Business
Momelotinib filing accepted in Japan
Momelotinib filing accepted in Japan.
About this update from Gsk Plc
[{"type":"text","content":"\n\nIssued: 11 September 2023, London UK\n \nGSK regulatory submission for momelotinib for the treatment of myelofibrosis accepted for review by Japanese regulator\n \n· Regulatory submission included data from pivotal trials addressing key clinical manifestations of myelofibrosis, namely splenomegaly, constitutional symptoms and anaemia\n \n \n\nGSK plc (LSE/NYSE: GSK) today announced that the Ministry of Health, Labour and Welfare (MHLW), Japan, has accepted for review a new drug application (NDA) for momelotinib, a potential new medicine with a differentiated mechanism of action that may address the significant medical needs of myelofibrosis patients, especially those with anaemia. The NDA is based on data from the pivotal phase III trials SIMPLIFY-1 and MOMENTUM.\n \nMyelofibrosis is a blood cancer that can lead to splenomegaly (enlarged spleen); constitutional symptoms such as fatigue, night sweats, and bone pain; and severely low blood counts, including anaemia and thrombocytopenia.[1],[2],[3] About 70% of patients diagnosed with primary myelofibrosis and about half of patients diagnosed with secondary myelofibrosis in Japan have moderate to severe anaemia at the time of diagnosis, and nearly all patients are estimated to develop anaemia over the course of the disease.[4],[5],[6],[7],[8],[9] Patients who are transfusion dependent have a poor prognosis and shortened survival.[10],[11],[12],[13],[14],[15],[16],[17],[18]\n \nMomelotinib is not currently approved in any market.\n \nAbout momelotinib\nMomelotinib has a differentiated mechanism of action, with inhibitory ability along three key signalling pathways: Janus kinase (JAK) 1, JAK2, and activin A receptor, type I (ACVR1).[19],[20],[21],[22] Inhibition of JAK1 and JAK2 may improve constitutional symptoms and splenomegaly.19,21,22 Additionally, inhibition of ACVR1 leads to a decrease in circulating hepcidin, which is elevated in myelofibrosis and contributes to anaemia.19,20,21,22\n \nAbout myelofibrosis\nMyelofibrosis is a rare blood cancer that disrupts the body's normal production of blood cells as a result of dysregulated JAK-signal transducer and activator of transcription protein signalling. The clinical hallmarks of myelofibrosis are progressive splenomegaly (enlarged spleen), anaemia and debilitating symptoms at...