Chithandizo Choyamba Chovomerezeka cha Cytopenic Myelofibrosis

CTI BioPharma Corp. yalengeza bungwe la US Food and Drug Administration (FDA) lavomereza VONJO (pacritinib) pochiza anthu akuluakulu omwe ali ndi chiopsezo chachikulu chapakati kapena chachiwiri (post-polycythemia vera kapena post-essential thrombocythemia) myelofibrosis yokhala ndi chiwerengero cha mapulateleti. pansi pa 50 × 109 / L. VONJO ndi buku la oral kinase inhibitor lodziwika bwino la JAK2 ndi IRAK1, popanda kuletsa JAK1. Mlingo wovomerezeka wa VONJO ndi 200 mg pakamwa kawiri tsiku lililonse. VONJO ndiye chithandizo choyamba chovomerezeka chomwe chimakwaniritsa zofunikira za odwala omwe ali ndi cytopenic myelofibrosis.             

"Lero kuvomereza kwa VONJO kumakhazikitsa njira yatsopano yothandizira odwala myelofibrosis omwe akudwala cytopenic myelofibrosis," atero a John Mascarenhas, MD, Pulofesa Wothandizira, Medicine, Hematology and Medical Oncology, Tisch Cancer Institute, Icahn School of Medicine ku Mount Sinai, New York. . "Myelofibrosis yokhala ndi thrombocytopenia yoopsa, yomwe imatanthauzidwa ngati mapulateleti a magazi pansi pa 50 × 109 / L, asonyezedwa kuti amabweretsa zotsatira zoipa za kupulumuka pamodzi ndi zizindikiro zofooketsa. Njira zochepetsera chithandizo chamankhwala zapangitsa kuti matendawa akhale gawo lofunikira kuchipatala mwachangu. Ndine wokondwa kuwona kuti njira yatsopano, yothandiza komanso yotetezeka tsopano yapezeka kwa odwalawa. ”

"Ku US, kuli odwala pafupifupi 21,000 omwe ali ndi myelofibrosis, magawo awiri mwa atatu omwe ali ndi cytopenias (thrombocytopenia kapena kuchepa kwa magazi m'magazi), omwe amayamba chifukwa cha poizoni wamankhwala ena ovomerezeka. Kuopsa kwa thrombocytopenia, komwe kumatanthauzidwa ngati kuwerengera kwa magazi m'munsi mwa 50 × 109/L, kumachitika mu gawo limodzi mwa magawo atatu a anthu onse a myelofibrosis, ndipo ali ndi vuto losauka kwambiri. Ndi chivomerezo cha VONJO, ndife okondwa kuti tsopano titha kupereka mankhwala atsopano omwe amavomerezedwa makamaka kwa odwala omwe ali ndi cytopenic myelofibrosis. Timalipidwa mokwanira kuti tiyambitse malonda, potsatira ngongole zathu ndi malonda ndi DRI, ndipo tikuyembekeza kupereka VONJO, chithandizo chapamwamba kwambiri cha odwala cytopenic myelofibrosis, kwa odwala mkati mwa masiku 10, "adatero Adam R. Craig. , MD, Ph.D., Purezidenti ndi Chief Executive Officer wa CTI Biopharma. "Ndikufuna kuthokoza odwala, osamalira, ogwira ntchito m'mayesero azachipatala komanso ofufuza omwe apangitsa kuti kuyesa kwachipatala kwa VONJO kutheke. Ndikuthokozanso gulu la CTI chifukwa chogwira ntchito molimbika komanso kudzipereka komanso kuyang'ana kwambiri zosowa za odwala. "

Chivomerezo chofulumizitsa chimachokera ku zotsatira zogwira mtima kuchokera ku kafukufuku wofunika kwambiri wa Phase 3 PERSIST-2 wa VONJO mwa odwala omwe ali ndi myelofibrosis (mapulateleti amawerengera osachepera kapena ofanana ndi 100 × 109/L). Odwala adasinthidwa 1: 1: 1 kuti alandire VONJO 200 mg kawiri tsiku lililonse (BID), VONJO 400 mg kamodzi patsiku (QD) kapena chithandizo chopezeka bwino (BAT). M'mbuyomu JAK2 inhibitor therapy idaloledwa. Mu kafukufukuyu, mu gulu la odwala omwe ali ndi mapulateleti oyambira pansi pa 50 × 109/L omwe amathandizidwa ndi pacritinib 200 mg BD, 29% ya odwala anali ndi kuchepa kwa ndulu ndi 35% poyerekeza ndi 3% ya odwala omwe amalandila. chithandizo chabwino kwambiri chopezeka, chomwe chinali ndi ruxolitinib. Monga gawo la chivomerezo chofulumira, CTI ikufunika kufotokoza phindu lachipatala mu mayesero otsimikizira. Kuti akwaniritse izi pambuyo povomerezedwa, CTI ikukonzekera kumaliza kuyesa kwa PACIFICA, ndi zotsatira zoyembekezeredwa pakati pa 2025.

Zoyipa zomwe zimachitika kwambiri (≥20%) pambuyo pa VONJO 200 mg kawiri tsiku lililonse zinali m'mimba, thrombocytopenia, nseru, kuchepa kwa magazi m'thupi ndi zotumphukira edema. Zowopsa zomwe zimachitika pafupipafupi (≥3%) zotsatizana ndi VONJO 200 mg kawiri tsiku lililonse zinali kuchepa kwa magazi m'thupi, thrombocytopenia, chibayo, kulephera kwamtima, kukwera kwa matenda, pyrexia ndi squamous cell carcinoma yapakhungu.