FDA Approves GSK’s Ojjaara for Rare Blood Cancer

Rare Daily Staff

The U.S. Food and Drug Administration has approved GSK’s Ojjaara for the treatment of intermediate or high-risk myelofibrosis in adults with anemia.

The approval includes primary myelofibrosis or secondary myelofibrosis, post-polycythemia, and post-essential thrombocythemia.

Myelofibrosis is a rare blood cancer affecting approximately 25,000 patients in the United States, which can lead to severely low blood counts, including anemia and thrombocytopenia; constitutional symptoms such as fatigue, night sweats, and bone pain; and splenomegaly (enlarged spleen). 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. Physicians have had limited treatment options to treat myelofibrosis patients with anemia. These patients often require transfusions, and more than 30 percent will discontinue treatment due to anemia.​ Patients who are transfusion dependent have a poor prognosis and shortened survival.

Ojjaara is a once-a-day, oral JAK1/JAK2 and activin A receptor type 1 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.

“The vast majority of myelofibrosis patients eventually develop anemia, causing them to discontinue treatments and require transfusions,” said Nina Mojas, senior vice president of Oncology Global Product Strategy for GSK.

The FDA approval of Ojjaara is supported by data from the pivotal MOMENTUM study and a subpopulation of adult patients with anemia from the SIMPLIFY-1 phase 3 trial. MOMENTUM was designed to evaluate the safety and efficacy of Ojjaara 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 Ojjaara versus danazol. SIMPLIFY-1 was designed to evaluate the efficacy and safety of Ojjaara versus ruxolitinib in myelofibrosis patients who had not received a prior JAK-inhibitor therapy. 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.

Photo: Nina Mojas, senior vice president of Oncology Global Product Strategy for GSK