Avapritinib Yuav Pom Zoo Hauv EU Hauv Lub Cuaj Hli, CStone Cov Tshuaj Kho Mob tau Xa Daim Ntawv Thov Tuam Tshoj!

Blueprint Cov Tshuaj Kho Mob, tus khub ntawm CStone, yog ib lub tuam txhab ua tshuaj tsom tsom rau kev kho mob hauv caj ces, mob tsis tshua muaj thiab tshuaj tiv thaiv kab mob cancer. Tsis ntev los no, Blueprint Tshuaj tau tshaj tawm tias Pawg Neeg Saib Xyuas Kev Noj Tshuaj hauv Tebchaws Europe (EMA) rau Cov Khoom Siv Tshuaj rau Cov Neeg Siv (CHMP) tau tshaj tawm cov lus pom zoo hais txog kev pom zoo ntawm cov tshuaj anticancer avapritinib ua ib qho kev tswj hwm rau Kev Kho Mob ntawm cov neeg mob uas tsis muaj peev xwm tiv thaiv lossis muaj mob plab zom mov. stromal qog (GIST) nqa platelet-derived kev loj hlob yam receptor alpha (PDGFRA) noob D842V hloov chaw.

Tam sim no, cov tswv yim zoo ntawm CHMP yuav rov saib xyuas los ntawm European Commission (EC), uas yuav tsum ua qhov kev txiav txim siab zaum kawg thaum kawg ntawm lub Cuaj Hli. Yog tias tau txais kev pom zoo, avapritinib yuav dhau los ua thawj qhov kev kho mob hauv EU rau cov neeg mob GIST nrog PDGFRA D842V kev hloov pauv, thiab yuav ua lag luam nyob hauv hom npe nrov Ayvakyt.

Avapritinib yog kinase inhibitor uas tau pom zoo los ntawm Asmeskas FDA rau Lub Ib Hlis xyoo no nyob rau hauv cov npe khoom lag luam Ayvakit rau kev kho mob tsis muaj kev tiv thaiv lossis metastatic GIST cov neeg laus laus nrog PDGFRA noob exon 18 hloov (suav nrog PDGFRA D842V kev hloov pauv). Nws tsim nyog teev tias avapritinib yog thawj qhov kev xav tau kev 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.

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 kev lag luam txoj cai ntawm peb tus neeg sib tw ua yeeb tshuaj suav nrog avapritinib hauv Greater China (Mainland, Hong Kong, Macau, Taiwan). Hauv lub Peb Hlis ntawm lub 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 ntawm lub xyoo no, CStone Cov Tshuaj Kho Mob tshaj tawm tias National Medical khoom Administration (NMPA) yuav lees txais cov ntawv thov rau cov tshuaj tshiab avapritinib, yuav tsum vov 2 qhov taw qhia, uas yog: (1) Rau kev kho mob ntawm cov neeg mob nqa platelet-derived kev loj hlob 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. Nov yog CStone' s thawj cov npe tshuaj tshiab tau lees txais los ntawm NMPA, khij ib kauj ruam tseem ceeb hauv tuam txhab' s kev hloov pauv lag luam.

CHMP cov tswv yim zoo yog txiav txim siab raws li cov ntaub ntawv siv tau los ntawm Theem I NAVIGATOR kev soj ntsuam kab mob, thiab kev nyab xeeb los ntawm NAVIGATOR thiab Theem III VOYAGER txoj kev sim. Cov ntaub ntawv qhia tau hais tias kev kho avapritinib ntawm PDGFRA D842V mutant GIST cov neeg mob tau qhia cov lus teb tob, ntev ntev, nrog cov lus teb tag nrho (ORR) ntawm 88% (95CI: 76-95), nruab nrab ntawm cov lus teb (DOR) tsis tau mus txog, thiab thev taus ntev Kev sib deev. Cov ntaub ntawv no tau tshaj tawm hauv The Lancet Oncology thaum Lub Rau Hli 29, 2020.

Dr. Andy Boral, Tus Thawj Saib Xyuas Kws Kho Mob ntawm Blueprint Tshuaj, tau hais tias: "Avapritinib tau pom tias tsis muaj kev ua haujlwm hauv chaw kho mob hauv PDGFRA D842V mutant GIST cov neeg mob. Xws li cov neeg mob tau muaj kev cia siab tsis zoo. Cov tswv yim zoo ntawm CHMP niaj hnub no nthuav qhia peb cov kev siv zog los txhim kho cov khoom no. Qhov kev nce qib loj hauv cov program kawm nce mus rau EU cov hom phiaj. Rau PDGFRA D842V mutant GIST cov neeg mob, avapritinib lub hom phiaj yuav hloov pauv tus qauv kev kho los ntawm kev xaiv yam tsis haum rau oncogenic tsav ntawm kev tsis kam mus rau kev kho mob GIST uas twb muaj lawm."

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 qhov cuam tshuam dav 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 kho mob tam sim no tau pom zoo.

Piv nrog kev pom zoo ntau cov kinase inhibitors, avapritinib yog qhov muaj kev xaiv ntau rau KIT thiab PDGFRA dua li lwm cov kinases. Ntxiv rau, 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 ntawm 95% ntawm cov neeg mob uas muaj kab mob 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 nrog rau GIST, Blueprint Cov Tshuaj Kho Mob kuj tseem tsim muaj avapritinib rau kev kho mob ntawm SM siab, tsis lees paub thiab haus luam yeeb 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 2 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 me me xws li SM (SM-AHN) thiab mast cell leukemia (MCL) nrog cov qog hematological hlav.