I-CTI BioPharma Corp. imemezele i-US Food and Drug Administration (i-FDA) igunyaze i-VONJO (pacritinib) yokwelashwa kwabantu abadala abasengozini eyinhloko emaphakathi noma ephezulu noma yesibili (i-post-polycythemia vera noma i-post-essential thrombocythemia) i-myelofibrosis ene-platelet count. ngaphansi kuka-50 × 109/L. I-VONJO iyinoveli ye-oral kinase inhibitor enemininingwane ye-JAK2 ne-IRAK1, ngaphandle kokuvimbela i-JAK1. Umthamo onconyiwe we-VONJO ngu-200 mg ngomlomo kabili ngosuku. I-VONJO yindlela yokwelapha yokuqala egunyaziwe ebhekana ngqo nezidingo zeziguli ezine-cytopenic myelofibrosis.
"Ukugunyazwa kwanamuhla kwe-VONJO kusungula izinga elisha lokunakekelwa kweziguli ze-myelofibrosis eziphethwe yi-cytopenic myelofibrosis," kusho uJohn Mascarenhas, MD, Uprofesa Ohlangene, Imithi, I-Hematology kanye Nezokwelapha Oncology, i-Tisch Cancer Institute, i-Icahn School of Medicine eMount Sinai, eNew York. . “I-Myelofibrosis ene-thrombocytopenia enzima, echazwa njengezibalo zeplatelet yegazi ezingaphansi kuka-50 × 109/L, kuboniswe ukuthi kubangele imiphumela emibi yokusinda ehambisana nezimpawu eziwohlozayo. Izinketho zokwelashwa ezinomkhawulo zinikeze lesi sifo njengendawo yesidingo esiphuthumayo sezokwelapha esingahlangatshezwana nazo. Kuyangijabulisa ukubona ukuthi indlela entsha yokwelapha ephumelelayo nephephile isiyatholakala kulezi ziguli.”
“E-US, cishe kuneziguli ezingama-21,000 ezine-myelofibrosis, izingxenye ezimbili kwezintathu zazo ezine-cytopenia (thrombocytopenia noma i-anemia), evame ukubangelwa ubuthi bezinye izindlela zokwelapha ezigunyaziwe. I-thrombocytopenia enzima, echazwa njengenani leplatelet yegazi elingaphansi kuka-50 × 109/L, yenzeka engxenyeni eyodwa kwezintathu yenani labantu be-myelofibrosis, futhi inesimo esibi kakhulu sokubikezela. Ngokugunyazwa kwe-VONJO, sijabule ukuthi manje sesikwazi ukunikeza ukwelashwa okusha okugunyazwe ngokuqondile iziguli ezine-cytopenic myelofibrosis. Sixhaswa ngokugcwele ngezimali zokuqaliswa kwezentengiselwano, kulandela izikweletu zethu kanye nokuthengiselana kwezindlu zasebukhosini nge-DRI, futhi sibheke ngabomvu ukuhlinzeka nge-VONJO, indlela yokwelapha esezingeni eliphezulu kakhulu yeziguli ze-cytopenic myelofibrosis, ezigulini zingakapheli izinsuku eziyi-10,” kusho u-Adam R. Craig. , MD, Ph.D., uMongameli kanye noMphathi Omkhulu we-CTI Biopharma. “Ngithanda ukubonga iziguli, abanakekeli, izisebenzi zesilingo sasemtholampilo kanye nabaphenyi abenze ukuhlolwa kwemitholampilo ye-VONJO kwaba nokwenzeka. Ngiphinde ngibonge ithimba le-CTI ngokusebenza kwabo ngokuzikhandla nokuzinikela kanye nokugxila kwabo ezidingweni zeziguli.”
Ukugunyazwa okusheshisiwe kusekelwe emiphumeleni yokusebenza ngempumelelo evela ocwaningweni olubalulekile lweSigaba 3 se-PERSIST-2 lwe-VONJO ezigulini ezine-myelofibrosis (i-platelet ibala ngaphansi noma ilingana no-100 × 109/L). Iziguli zenziwe ngokungahleliwe 1:1:1 ukuze zithole i-VONJO 200 mg kabili ngosuku (BID), VONJO 400 mg kanye ngosuku (QD) noma ukwelashwa okutholakalayo okungcono kakhulu (BAT). Ukwelashwa kwangaphambili kwe-JAK2 inhibitor kwakuvunyelwe. Kulolu cwaningo, eqenjini leziguli ezine-baseline platelet count ezingaphansi kuka-50 × 109/L ezalashwa nge-pacritinib 200 mg BD, ama-29% eziguli abe nokuncipha komthamo we-spleen okungenani ngama-35% uma kuqhathaniswa ne-3% yeziguli ezitholayo. Ukwelashwa okungcono kakhulu okutholakalayo, okuhlanganisa i-ruxolitinib. Njengengxenye yokugunyazwa okusheshisiwe, i-CTI iyadingeka ukuze ichaze inzuzo yomtholampilo esivivinyweni esiqinisekisayo. Ukuze kugcwaliseke le mfuneko yangemva kokugunyazwa, i-CTI ihlela ukuqedela uhlolo lwe-PACIFICA, ngemiphumela elindelekile maphakathi no-2025.
Ukusabela okungekuhle okuvame kakhulu (≥20%) okulandela i-VONJO 200 mg kabili ngosuku kwaba isifo sohudo, i-thrombocytopenia, isicanucanu, i-anemia kanye ne-peripheral edema. Izimo ezimbi ezivame kakhulu (≥3%) ezilandela i-VONJO 200 mg kabili ngosuku kwakuyi-anemia, thrombocytopenia, inyumoniya, ukwehluleka kwenhliziyo, ukuqhubeka kwesifo, i-pyrexia kanye ne-squamous cell carcinoma yesikhumba.
LOKHO ONGAKUTHATHE KULESI SIHLOKO:
- In this study, in the cohort of patients with baseline platelet counts below 50 × 109/L who were treated with pacritinib 200 mg BD, 29% of patients had a reduction in spleen volume of at least 35% compared to 3% of patients receiving best available therapy, which included ruxolitinib.
- With the approval of VONJO, we are excited to now be able to offer a new therapy that is specifically approved for patients with cytopenic myelofibrosis.
- Food and Drug Administration (FDA) has approved VONJO (pacritinib) for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L.
