I le kuata lona lua o le 2021, na tuʻuina atu ai e le FDA le faʻamuamua iloiloga mo le CTI's NDA mo tagata mamaʻi e maua i le myelofibrosis ma le PDUFA aso o Novema 30, 2021. I le faagasologa o faʻatalanoaga o igoa o oloa, na talosagaina e le FDA ni faʻamatalaga faʻapitoa faʻapitoa, lea na tuʻuina atu i le lala sooupu. i le aso 24 o Novema, 2021. I le amataga o aso nei, na logoina ai e le FDA le Kamupani e manatu o le tuʻuina atu o faʻamatalaga e fai ma "teuteuga tele" i le NDA ma o le mea lea ua faʻaopopoina ai le aso PDUFA i le tolu masina e tuʻuina atu ai se taimi faaopoopo mo se iloiloga atoa o le tuuina atu. I le taimi nei, e le o iloa e le CTI ni fa'aletonu tetele i le talosaga.

O le Pacritinib o se faʻamaʻi tautala kinase faʻapitoa mo le JAK2, IRAK1 ma le CSF1R, e aunoa ma le taofia o le JAK1. O le NDA na taliaina e faʻavae i luga o faʻamaumauga mai le Vaega 3 PERSIST-2 ma le PERSIST-1 ma le Vaega 2 PAC203 faʻataʻitaʻiga faʻataʻitaʻiga, faʻatasi ai ma le taulaʻi atu i le ogaoga o le thrombocytopenic (platelet faitau i lalo ifo o le 50 x 109 / L) maʻi na lesitala i nei suʻesuʻega oe na mauaina le pacritinib 200 mg faalua i le aso, e aofia uma ai le pito i luma o togafitiga-naive gasegase ma tagata mamaʻi na muamua faʻaalia i le JAK2 inhibitors. I le suʻesuʻega PERSIST-2, i tagata mamaʻi e maua i le thrombocytopenia ogaoga o loʻo togafitia i le pacritinib 200 mg faalua i le aso, 29% o tagata mamaʻi na faʻaititia le maualuga o le spleen e le itiiti ifo i le 35%, pe a faʻatusatusa i le 3% o tagata mamaʻi o loʻo maua togafitiga sili ona lelei. , lea e aofia ai le ruxolitinib; 23% o tagata mama'i na fa'aitiitia le aofa'i o fa'ailoga e le itiiti ifo i le 50%, fa'atusatusa i le 13% o tagata mama'i o lo'o maua togafitiga sili ona lelei. I le faitau aofaʻi lava o tagata mamaʻi na togafitia i le pacritinib, o faʻalavelave faʻalavelave e masani lava ona maualalo le vasega, faʻaogaina ma le tausiga lagolago, ma e seasea taitai atu i le faʻagata. Sa fa'amautu fo'i le aofa'i o platelet ma le maualuga o le hemoglobin.

O le Myelofibrosis o le kanesa o le ga'o ponaivi lea e mafua ai le fa'atupuina o le ma'ila ma'ila ma e mafai ona o'o atu ai i le thrombocytopenia ma le anemia, vaivai, vaivai ma le fa'ateleina o le toto ma le ate. I totonu o le US e tusa ma le 21,000 tagata gasegase e maua i le myelofibrosis, 7,000 o loʻo i ai le thrombocytopenia ogaoga (faʻamatalaina o le toto platelet e itiiti ifo i le 50 x109/L). O le ogaoga o le thrombocytopenia e fesoʻotaʻi ma le le lelei o le ola ma le maualuga o faʻamaʻi avega ma e mafai ona tupu ona o le alualu i luma o faʻamaʻi poʻo le faʻamaʻi o fualaau faʻamaʻi faʻatasi ma isi inhibitors JAK2 e pei ole JAKAFI ma INREBIC.

OA MEA E AVEA MAI LENEI TUSI:

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.