The European Commission has granted marketing authorization for Omjjara* (momelotinib) – an inhibitor of Jak1/Jak2 and activin A receptor type 1 (Acvr1) – for the treatment of splenomegaly (enlarged spleen) or symptoms related in adult patients with moderate to severe anemia suffering from primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis, never treated (naïve) with Janus kinase (Jak) inhibitors or who have already been treated with ruxolitinib. GSK announced this in a note, in which it underlines that the oral therapy to be taken once a day is the only drug in the European Union indicated for patients suffering from newly diagnosed or previously treated myelofibrosis, with moderate-severe anemia and symptoms of splenomegaly.
“People with myelofibrosis – says Nina Mojas, Senior Vice President, Oncology Global Product Strategy, GSK – may experience severe symptoms such as enlarged spleen, fatigue, night sweats and bone pain. Until now, there have been no options specifically indicated to treat these clinical manifestations in patients who also suffer from anemia. The authorization of Omjjara brings a new therapeutic option for European patients, with a differentiated mechanism of action.”
Myelofibrosis is a rare blood cancer in which the activity of Jak enzymes is impaired. Momelotinib has a differentiated mechanism of action that inhibits three key signaling pathways: those of Jak1 and Jak2, improving symptoms and splenomegaly; blocks the activin A type I receptor leading to a decrease in circulating hepcidin levels, potentially contributing to the anemia benefit. It is estimated that myelofibrosis affects 1 in 10,000 Europeans. Approximately 40% of patients have moderate to severe symptoms and anemia at diagnosis, but nearly all develop anemia over the course of the disease. Myelofibrosis patients with anemia require additional supportive care, such as transfusions, and more than 30% discontinue treatment due to anemia. Transfusion-dependent patients have a worse prognosis and reduced survival.
“The authorization of Omjjara in the EU – underlines Francesca Palandri of the Irccs S. Orsola-Malpighi-University of Bologna hospital – represents a significant progress for patients suffering from myelofibrosis and, in particular, for subjects suffering from moderate to serious that require new therapeutic options that can reduce the impact of the disease. The availability of a single therapy for the main manifestations of myelofibrosis represents a clear step forward for these patients.”
The authorization of momelotinib is based on the pivotal phase 3 Momentum study and a subpopulation of adult patients with moderate to severe anemia (hemoglobin <10 g/dl) from the phase 3 Simplify-1 study. The Momentum study was designed to evaluate the safety and efficacy of momelotinib compared to danazol for the treatment and reduction of the main manifestations of myelofibrosis in an anemic, symptomatic population already treated with Jak enzyme inhibitors. Simplify-1 was designed to evaluate the efficacy and safety of momelotinib versus ruxolitinib in myelofibrosis in patients who had not received prior Jak inhibitor therapy. In these clinical trials, the most common adverse reactions were diarrhea, thrombocytopenia, nausea, headache, dizziness, fatigue, asthenia, abdominal pain and cough.
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