Ngekota yesibili ka-2021, i-FDA inikeze ukubuyekezwa okubalulekile kwe-CTI's NDA ezigulini ezine-myelofibrosis enedethi ye-PDUFA yangomhla zingama-30 Novemba 2021. Ngesikhathi sezingxoxo zokulebula umkhiqizo, i-FDA yacela idatha yomtholampilo eyengeziwe, eyathunyelwa ejensi. ngoNovemba 24, 2021. Ngaphambilini namuhla, i-FDA yazisa Inkampani ukuthi ibheka ukuthunyelwa kwedatha ukwenza “isichibiyelo esikhulu” ku-NDA futhi ngenxa yalokho usuku lwe-PDUFA lunwetshwe ngezinyanga ezintathu ukuze kunikezwe isikhathi esengeziwe sokubuyekeza okugcwele. ukuhambisa. Okwamanje, i-CTI ayazi noma yikuphi ukushiyeka okukhulu esicelweni.
I-Pacritinib iyinoveli ye-oral kinase inhibitor enemininingwane ye-JAK2, IRAK1 ne-CSF1R, ngaphandle kokuvimbela i-JAK1. I-NDA yamukelwe ngokusekelwe kudatha evela kuSigaba 3 se-PERSIST-2 ne-PERSIST-1 kanye neSigaba 2 sokuhlolwa komtholampilo kwe-PAC203, ngokugxila ku-thrombocytopenic enzima (i-platelet ibala ngaphansi kuka-50 x 109/L) iziguli ezibhalise kulezi zifundo. owathola i-pacritinib engu-200 mg kabili ngosuku, okuhlanganisa kokubili iziguli ezihamba phambili ezingenalwazi kanye neziguli ezichayeke ngaphambili kuma-JAK2 inhibitors. Ocwaningweni lwe-PERSIST-2, ezigulini ezine-thrombocytopenia enzima ezalashwa nge-pacritinib 200 mg kabili ngosuku, ama-29% eziguli abe nokuncipha komthamo we-spleen okungenani ngama-35%, uma kuqhathaniswa ne-3% yeziguli ezithola ukwelashwa okungcono kakhulu. , okwakuhlanganisa i-ruxolitinib; Ama-23% eziguli abe nokuncipha kwenani lezimpawu eziphelele okungenani ngo-50%, uma kuqhathaniswa no-13% weziguli ezithola ukwelashwa okungcono kakhulu okutholakalayo. Emphakathini ofanayo weziguli eziphathwa nge-pacritinib, izehlakalo ezimbi ngokuvamile zazisezingeni eliphansi, zilawuleka ngokunakekelwa okusekelayo, futhi kwakungavamile ukuholela ekuyekisweni. Izibalo zamaplatelet namazinga e-hemoglobin nawo azinza.
I-Myelofibrosis umdlavuza womnkantsha oholela ekwakhekeni kwezicubu ezibomvu ze-fibrous futhi ungaholela ku-thrombocytopenia kanye ne-anemia, ubuthakathaka, ukukhathala kanye nokwanda kwe-spleen nesibindi. E-US kuneziguli ezingaba ngu-21,000 ezine-myelofibrosis, ezingu-7,000 zazo ezine-thrombocytopenia enzima (echazwa njengezibalo zeplatelet yegazi ezingaphansi kwama-50 x109/L). I-thrombocytopenia enzima ihlotshaniswa nokuphila okungalungile kanye nomthwalo ophezulu wezimpawu futhi kungenzeka ngenxa yokuqhubeka kwesifo noma ubuthi bezidakamizwa namanye ama-JAK2 inhibitors afana ne-JAKAFI ne-INREBIC.
LOKHO ONGAKUTHATHE KULESI SIHLOKO:
- The NDA was accepted based on the data from the Phase 3 PERSIST-2 and PERSIST-1 and the Phase 2 PAC203 clinical trials, with a focus on the severely thrombocytopenic (platelet counts less than 50 x 109/L) patients enrolled in these studies who received pacritinib 200 mg twice a day, including both frontline treatment-naive patients and patients with prior exposure to JAK2 inhibitors.
- In the PERSIST-2 study, in patients with severe thrombocytopenia who were treated with pacritinib 200 mg twice a day, 29% of patients had a reduction in spleen volume of at least 35%, compared to 3% of patients receiving the best available therapy, which included ruxolitinib.
- In the second quarter of 2021, the FDA granted priority review for CTI’s NDA for patients with myelofibrosis with a PDUFA date of November 30, 2021.
