GSK plc (LSE/NYSE: GSK) today announced that the US Food and Drug Administration (FDA) has approved Ojjaara (momelotinib) for the treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis (post-polycythemia vera and post-essential thrombocythemia), in adults with anemia. Ojjaara is a once-a-day, oral JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor. To date, it is the only approved medicine for both newly diagnosed and previously treated myelofibrosis patients with anemia that addresses the key manifestations of the disease, namely anemia, constitutional symptoms, and splenomegaly (enlarged spleen).4
Nina Mojas, Senior Vice President, Oncology Global Product Strategy, GSK, said: “The vast majority of myelofibrosis patients eventually develop anemia, causing them to discontinue treatments and require transfusions. Given this high unmet need, we are proud to add Ojjaara to our oncology portfolio and address a significant medical need in the community. We look forward to helping improve outcomes in this difficult-to-treat blood cancer.”
Myelofibrosis is a blood cancer affecting approximately 25,000 patients in the US.4,5,6 Myelofibrosis can lead to severely low blood counts, including anemia and thrombocytopenia; constitutional symptoms such as fatigue, night sweats, and bone pain; and splenomegaly. About 40% of patients have moderate to severe anemia at the time of diagnosis, and nearly all patients are estimated to develop anemia over the course of the disease.7,8,9,10 Physicians have had limited treatment options to treat myelofibrosis patients with anemia. These patients often require transfusions and more than 30% will discontinue treatment due to anemia.3 Patients who are transfusion dependent have a poor prognosis and shortened survival.1,11,12,13,14,15,16,17,18
Ruben A. Mesa, MD, FACP, President and Executive Director, Atrium Health Levine Cancer Center and Atrium Health Wake Forest Baptist Comprehensive Cancer Center, said: “With momelotinib we have the potential to establish a new standard of care for myelofibrosis patients with anemia. Addressing key manifestations of myelofibrosis, including anemia, constitutional symptoms and splenomegaly, makes a significant difference in the treatment regimen for these patients who have limited options to address these aspects of the disease.”
The FDA approval of momelotinib is supported by data from the pivotal MOMENTUM study and a subpopulation of adult patients with anemia from the SIMPLIFY-1 phase III trial. MOMENTUM was designed to evaluate the safety and efficacy of momelotinib versus danazol for the treatment and reduction of key manifestations of myelofibrosis in an anemic, symptomatic, JAK inhibitor-experienced population. The MOMENTUM trial met all its primary and key secondary endpoints, demonstrating statistically significant response with respect to constitutional symptoms, splenic response and transfusion independence, in patients treated with momelotinib versus danazol.2 SIMPLIFY-1 was designed to evaluate the efficacy and safety of momelotinib versus ruxolitinib in myelofibrosis patients who had not received a prior JAK-inhibitor therapy.1 Safety and efficacy results for SIMPLIFY-1 were based upon a subset of patients with anemia.
In these clinical trials, the most common adverse reactions were thrombocytopenia, hemorrhage, bacterial infection, fatigue, dizziness, diarrhea, and nausea.19
Kapila Viges, Chief Executive Officer, MPN (Myeloproliferative Neoplasms) Research Foundation, said: “We are thrilled to see momelotinib reach the clinic, giving patients and their physicians another option to help manage myelofibrosis. Any new treatment that takes steps toward unlocking the mysteries of this complex and chronic blood cancer represents great progress for the field.”
Momelotinib is currently not approved in any other market.
Source link : https://www.businesswire.com/
