg mob uas muaj mastocytosis (SM). Kev tshawb nrhiav pom tau pom tias avapritinib tuaj yeem cuam tshuam KIT D816V nrog cov sub-nanomolar potency thiab muaj qhov tsawg ntawm kev ua haujlwm.

Ntxiv rau GIST, Blueprint Cov Tshuaj Kho Mob kuj tseem txhim kho avapritinib rau kev kho mob ntawm SM siab, inert thiab smoky SM. Yav dhau los, Asmeskas FDA tau tso cai rau avapritinib dhau los ua ib qho tshuaj muaj txiaj ntsig rau kev kho mob ntawm ob qhov taw qhia: (1) kev kho mob tsis muaj kev tiv thaiv thiab metastatic GIST nqa PDGFRα D842V hloov; (2) kev kho mob ntawm SM siab heev, suav nrog kev txhoj puab heev SM (ASM), Cov mob Subtypes xws li SM (SM-AHN) thiab mast cell leukemia (MCL) nrog cov hlav ntshav sib thooj.