FDA Approves GSK’s Myelofibrosis Drug

The FDA has approved GSK’s Ojjaara (momelotinib) to treat adult patients with anemia who have intermediate or high-risk myelofibrosis.

Myelofibrosis is a cancer of the bone marrow and can lead to anemia. It affects about 25,000 patients in the United States, and about 40% of patients have moderate to severe anemia at the time of diagnosis. These patients often require transfusions, and those who do have a poor prognosis and shortened survival.

Ojjaara has the ability to inhibit three key signaling pathways: Janus kinase (JAK) 1, and JAK2 and activin A receptor, type I (ACVR1). Inhibition of JAK1 and JAK2 may improve symptoms and enlarged spleen, and direct inhibition of ACVR1 leads to a decrease in circulating hepcidin, which is elevated in myelofibrosis and contributes to anemia, according to GSK.

Ojjaara will be available soon, according to a GSK spokesperson. The wholesale acquisition cost is $26,900 for a bottle of 30 tablets. The spokesperson said the price of Ojjaara reflects the value it will deliver to patients, healthcare systems, and society and takes into consideration the fact it is the only medicine that addresses key manifestations of myelofibrosis, including anemia, constitutional symptoms, and splenomegaly. GSK offer access and reimbursement services for all GSK oncology products at TogetherWithGSKOncology.com. For Ojjaara, GSK offers $0 copay assistance, patient assistance for uninsured patients and a bridge program for those experiencing delays in insurance coverage.

Ruben A. Mesa, M.D.

“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,” Ruben A. Mesa, M.D., president and executive director, Atrium Health Levine Cancer Center, said in a press release.

The original Prescription Drug User Fee Act date was June 16, 2023, but the FDA added time to review additional data submitted by GSK, including data from the pivotal phase 3 MOMENTUM trial. This trial found in momelotinib improved symptoms, spleen size and anemia versus danazol in patients with symptomatic and anemic myelofibrosis who received a prior JAK inhibitor.

Results from the 24-week treatment period were presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting and published in The Lancet. The study, which compared momelotinib with danazol, found that a greater proportion of patients in the momelotinib group reported a 50% or more reduction in total symptom score than in the danazol group.

Momelotinib was developed by Sierra Oncology, which GSK acquired in July 2022.