FDA approves GSK’s momelotinib for myelofibrosis patients with anemia
Published: Sep 18, 2023
By Tristan Manalac
Myelofibrosis patients often develop anemia, which forces them to discontinue treatment and raise the need for transfusions. Ojjaara’s approval will help address this “significant medical need in the community” and lead to better outcomes in these patients, Nina Mojas, GSK senior vice president for oncology global product strategy, said in a statement.
With Friday’s approval, Ojjaara becomes the first authorized treatment for both newly diagnosed and previously treated myelofibrosis patients with anemia that also targets the key symptoms of the condition, according to the company’s press release.
The approval comes after a three-month delay in June 2023, which the FDA said was to give it more time to review additional data supporting the application. Momelotinib has not yet been approved in any other market.
Ojjaara is an oral medication that has a “differentiated mechanism of action” and exhibits inhibitory effects along three key signaling pathways: the JAK1 and JAK2 cascades and the ACVR1 pathway. By suppressing JAK1 and JAK2, Ojjaara alleviates splenomegaly and improves patients’ constitution. This is complemented by its ACVR1 activity, which lowers hepcidin levels—a key driver of anemia.
The molecule was most recently developed by Sierra Oncology, which GSK bought for $1.9 billion in 2022.
Friday’s approval was based on data from the Phase III MOMENTUM trial, which showed that Ojjaara elicited significant treatment response in terms of constitutional symptoms, splenic response and transfusion independence, as compared with the synthetic steroid danazol. GSK supplemented these data with results from the adult subpopulation of the Phase III SIMPLIFY-1 study, which compared Ojjaara against ruxolitinib in JAK inhibitor-naïve patients.
Ojjaara’s label recommends a 200-mg oral dose taken once-daily and covers patients with intermediate- or high-risk myelofibrosis, including primary or secondary disease, in adults suffering from anemia. The drug has no boxed warning but carries precautions against thrombocytopenia and neutropenia, hepatotoxicity, cardiac events and secondary malignancies, particularly in current or past smokers.
Friday’s regulatory approval follows another recent oncology victory for GSK. Last month, the FDA expanded Jemperli’s (dostarlimab-gxly) label to cover primary advanced or recurrent endometrial cancer, making it the first immuno-oncology asset and PD-1 inhibitor to be cleared for the frontline use in this indication.
This approval gives Jemperli the slight edge over Merck’s blockbuster asset Keytruda (pembrolizumab), which is also approved for endometrial cancer—but only in later-line settings.