Muchikamu chechipiri cha2021, FDA yakapa ongororo yekutanga yeCTI's NDA yevarwere vane myelofibrosis ine PDUFA yemusi waMbudzi 30, 2021. Mukati menhaurirano dzekunyora kwechigadzirwa, FDA yakakumbira rumwe ruzivo rwekiriniki, iro rakaendeswa kubazi. musi waMbudzi 24, 2021. Nhasi, FDA yakaudza Kambani kuti inoona kuendeswa kwedata kuita "kugadzirisa kukuru" kuNDA uye saka zuva rePDUFA rawedzerwa nemwedzi mitatu kuti ipe imwe nguva yekuongorora kuzere. kuzviisa pasi. Parizvino, CTI haizive chero kukanganisa kukuru mukushandisa.
Pacritinib inoveli oral kinase inhibitor ine hunyanzvi hweJAK2, IRAK1 uye CSF1R, isina inhibiting JAK1. Iyo NDA yakagamuchirwa zvichienderana nedata kubva kuChikamu 3 PERSIST-2 uye PERSIST-1 uye Phase 2 PAC203 yekiriniki yekuedza, nekutarisa kune yakanyanya thrombocytopenic (platelet inoverengera isingasviki 50 x 109/L) varwere vakanyoresa muzvidzidzo izvi. avo vakagamuchira pacritinib 200 mg kaviri pazuva, kusanganisira vose vari mberi kwekurapa-vasingazivi varwere uye varwere vane ruzivo rwekutanga kune JAK2 inhibitors. Muchidzidzo chePERSIT-2, mune varwere vane thrombocytopenia yakaoma vakabatwa ne pacritinib 200 mg kaviri pazuva, 29% yevarwere vakave nekuderera kwespleen volume yeinenge 35%, zvichienzaniswa ne3% yevarwere vanowana kurapwa kwakanakisisa kunowanikwa. , iyo yaisanganisira ruxolitinib; 23% yevarwere vakave nekuderedzwa kwezviratidzo zvese zve50%, zvichienzaniswa ne13% yevarwere vanowana kurapa kwakanakisa kunowanikwa. Muhuwandu hwakafanana hwevarwere vanobatwa ne pacritinib, zviitiko zvakashata zvaive zvakaderera giredhi, zvinogoneka nekutarisira kunotsigira, uye zvisingawanzo kutungamirira pakuregedzwa. Zviverengero zveplatelet uye mwero wehemoglobin zvakadzikamiswawo.
Myelofibrosis igomarara remapfupa rinokonzera kuumbwa kwefibrous scar tissue uye inogona kutungamirira ku thrombocytopenia uye anemia, kushaya simba, kuneta uye kuwedzera kwespleen uye chiropa. MuUS mune varwere vanosvika zviuru makumi maviri nerimwe vane myelofibrosis, 21,000 vane thrombocytopenia yakaoma (inotsanangurwa sehuwandu hwemaplatelet eropa ari pasi pe7,000 x50/L). Severe thrombocytopenia inosanganiswa nekusararama zvakanaka uye kuremerwa kwezviratidzo uye inogona kuitika nekuda kwekukura kwechirwere kana kubva muhupfu hwezvinodhaka nemamwe maJAK109 inhibitors akadai seJAKAFI neINREBIC.
ZVOKUBVA MUNYAYA INO:
- Iyo NDA yakagamuchirwa zvichienderana nedata kubva kuChikamu 3 PERSIST-2 uye PERSIST-1 uye Phase 2 PAC203 makiriniki ekuedzwa, nekutarisa kune yakanyanya thrombocytopenic (platelet inoverengera isingasviki 50 x 109/L) varwere vakanyoresa muzvidzidzo izvi. avo vakagamuchira pacritinib 200 mg kaviri pazuva, kusanganisira vose vari mberi kwekurapa-vasingazivi varwere uye varwere vane ruzivo rwekutanga kune JAK2 inhibitors.
- Muchidzidzo chePERSIT-2, mune varwere vane thrombocytopenia yakakomba avo vakabatwa ne pacritinib 200 mg kaviri pazuva, 29% yevarwere vakave nekuderera kwespleen volume yeinenge 35%, zvichienzaniswa ne3% yevarwere vanowana kurapwa kwakanakisisa kunowanikwa. , iyo yaisanganisira ruxolitinib.
- Muchikamu chechipiri cha2021, iyo FDA yakapa ongororo yekutanga yeCTI's NDA yevarwere vane myelofibrosis ine PDUFA musi waNovember 30, 2021.
