EC Approves GSK’s Omjjara for Adults Myelofibrosis Patients Patients with Anemia

Rare Daily Staff

The European Commission approved GSK’s Omjjara for the symptoms in adult patients with myelofibrosis with moderate to severe anemia.

Myelofibrosis is a rare blood cancer that disrupts the body’s normal production of blood cells because of dysregulated JAK-signal transducer and activator of transcription protein signalling. The clinical hallmarks of myelofibrosis are enlarged spleen, progressive anemia, and debilitating constitutional symptoms, such as fatigue, night sweats and bone pain, attributable to ineffective hematopoiesis and excessive production of proinflammatory cytokines.

Omjjara, a once-a-day, oral JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor, is the first authorized medicine in the EU for disease-related enlarged spleen or symptoms in adult patients with moderate to severe anemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are JAK inhibitor naïve or have been treated with ruxolitinib.

“The challenges of living with myelofibrosis can be burdensome, and symptomatic patients can experience spleen enlargement, fatigue, night sweats and bone pain,” said Nina Mojas, senior vice president of Oncology Global Product Strategy for GSK. “Until now, there have been no options specifically indicated to treat these symptoms in patients who also experience anemia. The authorization of Omjjara brings a new treatment option with a differentiated mechanism of action to these patients in the E.U.”

Myelofibrosis is estimated to affect 1 in 10,000 people in the European Union. About 40 percent 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, require additional supportive care, including transfusions, and more than 30 percent will discontinue treatment due to anemia. Patients who are transfusion dependent have a poor prognosis and shortened survival.

The approval of momelotinib is based on the MOMENTUM pivotal phase 3 trial and a subpopulation of adult patients with moderate to severe anemia from the SIMPLIFY-1 phase 3 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. 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.

In these clinical trials, the most common adverse reactions were diarrhea, thrombocytopenia, nausea, headache, dizziness, fatigue, asthenia, abdominal pain, and cough.

In September 2023, the U.S. Food and Drug Administration approved momelotinib under the brand name Ojjaara 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.