CTI BioPharma Corp. ta sanar da Hukumar Abinci da Magunguna ta Amurka (FDA) ta amince da VONJO (pacritinib) don kula da manya masu matsakaici ko babban haษari na farko ko na sakandare (bayan polycythemia vera ko thrombocythemia mai mahimmanci) myelofibrosis tare da ฦididdigar platelet. kasa da 50 ร 109/L. VONJO labari ne mai hana kinase na baka tare da keษancewa ga JAK2 da IRAK1, ba tare da hana JAK1 ba. Matsakaicin shawarar VONJO shine MG 200 na baki sau biyu a rana. VONJO shine farkon yarda da magani wanda ke magance bukatun marasa lafiya tare da myelofibrosis na cytopenic.
"Yau yarda da VONJO ya kafa wani sabon tsarin kula da myelofibrosis marasa lafiya fama da cytopenic myelofibrosis," in ji John Mascarenhas, MD, Mataimakin Farfesa, Medicine, Hematology da Medical Oncology, Tisch Cancer Institute, Icahn School of Medicine a Dutsen Sinai, New York. . "Myelofibrosis tare da thrombocytopenia mai tsanani, wanda aka bayyana a matsayin adadin platelet na jini a kasa da 50 ร 109/L, an nuna shi don haifar da mummunan sakamakon rayuwa tare da cututtuka masu lalacewa. Zaษuษษukan magani masu iyaka sun mayar da wannan cuta a matsayin yanki na gaggawar buฦatar likita da ba a biya ba. Na yi farin cikin ganin cewa yanzu akwai sabon zaษin magani mai inganci da aminci ga waษannan majinyata."
"A Amurka, akwai kusan marasa lafiya 21,000 masu fama da myelofibrosis, kashi biyu cikin uku na suna da cytopenia (thrombocytopenia ko anemia), yawanci sakamakon gubar wasu hanyoyin da aka yarda da su. Matsanancin thrombocytopenia, wanda aka bayyana azaman adadin platelet ษin da ke ฦasa da 50 ร 109/L, yana faruwa a cikin kashi ษaya bisa uku na yawan myelofibrosis gabaษaya, kuma yana da ฦima sosai. Tare da amincewar VONJO, muna farin cikin yanzu don samun damar ba da sabon magani wanda aka yarda da shi musamman ga marasa lafiya tare da myelofibrosis na cytopenic. An ba mu cikakken kuษi don ฦaddamar da kasuwanci, bin bashin mu da ma'amalar sarauta tare da DRI, kuma muna sa ido don samar da VONJO, mafi kyawun magani ga marasa lafiya na myelofibrosis na cytopenic, ga marasa lafiya a cikin kwanaki 10, "in ji Adam R. Craig. , MD, Ph.D., Shugaba da Babban Jami'in Gudanarwa na CTI Biopharma. โIna so in gode wa marasa lafiya, masu ba da kulawa, maโaikatan gwaji na asibiti da kuma masu binciken da suka sanya gwajin asibiti na VONJO ya yiwu. Har ila yau, ina godiya ga tawagar CTI saboda kwazon da suka yi da sadaukarwar da suka mayar da hankali kan bukatun marasa lafiya."
Amincewa da hanzari ya dogara ne akan sakamako mai tasiri daga muhimmin mataki na 3 PERSIST-2 nazarin VONJO a cikin marasa lafiya da myelofibrosis (platelet yana ฦidaya ฦasa da ko daidai 100 ร 109/L). An ba da izini ga marasa lafiya 1: 1: 1 don karษar VONJO 200 MG sau biyu a kowace rana (BID), VONJO 400 MG sau ษaya kowace rana (QD) ko mafi kyawun samuwa (BAT). An ba da izinin maganin hanawa na JAK2 na baya. A cikin wannan binciken, a cikin rukunin marasa lafiya tare da platelet na asali sun ฦidaya a ฦasa 50 ร 109/L waษanda aka yi musu magani tare da pacritinib 200 mg BD, 29% na marasa lafiya sun sami raguwar ฦarar ฦwayar ฦwayar cuta na aฦalla 35% idan aka kwatanta da 3% na marasa lafiya da ke karษar. Mafi kyawun samuwa far, wanda ya hada da ruxolitinib. A matsayin wani ษangare na haษakar yarda, ana buฦatar CTI don bayyana fa'idar asibiti a cikin gwajin tabbatarwa. Don cika wannan buฦatun amincewa bayan amincewa, CTI tana shirin kammala gwajin PACIFICA, tare da sakamakon da ake sa ran a tsakiyar 2025.
Mafi yawan halayen rashin lafiya (โฅ20%) biyo bayan VONJO 200 MG sau biyu a rana sune gudawa, thrombocytopenia, tashin zuciya, anemia da edema na gefe. Mafi yawan mummunan halayen haษari (โฅ3%) biyo bayan VONJO 200 MG sau biyu a kowace rana sune anemia, thrombocytopenia, ciwon huhu, gazawar zuciya, ci gaban cututtuka, pyrexia da squamous cell carcinoma na fata.
