pCAR-19B's NDA rau kev kho mob leukemia thaum yau tau txais kev pom zoo los ntawm NMPA

Thaum Lub Xya Hli 20, Daim Ntawv Thov Tshuaj Tshiab(NDA) ntawm pCAR-19B uas tsim los ntawm Precision Biology tau txais kev pom zoo los ntawm Tuam Tshoj National Medical Products Administration (NMPA). Cov khoom no yog thawj cov khoom CAR-T rau cov menyuam yaus leukemia hauv Suav teb, uas yog siv los kho cov neeg mob hnub nyoog 3-21 nrog CD19-positive relapsed/refractory acute lymphoblastic leukemia (ALL).

Kab mob leukemia lymphoblastic mob hnyav (ALL) yog ib qho ntawm cov qog nqaij hlav phem tshaj plaws hauv menyuam yaus thiab yog ib qho ua rau menyuam yaus thiab cov tub ntxhais hluas tuag ntau tshaj plaws. Kev kho mob chimeric antigen receptor (CAR-T) cell tau hloov pauv kev kho mob ntawm cov kab mob B-cell ALL uas rov qab mob/tsis kho tau dua, nrog rau thawj cov khoom lag luam uas FDA pom zoo, Kymriah (tisagenlecleucel) tsim los ntawm Novartis, uas ua rau cov nqi kho mob zoo dua qub thiab lub sijhawm ciaj sia zoo dua qub.

pCAR-19B yog ib yam khoom siv CAR-T cell uas muaj ib qho humanized CD19-specific single-chain variable fragment (scFv) uas tsim los daws cov teeb meem kev nyab xeeb.

Thaum Lub Kaum Ib Hlis 2023, pCAR-19B tau txais "Breakthrough Therapy Designation" los ntawm Tuam Tshoj National Medical Products Administration rau kev kho mob B-ALL.

Kev ua tau zoo thiab kev nyab xeeb ntawm pCAR-19B hauv kev sim tshuaj tseem ceeb uas muaj cov menyuam yaus thiab cov tub ntxhais hluas Suav teb uas muaj R/R B-cell ALLtau tshaj tawm thaumLub Rooj Sib Tham Txhua Xyoo ntawm American Society of Hematology (ASH) thib 66.

Txoj Kev: Qhov kev sim theem 2 no (NCT05334823) yog ib txoj kev tshawb fawb qhib rau ntau lub chaw. Cov neeg mob hnub nyoog ≥ 3 thiab ≤ 21 xyoo ntawm kev tshuaj ntsuam nrog CD19 + R / R B-ALL tau raug suav nrog. Lub hom phiaj tseem ceeb yog qhov kev zam txim tag nrho (ORR), txhais tias yog kev zam txim tag nrho (CR) ntxiv rau CR nrog kev rov qab ntshav tsis tiav (CRi) hauv 3 lub hlis tom qab kev txhaj tshuaj.

Cov Txiaj Ntsig: Txij li thaum Lub Plaub Hlis 18, 2024, muaj 89 tus neeg mob tau raug sau npe thiab tau txais kev kho mob leukapheresis, nrog rau 64 tus neeg mob tau txais kev txhaj tshuaj pCAR-19B. Tsis muaj cov xwm txheej ntawm kev tsim khoom tsis ua haujlwm. Ntawm cov neeg mob uas tau txhaj tshuaj, 6.25% (4/64) yog cov neeg mob uas tsis kho tau thiab 93.75% (60/64) tau rov qab mus rau ntau txoj kev kho mob yav dhau los lossis kev hloov pauv hlwb allogeneic. Lub hnub nyoog nruab nrab yog 11 xyoos (ntau yam 3-21), thiab 56.25% (36/64) yog txiv neej. Tsis tas li ntawd, 75% (48/64) ntawm cov neeg mob nqa tsawg kawg ib lub noob caj noob ces muaj kev pheej hmoo siab. Lub nra hnyav ntawm cov pob txha hauv lub cev (BM) thaum pib yog 58.3% (ntau yam 5% -98%) nrog lub nra hnyav ntawm BM (≥50%) pom hauv 56.25% ntawm cov neeg mob.

Ntawm qhov nruab nrab ntawm 211 hnub (ntau 35-750), qhov ORR zoo tshaj plaws yog 90.63% (58/64), nrog 78.13% ntawm cov neeg mob (50/64) ua tiav CR thiab 12.5% ​​ntawm cov neeg mob (8/64) ua tiav CRi, dhau qhov ua tau zoo ntawm cov khoom lag luam tiv thaiv CD19 CAR-T kho mob tau tshaj tawm hauv R/R B-ALL. 98.27% (57/58) ntawm cov neeg mob uas muaj ORR tau ua tiav qhov tsis zoo ntawm qhov tsis zoo ntawm qhov mob tsawg kawg nkaus (MRD)-tsis zoo. ORR ntawm 3 lub hlis yog 76.56% (49/64). Lub sijhawm nruab nrab ntawm kev teb (DOR) yog 10.61 lub hlis (95% kev ntseeg siab 7.66-20.96). Qhov nruab nrab ntawm kev ciaj sia tag nrho (OS) yog 23.92 lub hlis (95% kev ntseeg siab 9.86-NR).

Cytokine release syndrome (CRS) thiab immune effector cell-associated neurotoxicity syndrome (ICANS) tau tshwm sim hauv 98.44% thiab 50% ntawm cov neeg mob, raws li. Qib ≥3 CRS thiab ICANS tau pom hauv 29.69% thiab 39.06% ntawm cov neeg mob, raws li. Lub sijhawm nruab nrab ntawm Qib ≥3 CRS thiab ICANS yog 4 hnub thiab 3 hnub, raws li. Tsis muaj kev tuag uas tau hais txog CRS lossis ICANS.

Xaus Lus: Txawm hais tias muaj kev nyuaj siab ntawm BM blasts thiab muaj kev pheej hmoo siab ntawm kev hloov pauv caj ces, pCAR-19B tau qhia txog kev zam txim siab, ua tiav qhov kev zam txim siab MRD-negative, cov lus teb ruaj khov, thiab cov ntaub ntawv kev nyab xeeb uas them taus. Kev tshawb fawb no sawv cev rau thawj qhov kev sim tshuaj kho mob tseem ceeb ntawm cov menyuam yaus B-ALL hauv cov pej xeem Es Xias, muab kev xaiv kho mob tshiab rau cov menyuam yaus Suav thiab cov neeg mob hluas uas muaj R/R B-ALL.

Tam sim no, muaj ntau lwm yam kev sim tshuaj CAR-T hauv Suav teb, thiab lawv tab tom nrhiav cov neeg mob. Yog xav tau kev sab laj txog cov tshuaj tshiab thiab cov thev naus laus zis, koj tuaj yeem tiv tauj Beijing South Region Oncology Hospital International Department.

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Cov ntaub ntawv siv los ua piv txwv

1.https://www.cde.org.cn/main/xxgk/listpage/9f9c74c73e0f8f56a8bfbc646055026d

2.http://cq.news.cn/20240728/b0a74788930d4be7a45996be2808dd4f/c.html

3.https://mp.weixin.qq.com/s?__biz=MzAxMzgzOTk1OQ==&mid=2650794360&idx=1&sn=f1628888e194d1c2d0fb05894f92a969&chksm=83973240b4e0bb56e67dff6126946d2c9c92d8bc5f436b82552d99dca14072f37f2f360ca567#rd

4.https://ash.confex.com/ash/2024/webprogram/Paper206408.html

5.https://ashpublications.org/blood/article/140/Supplement%201/4625/491174/The-Safety-and-Efficacy-of-p-CAR-19B-a-Scfv