The FDA approved Ojjaara for intermediate or high-risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis (post-polycythaemia vera and post-essential thrombocythaemia), in adults with anemia.
The Food and Drug Administration (FDA) approved Ojjaara (momelotinib) for the treatment of myelofibrosis with anemia, according to GSK, the manufacturer of the drug.
“Approximately one out of every three patients have anemia at the time of their myelofibrosis diagnosis. We also know that individuals who have chronic anemia are at a much higher risk for a poor prognosis. (Ojjaara) provides incremental benefits and is now another treatment option available to patients whose disease has stopped responding to (Jakafi [ruxolitinib]) and need treatments for anemia,” Dr. Lee Greenberger, chief scientific officer at the Leukemia & Lymphoma Society said in an interview with CURE.
The approval is based on findings from the phase 3 MOMENTUM trial, which included 195 adults with primary myelofibrosis or post-polycythemia vera or post-essential thrombocythemia myelofibrosis who were symptomatic and previously treated with an FDA-approved JAK inhibitor. Two thirds of the patients (130 patients) were randomly assigned to receive Ojjaara plus Danocrine (danazol), while the other third (65 patients) was assigned to receive Danocrine plus a placebo.
Study findings, which were published in The Lancet, showed that a larger percentage of patients in the Ojjaara group experienced a 50% or more reduction in tumor symptom score compared to those in the Danocrine/placebo group (25% versus 9%, respectively). Of note, the tumor symptom scores consider factors such as the need for blood transfusions due to anemia and enlarged spleen, which is a common — and potentially dangerous — issue for patients with myelofibrosis.
After 24 weeks, all patients on the trial were eligible to receive Ojjaara and 93 (72%) and 41 (63%) in the Ojjaara and Danocrine-only groups, respectively, entered the Ojjaara open-label extension period. Among patients who continued with the drug, 30 (45%) and 15 (50%) in the Ojjaara and Danocrine groups, respectively, responded to therapy based on the total symptom score criteria.
“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,” Dr. Ruben A. Mesa, President and Executive Director, Atrium Health Levine Cancer Center and Atrium Health Wake Forest Baptist Comprehensive Cancer Center, said in a press release issued by GSK.
Ojjaara works by inhibiting JAK1 and JAK2, which are proteins that are involved in cell growth and survival, and are key players in myelofibrosis, a type of myeloproliferative neoplasms that disrupts the typical production of blood cells. Research has shown that inhibiting these proteins could decrease symptoms, such as spleen enlargement, that are associated with the disease. The drug also inhibits activin A receptor, type I (ACVR1), a receptor that is elevated in patients with myelofibrosis and could lead to anemia.
Long-term follow-up from MOMENTUM revealed no new side effects with the most common non-blood-related side effects from the drug being diarrhea (45 patients [26%] in the Ojjaara group) and weakness or lack of energy (28 [16%]). The most common moderate to severe (grade 3 or 4) side effects were thrombocytopenia (33 [19%]) and anemia (30 [18%]). A total of 79 patients (46%) given Ojjaara experienced at least one serious side effect, and 30 patients (18%) died from a treatment-emergent side effect, with two fatal treatment-emergent side effects considered to be potentially linked to Ojjaara.
“The study that led to this approval showed that (Ojjaara) was superior to danazol across a variety of outcomes, including a significant improvement in anemia. (Ojjaara) is the first approval of a new agent that may be useful in the treatment of anemia in patients with myelofibrosis that can also control the myelofibrosis,” Greenberger concluded.
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