Hoʻonui ʻia ke aʻo ʻana i ka maʻi maʻi ʻaʻai iwi

I ka hapaha lua o 2021, ua hāʻawi ka FDA i ka loiloi mua no ka CTI's NDA no nā poʻe maʻi me myelofibrosis me ka lā PDUFA o Nowemapa 30, 2021. I ka wā o nā kūkākūkā hōʻailona huahana, ua noi ka FDA i nā ʻikepili lapaʻau hou, i waiho ʻia i ke keʻena. ma Nowemapa 24, 2021. Ma mua o kēia lā, ua haʻi aku ka FDA i ka Hui e noʻonoʻo ana i ka waiho ʻana i ka ʻikepili e lilo i "hoʻololi nui" i ka NDA a no laila ua hoʻonui ʻia ka lā PDUFA i ʻekolu mahina e hāʻawi i ka manawa hou no ka loiloi piha ka waiho ana. I kēia manawa, ʻaʻole ʻike ʻo CTI i nā hemahema nui o ka noi.

ʻO Pacritinib kahi mea hoʻopaneʻe kinase waha me ka kikoʻī no JAK2, IRAK1 a me CSF1R, me ka ʻole o ka pale ʻana iā JAK1. Ua ʻae ʻia ka NDA ma muli o ka ʻikepili mai ka Phase 3 PERSIST-2 a me PERSIST-1 a me ka Phase 2 PAC203 clinical trials, me ka nānā ʻana i ka thrombocytopenic koʻikoʻi (helu platelet ma lalo o 50 x 109/L) nā maʻi i kākau ʻia i kēia mau haʻawina. ka mea i loaʻa i ka pacritinib 200 mg ʻelua i ka lā, me nā mea maʻi lapaʻau mua-naive a me nā mea maʻi me ka ʻike mua ʻana i ka JAK2 inhibitors. Ma ka noiʻi PERSIST-2, i nā poʻe maʻi me ka thrombocytopenia koʻikoʻi i mālama ʻia me ka pacritinib 200 mg ʻelua i ka lā, 29% o nā mea maʻi i hōʻemi i ka nui o ka spleen ma ka liʻiliʻi he 35%, hoʻohālikelike ʻia me 3% o nā mea maʻi e loaʻa ana ka lāʻau lapaʻau maikaʻi loa. , i komo pū me ruxolitinib; ʻO 23% o nā mea maʻi i hōʻemi i ka nui o nā helu hōʻailona ma kahi o 50%, i hoʻohālikelike ʻia me 13% o nā maʻi e loaʻa ana i ka lāʻau lapaʻau maikaʻi loa. Ma ka heluna kanaka like o ka poe ma'i i lapaau ia me ka pacritinib, he ha'aha'a ka ma'i 'ino, hiki ke mālama 'ia me ka mālama kāko'o, a 'a'ole hiki ke ho'opau 'ia. Ua hoʻopaʻa ʻia ka helu platelet a me ka pae hemoglobin.

ʻO ka Myelofibrosis ka maʻi ʻaʻai iwi iwi e hopena i ka hoʻokumu ʻana i ka ʻiʻo fibrous scar a hiki ke alakaʻi i ka thrombocytopenia a me ka anemia, nāwaliwali, luhi a me ka hoʻonui ʻia o ka spleen a me ka ate. Aia i loko o ka US ma kahi o 21,000 mau maʻi me ka myelofibrosis, 7,000 o lākou i loaʻa i ka thrombocytopenia koʻikoʻi (i wehewehe ʻia he helu platelet koko ma lalo o 50 x109/L). Hoʻopili ʻia ka thrombocytopenia koʻikoʻi me ke ola maikaʻi ʻole a me ke kaumaha o nā hōʻailona kiʻekiʻe a hiki ke loaʻa ma muli o ka piʻi ʻana o ka maʻi a i ʻole mai ka lāʻau ʻawaʻawa me nā mea paʻi JAK2 e like me JAKAFI a me INREBIC.