Danazol

The drug is for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are Janus kinase (JAK) inhibitor naïve or have been treated with ruxolitinib. Professor Claire Harrison, deputy chief medical officer in research, data, and analytics, at Guy’s and St Thomas’, said: “Myelofibrosis is a rare blood cancer with limited treatment options. The approval of Omjjara (momelotinib) is the culmination of many years of collaboration and participation by hundreds of patients in clinical trials. We are very grateful to those who took part as it has enabled this new option for treating myelofibrosis patients with moderate to severe anaemia.” Myelofibrosis is a rare type of blood marrow cancer that disrupts the body’s normal production of blood cells. In its research, GSK found that every year, approximately 380 people are diagnosed with myelofibrosis in the UK. It can lead to splenomegaly, constitutional symptoms such as fatigue, night sweats, and bone pain, as well as low blood counts, including anaemia (deficiency of red blood cells) and thrombocytopenia (deficiency of platelets). The company said nearly all people living myelofibrosis are estimated to develop anaemia over the course of the disease, and may require additional supportive care, including red blood cell (RBC) transfusions. googletag.cmd.push(function () { googletag.display('text-ad1'); }); Innovation for unmet clinical needs Jack Harris, vice-president UK Oncology, at GSK, said: “We are committed to driving innovation for those living with unmet needs. We welcome today’s decision. As the first JAK-inhibitor treatment licensed in Great Britain specifically indicated for adult myelofibrosis patients with moderate to severe anaemia, this milestone could have an impact on this patient population living with myelofibrosis across Great Britain. We look forward to working with all stakeholders to help allow eligible myelofibrosis patients to access this treatment on the NHS as soon as possible.” This MA approval is supported by data from the Momentum phase 3 trial and a subpopulation of adult patients with moderate to severe anaemia (haemoglobin <10 g/dL) from the Simplify-1 phase III trial. The Momentum trial was designed to evaluate the safety profile and efficacy of momelotinib versus danazol for the key manifestations of myelofibrosis (such as splenomegaly, constitutional symptoms, and RBC transfusions) in an anaemic, symptomatic, JAK inhibitor-experienced population. The SIMPLIFY-1 trial was designed to evaluate the efficacy and safety profile of momelotinib versus ruxolitinib in myelofibrosis patients who had not received a prior JAK inhibitor therapy.4

[30-January-2024] Health Canada's acceptance of the submission follows that of the Ministry of Health, Labour and Welfare (WHLW) in Japan and approval by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Regulatory submission includes data from pivotal trials addressing key clinical manifestations of myelofibrosis, namely splenomegaly, constitutional symptoms and anemia. MISSISSAUGA ON, Jan. 30, 2024 /CNW/ - GSK announces that Heath 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. [1][2][3] Approximately 82% [4] 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. [5][6][7] 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. [8][9][10][11][12][13][14][15][16] 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). [17][18][19][20] Inhibition of JAK1 and JAK2 may improve constitutional symptoms and splenomegaly. [17][19][20] Additionally, inhibition of ACVR1 leads to a decrease in circulating hepcidin, which is elevated in myelofibrosis and contributes to anemia. [17][18][19][20] 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. [21] Myelofibrosis patients with anemia require additional supportive care, including transfusions, and have poor outcomes. [22][23] An estimated 1,400-2,177 patients are living with myelofibrosis in Canada with an estimated 3 in 500,000 people worldwide affected. [24][25] 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. GSK in oncology GSK is committed to maximizing patient survival through transformational medicines, with a current focus on breakthroughs in immuno-oncology and tumor-cell targeting therapies, and development in hematologic malignancies, gynecologic cancers and other solid tumors. Our goal is to achieve a sustainable flow of new treatments based on a diversified portfolio of investigational medicines utilizing modalities such as small molecules, antibodies, antibody-drug conjugates, and cell therapy, either alone or in combination. About GSK GSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. For further information please visit . GSK Media Enquiries +1 855 593 6274 References SOURCE GlaxoSmithKline Inc.