Lub Hauv Paus Tswj Xyuas Kev Tshuaj Yeeb Tshuaj (NMPA) lees txais Cov Ntawv Thov rau Ob Qhov Ntsuas Rau Thawj-hauv-chav kawm Txoj Kev Plab Mob Qog Nqaij Nqaij Tawv Kom Ua Ntej Tshuaj Ntsiav Tshuaj Avapritinib

Lub Plaub Hlis 23, 2020, Keystone Tshuaj (Suzhou) Co., Ltd. (txuas ntxiv mus tom" Keystone Tshuaj" ;, Hong Kong Tshuag Sib Pauv Cov Cai: 2616) tshaj tawm tias Lub Tsev Haujlwm Saib Xyuas Kev Noj Tshuaj (NMPA) tau lees txais mob plab hnyuv loj (GIST) Daim ntawv thov kev lag luam tshuaj tshiab rau qhov tseeb-tsom tshuaj avapritinib, suav txog ob qhov ntsuas, rau kev rov ua dua ntawm exon 18 kev hloov pauv (suav nrog PDGFRA D { {5}} V hloov chaw) nqa cov platelet-tau los ntawm kev loj hlob yam receptor alpha (PDGFRA) lossis cov neeg mob neeg laus nrog Gist metastatic, thiab cov neeg laus muaj plaub-kab tsis phais lossis metastatic GIST. Avapritinib yog ib qho muaj zog, xaiv heev hauv lub qhov ncauj inhibitor tiv thaiv KIT thiab PDGFRA kev hloov pauv hauv kev tsim kho, tsim los ntawm cov tuam txhab tswv cuab Blueprint Tshuaj. Nov kuj tseem yog thawj daim ntawv teev npe tshuaj tshiab tau lees paub los ntawm NMPA los ntawm CStone Pharmaceuticals, khij ib kauj ruam tseem ceeb hauv tuam txhab' kev hloov pauv kev lag luam.

Txog 1-1. 5 / 100, 000 (14, 000-21,000) ntawm cov neeg kuaj mob GIST tshiab hauv Suav teb txhua xyoo [Zhou Yanbing. Cov xwm txheej tam sim no ntawm kev kuaj mob thiab kev kho mob txoj hnyuv pob txha caj qaum [J]. Cov Ntawv Xov Xwm Suav ntawm Kev Kho Mob Tshaj Tawm, 2017, 032 (007): 549-552.], Txog 90% ntawm cov neeg mob GIST cuam tshuam nrog kev hloov pauv KIT lossis PDGFRA hloov pauv. Lub Ib Hlis xyoo no, FDA tau pom zoo nws siv rau kev kho mob rau cov neeg mob cov neeg laus nrog PDGFRA exon 18 kev hloov pauv uas tsis yog phais mob los yog tawm tsam GIST, los ua thawj zaug thiab tsuas yog ua lag luam hauv Tebchaws Meskas rau qhov kev kho mob ncaj qha PDGFRA exon 18 kev hloov pauv. Hom phiaj tshuaj.

Dr. Jiang Ningjun, Tus Thawj Coj thiab CEO ntawm CStone Pharmaceuticals tau hais tias:" Tsuas yog peb lub hlis tom qab avapritinib tau pom zoo los ntawm Asmeskas FDA, CStone Pharmaceuticals tau xa cov avapritinib' s tshiab kev lag luam tshuaj thov hauv Taiwan thiab Tuam Tshoj Mainland, thiab cia siab tias yuav tuaj yeem ua kom tiav sai Nqa hom ua ntej no-hauv-chav kawm raug npliag tshuaj rau cov neeg mob siab heev GIST hauv Suav Teb. Raws li lub tuam txhab' s cov kev hloov pauv ntawm kev lag luam txuas ntxiv mus txuas ntxiv mus, nyob rau ob peb lub hlis tom ntej no, CStone Cov Tshuaj Muaj Nqes tseem yuav xa ntau cov khoom tseem ceeb hauv Tuam Tshoj Cov tshuaj tshiab rau ntau lub cim."

Professor Shen Lin, tus lwm thawj saib xyuas ntawm Peking University Cancer Tsev Kho Mob, tus thawj coj ntawm lub Department of Gastroenterology, thiab tus thawj xibfwb tshawb fawb txog avapritinib nyob hauv Suav teb, hais tias:" Avapritinib yog nyob rau hauv GIST nrog PDGFRA exon 18 hloov pauv thiab hauv cov neeg mob GIST nrog kev kho plaub-kab. Ua rau pom kev ua haujlwm zoo heev, thiab muaj kev nyab xeeb thiab zoo rau tiv thaiv. Vim tias muaj txiaj ntsig tsawg kawg ntawm cov neeg mob uas muaj cov kev kho mob uas twb muaj lawm, ob hom GIST ntawm cov neeg mob muaj cov kev xav tau kev pab zoo ib yam. Ua ib tus kws kho mob, peb cia siab Vam tias avapritinib yuav pom zoo kom sai li sai tau thiab dhau los ua qhov kev xaiv kho mob tshiab rau cov neeg mob uas muaj GIST siab."

Lub Kaum Ib Hlis 2019, CTOS lub rooj sib tham txhua xyoo tshaj tawm cov txiaj ntsig ntawm NAVIGATOR Ntu 1 qhov kev sim tshuaj rau avapritinib hauv PDGFRA exon 18 kev hloov pauv thiab cov neeg mob tau txais plaub-kab GIST. Cov ntaub ntawv txij li Lub Kaum Ib Hlis 16, 2018 qhia:

1. Lub 43 cov neeg mob PDGFRA exon 1 8 hloov thiab 1 1 1 cov neeg mob plaub-kab kev kho mob tau txais thawj koob tshuaj avapritinib 300 mg los yog 400 mg ib hnub ib zaug, thiab qhov ua tau zoo yuav tau raug ntsuas.

2. Ntawm cov neeg mob PDGFRA exon 18 hloov chaw, ORR yog 86% (1 tus neeg mob yuav tsum tau lees paub), qhov nruab nrab ntawm kev ncua sijhawm (DOR) tsis tau txog

3. Ntawm cov kab plaub ntawm cov neeg mob GIST, ORR tau mus txog 22% (1 tus neeg mob yuav tsum lees paub), nrog tus lej DOR ntawm 10. 2 lub hlis.

Nws muaj kev txaus siab tias cov ntaub ntawv ua ntej ntawm Tuam Tshoj Theem I / II bridging txoj kev tshawb fawb nqa los ntawm CStone Pharmaceuticals qhia tau hais tias kev nyab xeeb thiab cov qauv tshuaj ntawm cov neeg mob uas muaj GIST nyob rau Suav teb yog suav nrog NAVIGATOR cov ntaub ntawv kawm thoob ntiaj teb thiab muaj kev tiv thaiv zoo.

Dr. Yang Jianxin, Tus Thawj Saib Xyuas Kev Kho Mob ntawm CStone Pharmaceuticals, tau hais tias:" Tam sim no, kev kho mob siab ntawm GIST nyob rau hauv Suav teb yog feem ntau siv cov tshuaj tyrosine kinase inhibitors (TKIs). Cov nyhuv me me heev. Ntawm qhov tod tes, Suav tus plaub-kab GIST cov neeg mob ntsib ntau yam kev nyuaj xws li kev hloov tshuaj ntau yam thiab tsis muaj kev pom zoo kev kho kom zoo. Kuv zoo siab heev pom tias tam sim no kev tshawb nrhiav txuas ntawm cov ntaub ntawv avapritinib hauv Suav tau zoo raws li cov txiaj ntsig ntawm NAVIGATOR txoj kev kawm thoob ntiaj teb Thiab tos ntsoov rau nws cov txiaj ntsig thaum ntxov rau ntau cov neeg mob GIST uas xav tau kev kho kom zoo ntxiv."

CStone Cov Lag Luam Tshuaj tau mus txog kev sib koom tes tshwj xeeb thiab kev tso cai nrog Blueprint Tshuaj, thiab tau txais kev tsim tshwj xeeb thiab ua lag luam tso cai ntawm ntau cov tshuaj suav nrog avapritinib hauv Greater Tuam Tshoj. Blueprint Cov tshuaj kho mob muaj txoj cai los tsim thiab ua lag luam tshuaj nyob rau hauv lwm lub ntiaj teb.

Txog Avapritinib

Avapritinib yog KIT thiab PDGFRA mutant kinase inhibitor uas tab tom tshawb xyuas, muaj zog thiab xaiv tau zoo. Nws yog hom 1 kinase conformational inhibitor siv nyob rau hauv kev kho mob ntawm GIST thiab tuaj yeem cuam tshuam ncaj qha rau cov kev ua haujlwm kinase kev hloov pauv ntawm KIT thiab PDGFRA kev hloov pauv uas ua rau kom muaj kev cuam tshuam qis los ua kom muaj kev cuam tshuam. Avapritinib tau lees tias qhov tshwm sim inhibitory ntawm KIT thiab PDGFRA hloov pauv hauv GIST. Tam sim no, lub ntsiab tshuaj tiv thaiv cov tshuaj ntawm kev pom zoo kev kho mob hauv Suav Tuam Tshoj yog tshwm sim los ntawm kev hloov lub voj kev hloov pauv. Avapritinib suppresses hloov pauv ntawm qhov chaw no, uas ua rau muaj kev ua haujlwm hauv chaw kuaj mob muaj zog.

Blueprint Cov tshuaj kho mob yog kev txhim kho ntawm cov kev pabcuam dav dav kho rau avapritinib, suav nrog GIST rau cov kab kev kho ntau yam, thiab kev ua haujlwm siab, tsis muaj zog thiab haus luam yeeb mastocytosis (SM).

Avapritinib yog kinase inhibitor pom zoo los ntawm Asmeskas FDA raws li cov khoom lag luam npe AYVAKITTM rau kev kho mob rau cov neeg mob laus uas tsis yog phais lossis metastatic GIST nqa PDGFRA exon 18 hloov chaw (suav nrog PDGFRA D 842 V hloov chaw) Cov.

Avapritinib tseem tsis tau pom zoo los ntawm US FDA rau kev kho mob ntawm lwm qhov taw qhia, thiab tseem tsis tau pom zoo los ntawm TFDA, NMPA lossis lwm cheeb tsam kev tswj hwm kev noj qab haus huv rau ib qho kev qhia.