GSK's Omjjara (momelotinib) approved in Singapore as the first treatment indicated for myelofibrosis patients with anaemia
PR Newswire
8 min read
Approval is for use in myelofibrosis patients with moderate to severe anaemia who are JAK-naive or previously treated with ruxolitinib
Nearly all myelofibrosis patients globally are estimated to develop anaemia over the course of the disease, and over 30% will discontinue treatment due to anaemia[1-3]
Omjjara addresses the key manifestations of myelofibrosis, specifically anaemia, constitutional symptoms, and splenomegaly
SINGAPORE, March 24, 2025 /PRNewswire/ -- GSK Singapore today announced that Omjjara (momelotinib) has been locally approved for treatment of disease-related splenomegaly or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are Janus Kinase (JAK) inhibitor naive or have been treated with ruxolitinib.[4]
Omjjara is a once-a-day, oral JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor.[4]
Myelofibrosis can lead to low blood counts, including anaemia and thrombocytopaenia; constitutional symptoms such as fatigue, night sweats, and bone pain; and splenomegaly. About 40% of patients globally have moderate to severe anaemia at the time of diagnosis, and nearly all patients are estimated to develop anaemia over the course of the disease.[5-8] These patients may become transfusion-dependent and more than 30% will discontinue treatment due to anaemia.[3]Patients who are transfusion-dependent are often associated with a poor quality of life and have a poor prognosis with shortened survival.[1,9-16]
Earlier treatment options for managing myelofibrosis-related anaemia have demonstrated limited efficacy and durability of response; and while JAK inhibitors are often used, they can sometimes exacerbate anaemia.[17] This highlights an unmet need for effective treatments for myelofibrosis patients with anaemia.[17]
Dr. Stephanie Ambrose, GSK Singapore Medical Director, said: "We note that myelofibrosis is a complex disease and there are significant challenges in the management of the disease, particularly for patients with anaemia and splenomegaly. We look forward to working with the myelofibrosis community and making a difference by improving the outcome for and quality of life of the patients."
Clinical Professor Goh Yeow Tee, Senior Consultant, Department of Haematology, Singapore General Hospital (SGH), who was involved in both the MOMENTUM and SIMPLIFY trials, said: "SGH has been able to help bring new treatment options to our patients through collaborations in clinical studies with global healthcare providers and industry partners. With approximately 30 new myelofibrosis cases each year, we welcome this additional treatment option which could potentially reduce patients' dependence on blood transfusions and improve their quality of life."
Dr. Daryl Tan of Clinic for Lymphoma, Myeloma and Blood Disorder, Singapore, said: "The approval of Omjjara marks an important step forward for patients with myelofibrosis, especially those with moderate to severe anaemia who require new treatment options to alleviate the challenges of the disease. The introduction of this single therapy addressing all key manifestations of myelofibrosis is a significant advancement for patients with myelofibrosis."
GSK's submission for the approval of Omjjara was supported by data from the pivotal MOMENTUM study and a subpopulation of adult patients with anaemia from the SIMPLIFY-1 phase III 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 anaemic, 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 momelotinib versus danazol. 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. Safety and efficacy results for SIMPLIFY-1 were based upon a subset of patients with anaemia.[4]
The most common adverse reactions following use of Omjjara were diarrhoea, thrombocytopenia, nausea, headache, dizziness, fatigue, asthenia, abdominal pain, and cough.[4]
Myelofibrosis can lead to low blood counts, including anaemia and thrombocytopaenia; constitutional symptoms such as fatigue, night sweats, and bone pain; and splenomegaly; about 40% of patients globally have moderate to severe anaemia at the time of diagnosis, and nearly all patients are estimated to develop anaemia over the course of the disease. [5-8] (Image c/o GSK)
Artistic visual representation of myelofibrosis. Myelofibrosis is a rare blood cancer that results from dysregulated JAK-signal transducer and activator of transcription protein signalling and is characterised by constitutional symptoms, splenomegaly, and progressive anaemia. [1,19,20] (Image c/o GSK)
About myelofibrosis Myelofibrosis is a rare blood cancer that results from dysregulated JAK-signal transducer and activator of transcription protein signalling and is characterised by constitutional symptoms, splenomegaly, and progressive anaemia.[1,19,20]
About the pivotal MOMENTUM clinical trial MOMENTUM was a phase III, global, multicentre, randomised, double-blind study investigating momelotinib versus danazol in patients with myelofibrosis who were symptomatic and anaemic and had been previously treated with an approved JAK inhibitor. The trial was designed to evaluate the safety and efficacy of momelotinib for treating and reducing key hallmarks of the disease: symptoms, blood transfusions (due to anaemia) and splenomegaly.[2] Results from the 24-week treatment period were presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting and subsequently published in The Lancet.[21,22]
About the SIMPLIFY-1 clinical trial SIMPLIFY-1 was a multicentre, randomised, double-blind, phase III study that compared the safety and efficacy of momelotinib to ruxolitinib in patients with myelofibrosis who had not received prior treatment with a JAK inhibitor. Safety and efficacy results for SIMPLIFY-1 were based upon a subset of patients with anaemia (haemoglobin <10 g/dL) at baseline. The efficacy of momelotinib in the treatment of patients with myelofibrosis in SIMPLIFY-1 was based on spleen volume response (reduction by 35% or greater).[18]
About Omjjara (momelotinib) Omjjara has a different mechanism of action, with inhibitory ability along three key signalling pathways: Janus kinase (JAK) 1, JAK2, and activin A receptor, type 1 (ACVR1).[2,20,23,24] Inhibition of JAK1 and JAK2 may improve constitutional symptoms and splenomegaly.[2,20,24] Additionally, inhibition of ACVR1 leads to a decrease in circulating hepcidin, which is elevated in myelofibrosis and contributes to anaemia.[2,20,23,24]
GSK in Oncology GSK is committed to maximising patient survival through transformational medicines, with a current focus on breakthroughs in immuno-oncology and tumour-cell targeting therapies, and development in haematologic malignancies, gynaecologic cancers, and other solid tumours.
About GSK GSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. Find out more at https://www.gsk.com/en-gb/locations/singapore/
References
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2. Verstovsek S, et al. MOMENTUM: momelotinib vs danazol in patients with myelofibrosis previously treated with JAKi who are symptomatic and anemic. Future Oncol. 2021;17(12):1449-1458.
3. Kuykendall AT, Shah S, Talati C, et al. Between a rux and a hard place: evaluating salvage treatment and outcomes in myelofibrosis after ruxolitinib discontinuation. Ann Hematol. 2018;97(3):435-441.
5. Tefferi A, Lasho TL, Jimma T, et al. One thousand patients with primary myelofibrosis: the mayo clinic experience. Mayo Clin Proc. 2012;87(1):25-33. doi:10.1016/j.mayocp.2011.11.001
7. Scherber, R.M., Mesa, R. Management of challenging myelofibrosis after JAK inhibitor failure and/or progression. Blood Rev. 2020;42:100716. https://doi.org/10.1016/j.blre.2020.100716
8. Bassiony S, Harrison CN, McLornan DP. Evaluating the Safety, Efficacy, and Therapeutic Potential of Momelotinib in the Treatment of Intermediate/High-Risk Myelofibrosis: Evidence to Date. Ther Clin Risk Manag. 2020;16:889-901. Published 2020 Sep 25. doi:10.2147/TCRM.S258704
9. Tefferi A, et al. Use of the Functional Assessment of Cancer Therapy--anemia in persons with myeloproliferative neoplasm-associated myelofibrosis and anemia. Clin Ther. 2014;36(4):560-566. https://doi.org/10.1016/j.clinthera.2014.02.016
10. Tefferi A. Primary myelofibrosis: 2021 update on diagnosis, risk-stratification and management. Am J Hematol. 2021;96(1):145-162. https://doi.org/10.1002/ajh.26050
11. Rumi E, et al. The Genetic Basis of Primary Myelofibrosis and Its Clinical Relevance. Int J Mol Sci. 2020;21(23):8885. https://doi.org/10.3390/ijms21238885
12. How J, Hobbs GS. A Practical Guide for Using Myelofibrosis Prognostic Models in the Clinic. J Natl Compr Canc Netw. 2020;18(9):1271-1278. https://doi.org/10.6004/jnccn.2020.7557
14. QxMD. DIPSS plus score for prognosis of myelofibrosis.
15. Nicolosi M, et al. Sex and degree of severity influence the prognostic impact of anemia in primary myelofibrosis: analysis based on 1109 consecutive patients. Leukemia. 2018;32(5):1254-1258. https://doi.org/10.1038/s41375-018-0028-x
16. Elena C, et al. Red blood cell transfusion-dependency implies a poor survival in primary myelofibrosis irrespective of IPSS and DIPSS. Haematologica. 2011;96(1):167-170. https://doi.org/10.3324/haematol.2010.031831
17. Passamonti, F., Harrison, C. N., Mesa, R. A., Kiladjian, J. J., Vannucchi, A. M., & Verstovsek, S. (2022). Anemia in myelofibrosis: Current and emerging treatment options. Critical Reviews in Oncology/Hematology, Elsevier.
18. Mesa RA, Kiladjian JJ, Catalano JV, et al. SIMPLIFY-1: A Phase III Randomized Trial of Momelotinib Versus Ruxolitinib in Janus Kinase Inhibitor-Naïve Patients With Myelofibrosis. J Clin Oncol. 2017;35(34):3844-3850.
19. Data on file. Sierra Oncology. 2021.
20. Chifotides, HT, Bose, P, Verstovsek, S. Momelotinib: an emerging treatment for myelofibrosis patients with anemia. J Hematol Oncol. 2022;15(7):1-18.
21. Mesa R, et al. Presented at: American Society of Clinical Oncology; June 2022. Abstract 7002.
22. Verstovsek S, et al. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study. The Lancet. 2023;401(10373):269-280.
23. Asshoff M, et al. Momelotinib inhibits ACVR1/ALK2, decreases hepcidin production, and ameliorates anemia of chronic disease in rodents. Blood. 2017;129(13):1823-1830.
24. Oh S, et al. ACVR1/JAK1/JAK2 inhibitor momelotinib reverses transfusion dependency and suppresses hepcidin in myelofibrosis phase 2 trial. Blood Adv. 2020;4(18):4282-4291.
