FDA Grants Green Light to GSK’s Ojjaara: A Game-Changer for Myelofibrosis Patients Battling Anemia!
In a monumental decision on Friday, the FDA gave the nod to GSK’s groundbreaking oral drug, momelotinib, now to be known as Ojjaara, for the treatment of myelofibrosis in adults suffering from anemia.
Myelofibrosis patients often grapple with anemia, a cruel side effect that forces them to halt their treatment and rely on transfusions. GSK’s Ojjaara is set to answer this critical medical need, bringing new hope and improved outcomes for these patients, as declared by Nina Mojas, GSK’s Senior Vice President for Oncology Global Product Strategy.
With this historic approval, Ojjaara has earned the distinction of being the first authorized treatment for both newly diagnosed and previously treated myelofibrosis patients with anemia. It not only targets the key symptoms of the condition but also promises a brighter future, according to GSK’s press release.
This victory follows a three-month delay in June 2023, during which the FDA took extra time to scrutinize additional data supporting the application. Importantly, momelotinib has not yet gained approval in any other market.
Ojjaara, an oral medication, boasts a unique mechanism of action, effectively inhibiting three vital signaling pathways: JAK1, JAK2, and the ACVR1 pathway. By taming JAK1 and JAK2, Ojjaara eases splenomegaly and enhances patients’ overall well-being. Its ACVR1 activity is equally impressive, as it reduces hepcidin levels—a key driver of anemia.
The journey of this remarkable molecule began with Sierra Oncology, later acquired by GSK for a whopping $1.9 billion in 2022.
Friday’s approval stems from the Phase III MOMENTUM trial, which revealed Ojjaara’s remarkable treatment responses, including relief from constitutional symptoms, improved spleen function, and reduced reliance on transfusions compared to the synthetic steroid danazol. GSK bolstered these findings with results from the adult subpopulation of the Phase III SIMPLIFY-1 study, pitting Ojjaara against ruxolitinib in JAK inhibitor-naïve patients.
Ojjaara’s label recommends a daily oral dose of 200 mg and covers adult patients with intermediate- or high-risk myelofibrosis, whether it’s primary or secondary. While the drug bears no boxed warning, it comes with precautions regarding thrombocytopenia, neutropenia, hepatotoxicity, cardiac events, and secondary malignancies, especially for current or former smokers.
This triumph for GSK follows another recent milestone in oncology. Just last month, the FDA expanded the label for Jemperli (dostarlimab-gxly) to include primary advanced or recurrent endometrial cancer, marking it as the first immuno-oncology asset and PD-1 inhibitor greenlit for frontline use in this indication.
This achievement puts Jemperli slightly ahead of Merck’s blockbuster Keytruda (pembrolizumab), which is approved for endometrial cancer but only in later-line settings.