MISSISSAUGA, Canada – GSK announced that Health Canada has accepted into review the New Drug Submission (NDS) for momelotinib, a potential new medicine with a differentiated mechanism of action that may help address the significant medical needs of myelofibrosis patients, especially those with anemia. The NDS is based on key phase III trials SIMPLIFY-1 and MOMENTUM.
Myelofibrosis is a rare 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 anemia and thrombocytopenia. Approximately 82% of patients diagnosed with myelofibrosis are intermediate or high-risk and about one-third to one-half of patients are anemic at the time of diagnosis. The majority of myelofibrosis patients are expected to develop anemia over the course of the disease, and those who are transfusion dependent have a poor prognosis and shortened survival.
Momelotinib is an investigational product that is not currently authorized for sale in Canada.
About momelotinib
Momelotinib has a differentiated mechanism of action, with inhibitory ability along three key signaling pathways: Janus kinase (JAK) 1, JAK2, and activin A receptor, type I (ACVR1). Inhibition of JAK1 and JAK2 may improve constitutional symptoms and splenomegaly. Additionally, inhibition of ACVR1 leads to a decrease in circulating hepcidin, which is elevated in myelofibrosis and contributes to anemia.
About myelofibrosis
Myelofibrosis 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 signaling. The clinical hallmarks of myelofibrosis are progressive splenomegaly (enlarged spleen), anemia and debilitating symptoms attributable to ineffective hematopoiesis and excessive production of proinflammatory cytokines. Myelofibrosis patients with anemia require additional supportive care, including transfusions, and have poor outcomes. An estimated 1,400-2,177 patients are living with myelofibrosis in Canada with an estimated 3 in 500,000 people worldwide affected.
About the pivotal SIMPLIFY-1 clinical trial
SIMPLIFY-1 was a global randomized, double-blind, phase III study that compared the safety and efficacy of momelotinib to ruxolitinib in patients with myelofibrosis who had not received prior treatment with a JAK inhibitor.
About the pivotal MOMENTUM clinical trial
MOMENTUM was a global, randomized, double-blind phase III clinical trial that compared the safety and efficacy of momelotinib to danazol in patients with myelofibrosis who were symptomatic and anemic and had been previously treated with an approved JAK inhibitor. The trial was designed to evaluate the safety and efficacy of momelotinib for treating and reducing key hallmarks of the disease: symptoms, blood transfusions (due to anemia) and splenomegaly.
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