FDA Approves Momelotinib for Patients With Intermediate or High-Risk Myelofibrosis, Including Primary or Secondary MF, With Anemia

On September 15, 2023, the FDA granted approval to momelotinib, an oral Janus kinase (JAK)1/JAK2 inhibitor and activin A receptor type 1 (ACVR1) inhibitor, for patients with intermediate or high-risk myelofibrosis (MF), including patients with primary and secondary MF, and with anemia. 

Momelotinib is currently the first and only approved medicine for both newly diagnosed and previously treated patients with myelofibrosis and anemia, also addressing manifestations of the disease such as constitutional symptoms and splenomegaly. 

Ruben A Mesa, MD, FACP, Atrium Health Levine Cancer Center, and Atrium Health Wake Forest Baptist Comprehensive Cancer Center, Charlotte, North Carolina, stated in a press release, “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.”

This approval was based on findings from the pivotal MOMENTUM trial, as well as a subset of adult patients with anemia from the SIMPLIFY-1 phase 3 trial. The MOMENTUM trial demonstrated a statistically significant response among patients with MF who were symptomatic, anemic, had previously been treated with an approved JAK inhibitor, and who received momelotinib compared to danazol. These findings indicated the efficacy of momelotinib in treating key manifestations of myelofibrosis, such as symptoms, blood transfusions due to anemia, and splenomegaly, among this patient population. 

The phase 3 SIMPLIFY trial compared the safety and efficacy of momelotinib with ruxolitinib among patients with MF who had not previously undergone treatment with a JAK inhibitor. The efficacy of momelotinib was assessed based on the drug’s impact on spleen volume response. 

The most common adverse events observed in both clinical trials were thrombocytopenia, hemorrhage, bacterial infection, fatigue, dizziness, diarrhea, and nausea.

Source: 

Ojjaara (momelotinib) approved in the US as the first and only treatment indicated for myelofibrosis patients with anaemia; GSK. Published online September 15, 2023. https://www.gsk.com/en-gb/media/press-releases/ojjaara-momelotinib-approved-in-the-us-as-the-first-and-only-treatment-indicated-for-myelofibrosis-patients-with-anaemia/#:~:text=GSK%20plc%20