Daawaynta ugu Horreysa ee La Ansixiyay ee Cytopenic Myelofibrosis

CTI BioPharma Corp. ayaa ku dhawaaqday Maamulka Cuntada iyo Dawooyinka ee Maraykanka (FDA) ayaa ansixiyay VONJO (pacritinib) daaweynta dadka qaangaarka ah ee dhexdhexaadka ah ama khatarta sare leh ee hoose ama sare (polycythemia vera post-polycythemia or post-esential thrombocythemia) myelofibrosis oo leh tiro platelet ah. ka hooseeya 50 × 109/L. VONJO waa kinaase afka laga qaato oo cusub oo gaar u ah JAK2 iyo IRAK1, iyada oo aan la joojin JAK1. Qiyaasta lagu taliyey ee VONJO waa 200 mg afka laba jeer maalintii. VONJO waa daawaynta ugu horraysa ee la ansixiyay oo si gaar ah wax uga qabata baahiyaha bukaanada qaba myelofibrosis cytopenic.             

"Oggolaanshaha maanta ee VONJO waxay dejineysaa heer cusub oo daryeel loogu talagalay bukaanada myelofibrosis ee la ildaran cytopenic myelofibrosis," ayuu yiri John Mascarenhas, MD, Professor Associate, Medicine, Hematology iyo Oncology Medical, Machadka Kansarka ee Tisch, Dugsiga Icahn ee Daawada ee Mount Sinai, New York . "Myelofibrosis oo leh thrombocytopenia daran, oo lagu qeexay sida platelet dhiig oo ka hooseeya 50 × 109/L, ayaa la muujiyey inay keento natiijooyin badbaado oo liita oo ay weheliso calaamado daciif ah. Ikhtiyaarada daawaynta xaddidan ayaa ka dhigay cudurkan meel baahi caafimaad oo degdeg ah aan la daboolin. Waan ku faraxsanahay inaan arko in doorasho cusub, wax ku ool ah oo badbaado leh oo daaweyn ah ay hadda diyaar u yihiin bukaannadan."

"Mareykanka, waxaa jira qiyaastii 21,000 bukaan oo qaba myelofibrosis, saddex-meelood laba meel oo ka mid ah waxay qabaan cytopenia (thrombocytopenia ama dhiig-yaraan), oo badanaa ka dhasha sunta daawooyinka kale ee la ansixiyey. thrombocytopenia ba'an, oo lagu qeexay tirada platelet ee ka hooseeya 50 × 109/L, waxay ku dhacdaa saddex-meelood meel ka mid ah guud ahaan dadweynaha myelofibrosis, waxayna leedahay saadaalin gaar ah oo liidata. Ansixinta VONJO, waxaan ku faraxsanahay inaan hadda awoodno inaan bixinno daaweyn cusub oo si gaar ah loogu oggolaaday bukaannada qaba myelofibrosis cytopenic. Waxa si buuxda naloo maalgeliyay bilawga ganacsiga, ka dib deyntayada iyo macaamilada royalty ee DRI, waxaanan rajeyneynaa inaan siino VONJO, daawaynta ugu wanaagsan ee fasalka loogu talagalay bukaanada cytopenic myelofibrosis, bukaanada 10 maalmood gudahood, "ayuu yiri Adam R. Craig. , MD, Ph.D., Madaxweynaha iyo madaxa fulinta ee CTI Biopharma. "Waxaan jeclaan lahaa inaan u mahadceliyo bukaannada, daryeel-bixiyeyaasha, shaqaalaha tijaabada caafimaadka iyo baarayaasha suurtageliyay tijaabooyinka bukaan-socodka ee VONJO. Waxaan sidoo kale uga mahadcelinayaa kooxda CTI dadaalkooda iyo dadaalkooda iyo sida ay diiradda u saareen baahiyaha bukaannada."

Oggolaanshaha la dedejiyey waxay ku salaysan tahay natiijooyinka waxtarka leh ee ka soo baxa daraasadda muhiimka ah ee Wajiga 3 PERSIST-2 ee VONJO ee bukaannada qaba myelofibrosis (platelet wuxuu tiriyaa wax ka yar ama la mid ah 100 × 109/L). Bukaannada ayaa la kala soocay 1: 1: 1 si ay u helaan VONJO 200 mg laba jeer maalintii (BID), VONJO 400 mg hal mar maalintii (QD) ama daaweynta ugu fiican ee la heli karo (BAT). Kahor daawaynta ka hortagga JAK2 waa la oggolaaday. Daraasaddan, kooxda bukaanada leh platelet-ka aasaasiga ah ayaa ka hooseeya 50 × 109/L kuwaas oo lagu daaweeyay pacritinib 200 mg BD, 29% bukaanada ayaa hoos u dhacay mugga beeryarada ugu yaraan 35% marka la barbardhigo 3% bukaanada helaya. daaweynta ugu fiican ee la heli karo, oo ay ku jiraan ruxolitinib. Iyada oo qayb ka ah oggolaanshaha la dedejiyey, CTI ayaa looga baahan yahay inay qeexdo faa'iidada kiliinikada ee tijaabada xaqiijinta. Si loo buuxiyo shuruudaha ansixinta kadib, CTI waxay qorshaynaysaa inay dhamaystirto tijaabada PACIFICA, iyadoo natiijooyin la filayo badhtamaha 2025.

Dareen-celinta xun ee ugu caansan (≥20%) ka dib VONJO 200 mg laba jeer maalintii waa shuban, thrombocytopenia, lallabbo, dhiig-yaraan iyo barar durugsan. Dareen-celinta xun xun ee soo noqnoqda (≥3%) ka dib VONJO 200 mg laba jeer maalintii waa dhiig-yaraan, thrombocytopenia, oof wareenka, wadnaha oo shaqayn, horumarka cudurka, pyrexia iyo kansarka unugyada squamous ee maqaarka.