Ojjaara Approved for Intermediate or High-Risk Myelofibrosis With Anemia

The Food and Drug Administration (FDA) has approved Ojjaara (momelotinib) for the treatment of intermediate or high-risk myelofibrosis (MF), including primary MF or secondary MF (post-polycythemia vera [PV] and post-essential thrombocythemia [ET]), in adults with anemia.

Momelotinib is designed to inhibit 3 signaling pathways including Janus kinase (JAK) 1, JAK2, and activin A receptor type 1 (ACVR1). Inhibition of JAK1 and JAK2 is expected to improve constitutional symptoms and splenomegaly, while inhibition of ACVR1 leads to subsequent inhibition of liver hepcidin expression and increased iron availability resulting in increased red blood cell production.

The approval was based on data from the phase 3 MOMENTUM (ClinicalTrials.gov Identifier: NCT04173494) and SIMPLIFY-1 (ClinicalTrials.gov Identifier: NCT01969838) trials. 

In MOMENTUM, the efficacy and safety of momelotinib was compared with danazol in 195 adults with symptomatic and anemic MF who were previously treated with a JAK inhibitor. Findings showed that the trial met its primary and all key secondary endpoints, demonstrating statistically significant response for constitutional symptoms, splenic response, and transfusion independence. 

In SIMPLIFY-1, momelotinib was compared with ruxolitinib in a subpopulation of adults with MF who had not previously received a JAK inhibitor. Findings showed that 31.4% (95% CI, 21.8-42.3) of patients treated with momelotinib met the primary endpoint achieving a splenic volume reduction of at least 35% vs 32.6% (95% CI, 23.4-43.0) of those treated with ruxolitinib.

The most common adverse reactions reported with momelotinib were thrombocytopenia, hemorrhage, bacterial infection, fatigue, dizziness, diarrhea, and nausea.

“The vast majority of myelofibrosis patients eventually develop anemia, causing them to discontinue treatments and require transfusions,” said Nina Mojas, Senior Vice President, Oncology Global Product Strategy, GSK. “Given this high unmet need, we are proud to add Ojjaara to our oncology portfolio and address a significant medical need in the community.”

Ojjaara is supplied as 100mg, 150mg, and 200mg tablets in 30-count bottles.